Economic Viability Policy Analysis

Chapter 12 The Policy Analysis Process: Analysis of Values

An analysis that considers technological capabilities, economic outcomes, and political support will usually point toward a single health policy recommendation. Yet any final recommendation must take into account values, especially where there are competing trade-offs. You might ask this: Does not the political process, which a recommendation often goes through before it can be adopted and implemented, take care of those value concerns? Do not decision makers reflect their personal values and those of their constituencies in the positions they take during a policy debate and in their votes? Yes, the political process will reflect the current wider conflicts in American society over social mores, the nature of human life, the role of government, the economic marketplace, and collective versus individual rights. In this chapter, however, we consider some value issues relating specifically to health care and the health care professions. These values may or may not be considered in the political process and should be assessed as part of any robust analysis.

Value issues discussed briefly in this chapter include:

• Equitable access

• Efficiency and value

• Patient privacy and confidentiality

• Informed consent

• Personal responsibility

• Quality, variability, and malpractice reform

• Professional ethics

• Consumer sovereignty

Social welfare

• Rationing

• Process equity

This chapter ends with the broader question of how a wide range of social institutions might be influenced by the growth of the medical sector of the economy.

12.1 EQUITABLE ACCESS

Priester (1992) suggested that the United States should reorder its values to give the greatest emphasis to fair equity, which he defined as giving each individual access to an “adequate level of care.” Skipping over issues of residency and eligibility, he argued, “Assuring access to health care, regardless of cause or source of need, is society’s responsibility”; however, he added that “this does not require access to all potentially beneficial care” (p. 92). He maintained that this is the only approach that would not exceed the available resources or deprive some segments of society of their opportunity for a reasonably full life. He saw this as a floor, not a ceiling; someone with more personal resources could choose to consume more health services. He saw the U.S. system as overemphasizing provider autonomy, individualism, and assumed abundance. He felt that we have too often let economic considerations outweigh ethical ones.

A similar argument could be made for removing health disparities; among the major causes of which is differential access to care. The Affordable Care Act (ACA) has made a step in the direction of fair access by moving toward universal coverage and by calling for a defined essential benefit package.

12.2 EFFICIENCY AND VALUE

If resource scarcity leads society to overlook some populations or to restrict necessary services to them, then efficiency is important. “Waste not, want not” is not always a fundamental value in a system in which one person’s waste is another person’s enhanced income. Porter and Teisberg (2006) argued against a “zero-sum” mentality that attempts to maximize each individual provider’s share of the existing pie and favored reducing waste to enhance the value received by individual consumers. One could also promote the collective view that the services available to consumers collectively form a “zero-sum” game in which all will get more if the collective waste is minimized. One way or another, there is considerable merit in viewing waste negatively, rather than as enhanced income.

Because it is so hard to get individuals to pull together on efficiency and cost minimization, some policy experts have tended to emphasize “value.” We now have value-based compensation experiments that allow sharing of the savings with providers and provide compensation for higher-quality outcomes. The sense here is that there is value created when you get greater quality for the same cost or the same quality for lower cost, or both positive outcomes concurrently.

In a survey that asked health industry leaders to rank 11 areas of innovation for their potential impact in terms of quality and cost over the next 5 years, the highest ranking went to process improvement. The other innovation areas likely to contribute to both improved quality and cost were data analytics, nonphysician delivery alternatives, disease management, and alternatives to fee-for-service. The respondents believed innovations in diagnostics, pharmaceuticals, and electronic medical records would help with costs but not quality, and they forecasted a low likelihood of help along either dimension from consumer incentives or from basic research (Chin

et al., 2013). The good news is that process improvement is an area where any provider who wants to make a difference can contribute.

12.3 PATIENT PRIVACY AND CONFIDENTIALITY

Increasingly, electronic patient records, digitized information already collected for billing and claims, and specialized databases offer potential for finding out more about disease processes and care outcomes. At the same time, they offer possibilities for excluding individuals from care or for breeches of the confidentiality that one expects when encountering the health care system and is often guaranteed by laws such as the Health Insurance Portability and Accountability Act (HIPAA). News stories about stolen laptops with personal data crop up frequently. This is an area where trade-offs will continue to be difficult and frustrating, and it will continue to be important in policy analysis and decision making.

12.4 INFORMED CONSENT

Requirements for informed consent for patients and human subjects in research represent a constraint on provider autonomy. They add to the staff burden, but are a regulatory requirement. Table 12-1 illustrates part of the federal regulations governing informed consent by research subjects in federally funded research. You might ask yourself this: What values are represented here, and why were they made an added requirement of all research in the first place?

Table 12-1 Code of Federal Regulations Section 50, Subpart B, Informed Consent of Human Subjects

§50.20: General requirements for informed consent

Except as provided in 50.23 and 50.24, no investigator may involve a human being as a subject in research covered by these regulations unless the investigator has obtained the legally effective informed consent of the subject or the subject’s legally authorized representative. An investigator shall seek such consent only under circumstances that provide the prospective subject or the representative sufficient opportunity to consider whether or not to participate and that minimize the possibility of coercion or undue influence. The information that is given to the subject or the representative shall be in language understandable to the subject or the representative. No informed consent, whether oral or written, may include any exculpatory language through which the subject or the representative is made to waive or appear to waive any of the subject’s legal rights, or releases or appears to release the investigator, the sponsor, the institution, or its agents from liability for negligence.

Section 50.24 Basic elements of informed consent.

(a) In seeking informed consent, the following information shall be provided to each subject:

(1) A statement that the study involves research, an explanation of the purposes of the research and the expected duration of the subject’s participation, a description of the procedures to be followed, and identification of any procedures which are experimental. (2) A description of any reasonably foreseeable risks or discomforts to the subject. (3) A description of any benefits to the subject or to others which may reasonably be expected from the research. (4) A disclosure of appropriate alternative procedures or courses of treatment, if any, that might be advantageous to the subject. (5) A statement describing the extent, if any, to which confidentiality of records identifying the subject will be maintained and that notes the possibility that the Food and Drug Administration may inspect the records. (6) For research involving more than minimal risk, an explanation as to whether any compensation and an explanation as to whether any medical treatments are available if injury occurs and, if so, what they consist of, or where further information may be obtained. (7) An explanation of whom to contact for answers to pertinent questions about the research and research subjects’ rights, and whom to contact in the event of a research-related injury to the subject. (8) A statement that participation is voluntary, that refusal to participate will involve no penalty or loss of benefits to which the subject is otherwise entitled, and that the subject may discontinue participation at any time without penalty or loss of benefits to which the subject is otherwise entitled. (b) Additional elements of informed consent. When appropriate, one or more of the following elements of information shall also be provided to each subject: (1) A statement that the particular treatment or procedure may involve risks to the subject (or to the embryo or fetus, if the subject is or may become pregnant) which are currently unforeseeable. (2) Anticipated circumstances under which the subject’s participation may be terminated by the investigator without regard to the subject’s consent. (3) Any additional costs to the subject that may result from participation in the research. (4) The consequences of a subject’s decision to withdraw from the research and procedures for orderly termination of participation by the subject. (5) A statement that significant new findings developed during the course of the research which may relate to the subject’s willingness to continue participation will be provided to the subject. (6) The approximate number of subjects involved in the study. (c) When seeking informed consent for applicable clinical trials, as defined in 42 U.S.C. 282(j)(1)(A), the following statement shall be provided to each clinical trial subject in informed consent documents and processes. This will notify the clinical trial subject that clinical trial information has been or will be submitted for inclusion in the clinical trial registry databank under paragraph (j) of section 402 of the Public Health Service Act. The statement is: “A description of this clinical trial will be available on http://www.ClinicalTrials.gov, as required by U.S. Law. This Web site will not include information that can identify you. At most, the Web site will include a summary of the results. You can search this Web site at any time.”

(d) The informed consent requirements in these regulations are not intended to preempt any applicable Federal, State, or local laws which require additional information to be disclosed for informed consent to be legally effective. (e) Nothing in these regulations is intended to limit the authority of a physician to provide emergency medical care to the extent the physician is permitted to do so under applicable Federal, State, or local law. Source: Reproduced from: Code of Federal Regulations (2013, April 1). Protection of human subjects. Washington, D.C: Government Printing Office. Title 21, Volume 1, Part 50. Retrieved on December 17, 2013, at www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=50&showFR=1&su bpartNode=21:1.0.1.1.20.2.

12.5 PERSONAL RESPONSIBILITY

A significant portion of the cost of health care can be attributed to lifestyle choices, such as smoking, lack of exercise, overeating or poor nutrition, not wearing seat belts or cycling helmets, and use of drugs and alcohol. Many policy proposals seek to change the behavioral risk factors or shift those costs to the individuals at risk. Smokers pay higher insurance premiums for long-term care, for example. Some analysts have suggested that those involved in risky behaviors, such as not wearing motorcycle helmets, post a bond to cover their incremental medical care costs in case of an accident. Most people would agree that individuals should take more responsibility for their behavior rather than have it borne as a collective risk, but there is less agreement on the effort society should expend to make healthy choices more attractive. There is considerable evidence, for example, that the development choices we make—our built environment—can encourage or discourage physical activity. A politician asked to support a bill to encourage “walkability” or “multimodal transportation hubs,” however, may be inclined to attribute lack of exercise solely to, as one state legislator put it, “a lack of personal fortitude.”

The question of personal responsibility comes up repeatedly in debates about health reform and the ACA in particular. Key elements of the reform can be seen as an attempt to balance a society’s responsibility to ensure equitable access with an individual’s responsibilities. The individual mandate legislates personal responsibility, using tax fines to ensure that young, healthy people can pay for their own care if they need it and do their share to support a larger system they will likely rely on as they age. When Governor Mitt Romney was considering the individual mandate as part of the Massachusetts reforms, he noted that the personal mandate was about personal responsibility, which is a fundamental Republican value (Starr, 2011). Yet, in a video released by Mother Jones during the campaign, Romney said that 47% of the population feel entitled to government handouts. “I’ll never convince them that they should take personal responsibility and care for their lives,” he said (Mother Jones, 2012). In his acceptance speech after winning another term, President Obama emphasized social responsibility:

What makes America exceptional are the bonds that hold together the most diverse nation on earth. The belief that our destiny is shared; that this country only works when we accept certain obligations to one another and to future generations. The freedom which so many Americans

have fought for and died for come with responsibilities as well as rights. And among those are love and charity and duty and patriotism. That’s what makes America great. Previous sectionNext section

12.6 QUALITY, VARIABILITY, AND MALPRACTICE REFORM

We hear a lot about malpractice reform and frivolous lawsuits and unreasonable awards for pain and suffering. We see local television advertisements by law firms seeking to represent clients “wronged” by providers and insurers. State and federal legislatures and courts debate whether to cap the size of awards or determine when they are excessive. Funding flows freely into campaign coffers from organizations on both sides of the debate; trial lawyers favor the Democratic Party, whereas insurers and organizations representing health providers and facilities favor the Republican Party. Putting political posturing aside, however, several values-related policy issues recur in the debates on this area, including the following:

• The value of a human life lost and of other negative consequences suffered unnecessarily

• The allocation of the responsibility for error between the individual provider and the overall care system

• The amount of variability in outcomes and events that is unavoidable and how much is unacceptable

• The appropriate way to compensate advocates for patient rights and to overcome inappropriate provider behavior

• How much to let the provider community police itself and how much and when to intervene in the public interest. 12.7 PROFESSIONAL ETHICS

Access and rationing are aspects of distributive justice. Confidentiality, truthfulness, informed consent, respect for patient and professional autonomy, and the safety of the patient are all topics cited in discussions of professional ethics and in professional codes of ethics. Most professionals have had some indoctrination in biomedical ethics and are aware of key issues. Most professions have an ethical code or statement. Most such statements have become more general over time as patient autonomy has become more respected and monopolistic practices have come under government scrutiny. At the same time, professional societies may take strong positions without incorporating them into a code. For example, the American Nurses Association has long supported health care as a basic right delivered through a single-payer system.

What About Health Policy Experiments?

Daniels (2006) raised an interesting ethical question for health policy professionals: What if we required that health system transformations had to undergo an ethical review process similar to

that required of medical research experiments? He noted that these social experiments can put significant populations at risk. He did not suggest what the mechanism for such reviews might be; however, he raised the issues of balancing the social value of the experimental policy changes with the risks to which those affected are exposed and how we might go about conducting such a review. He suggested three levels of analysis:

1. Select benchmarks for the ethical analysis in terms of their effect on:

a. Equity b. Efficiency c. Accountability 2. Conduct an ethical evaluation of how well the design of the proposed reform meets its goals:

a. How good is the evidence used to justify the intervention? b. Is the implementation planned adequate to test the results? c. Will the intervention measure the effect with sufficient sensitivity to evaluate the results? d. Are the key implementers involved in the planning and committed to an unbiased evaluation? 3. Determine whether there is sufficient oversight to protect the rights of those involved in the experiment. Informed consent might be impractical in many settings.

He mostly offered examples of international situations, but, overall, he noted, “Unfortunately, there is little experience in measuring how systems establish transparency, accountability, and fair process in decisions involving resource allocation” (p.450).

12.8 CONSUMER SOVEREIGNTY

Those who want a highly competitive marketplace want the consumer to make decisions rather than the government. The examples in the box on the next page illustrate views of health policy analysts on both sides of this economic and political ideological divide. The typical health professional is in a bit of a straddle here. Most believe in patient autonomy but also recognize that consumer sovereignty often comes at the expense of professional power and influence, which some would call paternalism, but may positively affect patient compliance and clinical outcomes as well.

12.9 SOCIAL WELFARE

Interest groups at the table during policy formulation may or may not adequately represent the public’s interests. There are a number of ways of evaluating outcomes in terms of social welfare. Some are economic; some are not. Economic models that would base policy decisions on benefit–cost or cost-effectiveness criteria raise issues of valuation, such as

CONTRASTS IN ECONOMIC AND POLITICAL IDEOLOGY

Cannon and Tanner (2005) agreed in one respect with many who support a greater government role in health: that the health care sector of the economy is special. But they argued that because it is special, reliance on competition is even more critical:

Unlike software, wireless communications, or banking, health care involves very emotional decisions, which often entail matters of human dignity, life, and death. However, we do not see the gravity of these matters as a reason to divert power away from individuals and toward government. Rather we see the special nature of health care as all the more reason to increase each consumer’s sphere of autonomy. (pp. 146–147)

Richmond and Fein (2005) concluded otherwise. They would prefer to use public policy to create a more equitable society:

Health and health care are vitally important in influencing life’s chances and one’s income and wealth should not determine the amount and quality of care one receives. We seek a system in which the financing and distribution of health services reflect our image of a just society, a society in which economic arrangements reflect a moral dimension. (p. 4)

• Willingness to pay

• Contribution to gross domestic product

• Social costs avoided, as in the case of support of a family when the breadwinner dies

At the same time there are other considerations, such as

• Lives saved

• Quality-adjusted life-years (QALYs)

• Longevity

• Pain and suffering avoided

In this arena, there are counterattacks to the economic arguments (Ackerman & Heinzerling, 2004):

Cost–benefit analysis of health and environmental policies trivializes the very values that gave rise to these policies in the first place. Moreover, through opaque and intimidating concepts like willingness to pay, quality-adjusted life-years, and discounting, economic analysts have managed to hide the moral and political questions lying just under the surface of their precise and scientific-looking numbers. It is time to blow their cover. (p. 234)

Discounting is easy to do mathematically but difficult to interpret practically, politically, and ethically. The problem is in the trade-off between the present and the future, a difficult problem in all policy making. The standard economists’ approach of bringing the costs and benefits back to a net present value explicitly biases the analysis against future events. Some advocates, therefore, argue for a discount rate of 0%.

Ackerman and Heinzerling also questioned the use of QALY metrics because they discriminate against the older population, who will naturally have fewer years ahead of them. The same can be said of any analysis dealing with contribution to gross domestic product, because the older population generates little output or will soon stop generating it and the output of children is so far into the future that any reasonable discount rate obliterates the benefits. This is why some suggest that economic analysis is useful for payers and for comparing treatment alternatives for specific illness, but that it really does not work well when comparing an array of different alternative public investments.

12.10 RATIONING

Bodenheimer and Grumbach (2005) suggested a two-part definition of rationing: (1) limiting care that is likely to be beneficial due to scarce resources, including money, and (2) a method of fairly distributing the resources that are available. To this we would add a third condition: (3) that the decision-making method is determined by society rather than a corporation or an individual. Bodenheimer and Grumbach noted that popular use of the term rationing tends to equate it with withholding of care and ignores the second necessary condition. That distinction separates arbitrary actions to decrease the cost of care from situations in which the system attempts to distribute its limited resources in a systematic and equitable way. There are a variety of notions of equity, and thus a rationing system may be designed to

• Reduce overall costs to a targeted level

• Maximize access

• Maximize the social welfare based on the contributions of the individuals within the population

• Maximize medical effectiveness:

• Subject to a cost constraint

• Subject to other resource limitations

• Combinations of the above

For example, the attempt of the British National Health Service (NHS) to limit the costs of care has led to at least one charge of rationing in which a woman with breast cancer was denied the

drug Herceptin by a local health authority on the basis of cost. An account of this has been included in Table 12-2.

Reducing Overall Health Care Costs to a Target Level

An effective rationing system is not aimed at minimizing health care costs. They cannot be driven to zero; however, when there is a budgetary limitation or a fixed-revenue situation, a system must be in place to decide which services will not be provided to which individuals. In many cases, this is done by limiting the population served and limiting the services offered.

For example, one of the largest Medicaid expenses is paying for longterm care for older people, a service not covered by Medicare. States have quietly limited the amount they pay by limiting the number of new nursing home beds they license. They know that there is a strong linear relationship between the number of beds available and the amount of Medicare claims received.

Maximize the Social Welfare

When transplanted organs first became available, it was clear that there were not enough to go around. Institutions doing transplants set up committees of individuals concerned with medical effectiveness and medical ethics to determine who would get the next available organ. This process provides an example of how social welfare considerations can influence rationing and access decisions. The committees examined a mixture of personal, family, and medical data to determine literally who should live and who would, in all probability, die. Considerations included family status, work status, medical factors such as alcoholism and comorbidities, and psychiatric factors. Outside observers were also suspicious that ability to pay might be creeping into the decision making.

Table 12-2 Rationing and the Courts

Ann Marie Rogers, 54, sued the Swindon Primary Care Trust after it refused treatment for her early-stage HER2 breast cancer with the drug Herceptin (trastuzumab), even after her doctor prescribed it. The Roche drug is licensed for late-stage breast cancer, but some studies showed it to be effective for early-stage cancer as well. Ms. Hewitt, the British Health Secretary, had praised the efforts of other women to get the treatment, and the Health Department had ordered the local health services “not to withhold it solely on the grounds of cost,” even while a government-appointed review panel was in the process of setting new guidelines for the use of the drug. Treatment with Herceptin costs $36,000–47,000 a year for each patient and was supplied under very different circumstances by the local health trusts. The Swindon trust had a policy of supplying Herceptin for early-stage breast cancer only in “exceptional circumstances.” Ms. Rogers had pointed to her cancer being of the type responsive to the drug and the risk factors of the death of her mother and a cousin from cancer.

When the trust refused to fund her treatment, Ms. Rogers starting paying for the drug on her own, but had to stop when her own resources were used up. Thus, she sued the authority, citing

the “postal code lottery,” which determined who got treated and who did not. A lower court judge ruled in February 2006 that the Swindon health service’s denial was lawful. In April, however, a three-judge appeals court overturned that verdict and said the local health service had acted unlawfully, noting that “once the Primary Care Health Trust decided, as it did, that it would fund Herceptin for some patients and that the cost was irrelevant, the only reasonable approach was to focus on the patient’s clinical needs and fund patients within the eligible group who were properly prescribed by their physician.” It observed that there had been “no rational basis for distinguishing between patients within the eligible group on the basis of exceptional clinical circumstances any more than on the basis for personal, let alone, social circumstances” (Lyall, 2006). The National Institute for Clinical Excellence (NICE) issued a press release on April 12, 2006, in which NICE Chief Executive Andrew Dillon reaffirmed the importance of ensuring that new drugs are both safe to use, by having an effective system for licensing, and used in the right way through the work that NICE does. “Without these things, we risk exposing patients to risks and the health service to using its money unwisely,” he said (NICE, 2006a). After a public comment period that closed in June 2006, NICE issued guidance in August 2006. “Trastuzumab, given at 3-week intervals for 1 year or until disease recurrence (whichever is the shorter period), is recommended as a treatment option for women with early-stage HER2-positive breast cancer following surgery, chemotherapy (neoadjuvant or adjuvant), and radiotherapy (if applicable)” (NICE, 2006b, p.4). In June 2012, NICE again addressed the breast cancer issue with Technical Appraisal 257, “Breast cancer (metastatic hormone receptor)—lapatinib and trastuzumab (with aromatiase inhibitor).” It stated that NICE did not recommend this as first-line treatment for post-menopausal women who have metastatic breast cancer that is hormone-receptor and HER2 positive. It explained its response as follows: NICE looks at how well treatments work, and also at how well they work in relation to how much they cost the NHS. NICE applies special considerations to treatments that can extend the lives of people who are nearing the end of their life. Lapatinib and trastuzumab (herceptin) do not provide enough benefit to patients to justify their high cost even when the special considerations were applied, so NICE did not recommend them. (NICE, 2012) One problem with the social welfare criterion is that it goes well beyond issues that health professionals are comfortable with. Issues might include citizenship status, economic contribution, utility of one’s work, and emotional impact on others. Reaching some agreement on the importance of each of these is unlikely given current value conflicts in our society. That does not mean that we cannot reach a social consensus, but rather that it would be very difficult to justify once we have reached it.

Maximize Utility and Medical Effectiveness

“The providing or withholding of care is ideally determined by the probability that the treatment will maximize benefits and minimize harm, i.e. by the criterion of medical effectiveness” (Bodenheimer & Grumbach, 2005, p. 137). This concept was operationalized in the Oregon Basic Health Services Act of 1989, which tried to guarantee health care for all and still control health care costs through an open, publicly accountable rationing process. At that time, one had

to be at or below 58% of the federal poverty level to receive Medicaid benefits in Oregon, and this bill was intended to open that up to those below the 100% level. The state created a Health Services Commission to recommend the prioritization of health services. The 11-member body was to report its priorities to the governor and the Joint Legislative Committee on Health Care. An actuarial contractor would then estimate the cost of each of the prioritized services for the committee. After the Medicaid budget for services was set, the package of services offered to the Medicaid population would be developed using the priority lists. Essentially, the process would be to go down the list taking the next highest priority coverage until the estimated budget package was exhausted. Other services then would be outside this basic coverage. If the demand exceeded the funding provided, Oregon was prepared to reduce the benefit package rather than ration through eligibility thresholds or Medicaid reimbursement levels, as so many other states were doing (Calkthan, 1991).

The commission used three techniques to develop the prioritization of some 800 services. It held 11 open public hearings around the state. It also had Oregon Health Decisions, a respected advocacy group, conduct 47 community forums, including one in every county. Each forum featured a slide presentation, group discussions, and a questionnaire on the participants’ opinions about the relative importance of specific health situations and categories. About 70% of the more than 1,000 individuals attending these forums were health care workers. The commission also supported a statewide random-digit dialing survey asking 1,000 individuals to rate 31 health care situations on a modified Quality of Well-Being (QWB) scale that had been validated elsewhere. That information was used to build a costutility scale called a “net benefit value” scale. That study produced a listing of 1,600 medical condition–treatment pairs. The orderings produced by the telephone survey were highly debatable. Fox and Leichter (1991) reported that “crooked teeth received a higher ranking than early treatment for Hodgkin’s disease, and dealing with thumb sucking was ranked higher than hospitalization of a child for starvation” (p. 22). Because it was clear that the derived utilities were not workable in terms of overall values, commission members negotiated among themselves for an acceptable ordering. For example, the four consumer representatives on the panel argued for higher rankings for preventive services because that is what the public seemed to have been saying during this process. Rather than a continuous ranking, they came up with 709 pairs grouped according to three basic categories: essential, very important, and valuable to some individuals (Kaplan, 1995), and then under 17 subcategories of outcomes, using descriptors such as survival, degree of recovery, and degree of improvement on the QWB scale (Fox & Leichter, 1991).

Implementing this process required a federal Medicaid waiver, which the federal government denied on the grounds that the assessments of quality of life by healthy individuals tended to stereotype and discount the value of quality for the disabled, and the process, therefore, violated the Americans with Disabilities Act. Kaplan (1995) argued that this finding was in error and offered counterevidence.

Instead of debating these issues, Oregon chose to resubmit its application with the utility portion of the model excluded. Its revised waiver application considered probability of death and probability of moving up from a symptomatic to an asymptomatic state. By giving up the utility

component of the model, Oregon ignored the fact that health states are valued (Kaplan, 1995). Priester (1992) argued that the plan had been flawed because it denied access to a reasonable level of care to some, but not to others. In 1993, the Clinton administration approved the revised Medicaid waiver.

Combinations of One or More

Table 12-3 contains a description of how the United Network for Organ Sharing (UNOS) allocates available organs (not including related donors). This contractor was set up after passage of the National Organ Transplantation Act of 1984. It emphasizes the likely medical outcome and then pays attention to one’s length of time on the waiting list; however, the issue of whether the transplant takes place is also contingent on the availability of private insurance or Medicare or Medicaid funds to pay for it. By now you should be aware that trying to maximize one thing while trying to minimize or maximize another is often a mathematical impossibility. It is legitimate, however, to have a system in which one objective is traded off against another. The problem is that most attempts to do so never specify what the trade-off ratios are because trying to arrive at a consensus ratio is likely to generate conflict over the weightings that cannot be resolved without considerable loss of momentum and goodwill. Allocation rules, therefore, tend to remain fuzzy. You can see this even in the UNOS description. It is clear that the concept of a social welfare criterion is not really operative there.

Table 12-3 How the Transplant System Works: Matching Donors and Recipients

Under the Organ Procurement and Transplantation Network (OPTN) contract with the U.S. Department of Health and Human Services’ Health Services & Resources Administration, UNOS maintains a centralized computer network linking all organ procurement organizations and transplant centers. This computer network is accessible 24 hours a day, 7 days a week, with organ placement specialists in the UNOS Organ Center always available to answer questions.

A person who may benefit from a transplant is referred by his or her doctor to a transplant center, which evaluates the patient. The transplant center runs a number of tests and considers the patient’s mental and physical health, as well as his or her social support system. If the center decides to accept this person as a transplant candidate, it will add his or her medical profile to the national patient waiting list for organ transplant. The candidate is not placed on a ranked list at that time. Rather, his or her information is kept in a constantly updated, computerized database. When a deceased organ donor is identified, a transplant coordinator from an organ procurement organization accesses the UNOS computer. Each patient in the database in matched against the donor characteristics. The computer then generates a ranked list of candidates for each available organ in ranked order according to OPTN organ allocation policies. The match for each donor would be different and unique to the circumstances of the donor and the patients waiting. Factors affecting ranking may include tissue match, blood type, length of time on the waiting list, immune status, and the distance between the potential recipient and the

donor. For heart, liver, lung, and intestines, the potential recipient’s degree of medical emergency is also considered. The organ is offered to the transplant team for the first person on the list. Often, the top patient will not get the organ for one of several reasons. When a patient is selected, he or she must be available, healthy enough to undergo major surgery, and willing to be transplanted immediately. Also, a laboratory test to measure compatibility between the donor and recipient may be necessary. For example, patients with high antibody levels often prove incompatible to the donor organ and cannot receive the organ because the patient’s immune system would reject it. Source: Reproduced from: United Network for Organ Sharing. (n.d.). How the Transplant System Works: Matching Donors and Recipients. Richmond, VA: UNOS. Retrieved on December 27, 2013, from www.unos.org/donation/index.php?topic=fact_sheet_1.

12.11 PROCESS EQUITY

Not only must the outcome of the policy analysis and selection process—that is, the policy itself—be perceived as fair, but so should the process that produced it. For example, before settling on a policy, the policy analysts must ask themselves whether all interested parties participated. If not, have the underrepresented or disenfranchised had their issues addressed by the analysts? This was a relevant concern in the Oregon process, which, although highly rational, seemed dominated by health professionals and by more educated and civically involved individuals. It is not unusual for opponents of a policy to argue against the inclusiveness or integrity of the policy development process. Many governments have requirements for public hearings that are regionally representative in hopes that their process will be fairer to those concerned and to protect themselves against complaints about the process.

12.12 INFLUENCE ON SOCIETY: A BROADER QUESTION

With the medical care sector headed toward constituting 20% of the economy, we have to wonder about the long-term effects of this much emphasis on health in our society. Areas of concern might include:

• A focus on illness and the possible loss of a sense of well-being

• Commercial pressure to overemphasize medical problems

• The power of the medical–industrial–university complex

Is it healthy to be so conscious of our health? Is it healthy to be so stressed about our medical bills? Just about every sector of our society adds a health concern to its primary and previous missions. Churches add health ministry committees and/or health-trained staff members. Schools take on more responsibility in terms of children’s medications and their special needs; some have even established school-based health clinics. Tax revenues previously devoted to education and infrastructure get diverted to health care. All of these individual decisions may be

virtuous, but they also signal a changing societal focus from toughing it out to getting it fixed through health care, whether that works or not.

Because health care represents an opportunity for growth, we see more and more commercial pressure to expand its markets—and its “top of mind” awareness for the public. In a way, this is good because it creates more innovation. However, it can create demand for which there is not a significant need and siphon resources away from areas of true need. Two issues that illustrate this are the explosive growth in direct-to-consumer advertising, especially on television, and the creation of new syndromes to justify new products, such as the hotly debated emergence of prehypertension.

PREHYPERTENSION

Many older doctors report that they were trained that the normal upper limit for systolic blood pressure was your age (at least up to 55 years) plus 100 millimeters of mercury over 90 (diastolic). Then, in 1977, the Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure issued its first report establishing the guideline of 120/80 mm Hg as optimal, 120–129/80–84 mm Hg as within the normal range, and 130–139/85–89 mm Hg as high normal. Then, in May 2003, it issued a new guideline stating that levels above 120/80 mm Hg constituted prehypertension and indicated increased risk for heart attack, stroke, or kidney disease. This new diagnostic entity was to be treated with the same methods as before—diet, exercise, and other lifestyle changes and, if these fail to do the trick, a diuretic. Critics argue that there is no randomized clinical trial evidence below 140 mm and suggest that the pharmaceutical industry, which might double its number of treatable patients, not only approved of the decision, but may have influenced it. They note that reducing salt intake will help some, but hinder others, and that treating blood pressure “by the numbers” is not good medicine, although it is tempting when a provider is pressed for time and faced with patients who expect a prescription. They point out that hypertension can have a number of causes and that it should be viewed as part of a “mosaic” of risk factors associated with cardiovascular care.

In November 2013, the American Heart Association and the American College of Cardiology issued a new set of joint guidelines for cholesterol treatment, calling for treatment for anyone with a 10-year risk factor for a cardiac event of 7.5% or more. The two organizations also issued a new risk calculator, a spreadsheet that considers age, race, cholesterol levels, blood pressure, and other factors. The risk calculator combined with the new threshold will result in a dramatic increase in the number of individuals that should be treated. The risk calculator came under immediate criticism because reviewers said the model overpredicted risk by 75% or more. The main complaint was that it relied on outdated demographic data.

Most of the public discussion focused on the use of statins to lower cholesterol, but treatment could also include prescribing drugs that lower blood pressure. Dr. H. Gilbert Welch, a Dartmouth medical professor, said the calculator treated the relationship between blood pressure and heart risk as if it was a straight line. “The model suggests that lowering systolic

blood pressure from 130 to 100 is nearly as important as from 180 to 150,” he told the New York Times. “I doubt there is a cardiologist that believes that” (Kolata, 2013).

A good classroom exercise would be to search out other newly emphasized conditions and track down their medical and commercial origins. You can start by turning on your television and noting all the ads for drugs that one must take regularly to suppress some distress such as acid reflux, erectile dysfunction, or insomnia.

In the United States, much debate centers on pharmaceutical industry advertising aimed directly at consumers, the cost of which ranges between $3 billion and $5 billion annually. Health care advertising, especially for prescription drugs, seems to have replaced all of the airtime and billboard space that had been taken up by cigarette and tobacco ads before they were banned. Are we that much better off from stopping one set of ads and substituting another? Other countries, including New Zealand, have barred this type of commercial activity.

Sterling offered a vision of a medically obsessed future in his novel Holy Fire (1996). In it, protagonist Mia Ziemann is 94 years old and still professionally active as a medical economist. In her world, wealth is all about how many medical procedures one can afford to extend one’s longevity. The medical–industrial–research complex clearly dominates the entire economy, and young people are disenfranchised because employment opportunities rarely open through retirement or death. It is a disturbing vision, and yet one that is strangely familiar and highly plausible because Sterling draws on existing trends to shape his view of the late 21st century. Indeed, his major failing as a futurist may have been underestimating the rate of change.

12.13 CONCLUSION

Values are the fourth leg of the analyst’s desk, working alongside technology, politics, and economics to support identifying a policy recommendation.

This chapter outlines a number of concerns that a policy analysis group must consider in addition to the other three as they factor in concern for the general welfare of our society and our democratic traditions. The issues of ethics and values are not just limited to professional decisions, but also play an important role in all analyses of policy alternatives.

Case 12 The Folic Acid Fortification Decision: Before and After BEFORE

At 5 p.m. on July 20, 1991, Dr. Godfrey P. Oakley, Jr., head of the Division of Birth Defects and Developmental Disabilities at the Centers for Disease Control and Prevention (CDC), took a phone call that, he says, “forever changed my life.” On the phone was a member of the British Medical Research Council (MRC) Vitamin Study Group, calling to share study results that would be published the following month in The Lancet (SerVaas & Perry, 1999).

The MRC study focused on women who had previously had a pregnancy in which the fetus or child had a neural tube defect (NTD), a birth defect of the spinal cord or brain. In April 1991, the MRC halted its study after almost 8 years because the data indicated that daily folic acid supplementation before pregnancy and during early pregnancy resulted in a 71% reduction in the recurrence of NTDs (CDC, 1991). It was no longer ethical not to provide folic acid to all of the women.

Dr. Oakley told the Saturday Evening Post Society in 1999,

Until that time, I thought that prevention of neural tube defects by taking folic acid supplementation was certainly no better than 50–50. If you really pressed me on what I would have thought the likelihood that a vitamin would have prevented neural tube defects, I would have said no more than 10% or 20%. But this was the first randomized, controlled trial designed and executed in a way that it proved folic acid would prevent spina bifida—not all but most of it. You could bet the farm that folic acid prevents neural tube defects. (SerVaas & Perry, 1999, p. 3)

The study ignited a policy debate lasting several years. In the United States, the question of how to increase folic acid consumption in woman of child-bearing years was contentious in itself, but the issue was complicated further by the controversy that surrounded implementation of the Nutrition Labeling and Education Act (NLEA) of 1990, which required the Food and Drug Administration (FDA) to regulate the health claims of food manufacturers.

Among the tools policy makers had to help them sift through their options were three economic analyses: cost–benefit analyses produced in 1993 and 1995 and a cost-effectiveness analysis completed in 1996. All three predicted positive net economic benefits from fortifying foods with folic acid.

BACKGROUND

NTDs are a class of birth defects that involve the brain and spinal cord. The most extreme form is anencephaly, in which all or part of the brain is missing. Another form is spina bifida, in which the spinal cord is not fully encased in the spine. In the early 1990s, there were about 4,000 NTD-associated pregnancies per year in the United States.

Folates are a form of B vitamin that occurs naturally in leafy vegetable, legumes, nuts, and other foods. Folic acid is its synthetic form. Folates help cells replicate quickly.

Reports that increased folic acid intake could help prevent birth defects date back to 1965 (Hibbard & Smithells, 1965). Vitamin manufacturers learned how to add it to their supplements in the 1970s. The Saturday Evening Post Society launched its campaign to promote folic acid supplementation in October 1982 in the wake of a report that women who took a vitamin supplement with 400 mcg of folic acid experienced fewer NTD-affected pregnancies. In the late 1980s, the CDC held a workshop to discuss emerging research linking folic acids to reductions in the numbers of NTDs, which renewed interest among supplement manufacturers.

Support for folic acid was limited, however. Post writers Cory SerVaas and Pat Perry (1999) reported that they were ridiculed for their efforts to promote folic acid. Scientific evidence was sparse, inconclusive, and based on observational epidemiological studies. Around the time of the CDC workshop, in fact, the National Research Council advised the FDA that it should lower the recommended daily allowance. A 1990 report by the Institute of Medicine’s Food and Nutrition Board called taking vitamin supplements to prevent NTDs “unjustified.” The National Academy of Sciences also discounted the link between NTDs and folic acid in its 1990 Report on Nutrition and Pregnancy.

In the midst of this debate, the NLEA skated through Congress and was signed by President George H. W. Bush. The act directed the FDA to establish standards for nutrition labels and define how certain terms such as “low fat” and “low cholesterol” could be used on food packaging. Congress also directed the FDA to investigate 10 specific health claims and develop language food manufacturers could use to convey any valid claims on their packaging. One of those claims was that folic acid reduced the risk of neural tube defects. The FDA was hard at work on its proposed rules when The Lancet published the MRC study (MRC Vitamin Study Research Group, 1991).

The study involved more than a thousand women in 33 centers across 17 countries. Researchers divided the subjects into four groups. Those in the first group received 400 mcg of folic acid. Those in the second group received the same amount of folic acid plus a multivitamin supplement. Those in the third group received neither the multivitamin supplement nor folic acid, and those in the final group received only the multivitamins. NTDs recurred 1% of the time when mothers received folic acid, with or without other vitamins, and 3.5% of the time when they received nothing or only the multivitamin supplement.

The MRC study did not settle the debate. It investigated women who had already had a NTD-associated pregnancy and therefore might be predisposed to having another; the results were not necessarily applicable to the general population. An FDA-contracted study released in November 1991 said it was not possible to positively conclude that folic acid prevented NTDs, but it criticized the Institute of Medicine and National Academy of Sciences reports. FDA draft rules issued later that month rejected claims that high-folate foods prevented NTDs, but it called its conclusion “tentative” and left open the possibility of further review.

The CDC, however, was less equivocal. At a 1991 conference on “Vitamins, Spina bifida, and Anencephaly,” participants generally accepted the notion that women who were pregnant or might become pregnant required more folic acid. They began to wrestle with what FDA Commissioner David Kessler would call “one of the most difficult issues” of his tenure—exactly how to go about providing that folic acid.

ALTERNATIVES

There were only a few ways to ingest more folic acid at the time: consume more foods naturally high in folates, take vitamin supplements, take folic acid pills, or eat fortified foods. Each option brought its own challenges. Working against the natural nutritionists was the fact that natural folates have lower bioavailability than folic acid and break down during cooking. A typical U.S. woman ingests only 25 mcg of naturally occurring folates daily, and thus it is hard to envision women ingesting 400 mcg through dietary changes.

At the time, folic acid by itself was only available in a 100-microgram prescription formulation. The level of folic acid in most multivitamin supplements was so low that a woman trying to hit the 400-microgram target would have to ingest toxic levels of other vitamins. One option was to make folic acid more readily available in larger doses. Another was to reformulate multivitamins. Both options suffered from the same problem: Only periconceptional intake (ingestion before pregnancy and during the first few weeks after conception) is effective; yet roughly half of all pregnancies occur without early prenatal care. You would have to convince all women to take pills to supplement their folic acid intake throughout their childbearing years or risk missing more than half the pregnancies.

That left the possibility of fortification (adding a nutrient to food that does not otherwise contain it). This approach would reach everyone, including all women who are or might soon become pregnant. Food fortification and enrichment (increasing the levels of a nutrient already in a food) has a long history in the United States, beginning in 1924 with the decision to add iodine to salt to prevent goiter and other iodine-deficiency disorders. Vitamin D was added to milk in the 1930s (much later, vitamin A was added to low-fat dairy products). Flours and bread have been enriched with various nutrients—1938 through 1942 saw the addition of thiamine, niacin, riboflavin, and iron.

Four hundred micrograms of folic acid, however, struck many as a pharmacological dose. Best-selling longevity authors Durk Pearson and Sandy Shaw said fortification was equivalent to medicating competent adults without informed consent (Junod, 2006). Scientists worried about adverse reactions. One known issue was that folic acid at daily doses of a microgram or more could mask vitamin B12 deficiency,

particularly among the older population, prompting some scientists to worry that fortification would simply shift risks from developing embryos to adults with pernicious anemia (Gaull et al., 1996).

SUPPORT BUILDS

Fortification gained adherents after the MRC study appeared and supporters’ ranks swelled as people became aware of two studies, as yet unpublished, conducted on women with no history of NTD-affected births. One, conducted in Hungary by Andrew Czeizel and his colleagues, showed benefits in the general population from consuming 800 mcg per day. Another, the “Werler study,” studied women in Boston, Philadelphia, and Toronto. Although Werler and her colleagues recommended 400 mcg daily, they found that even 250 mcg offered some

protection. In May 1992, Dr. Walter C. Willet argued in an American Journal of Public Health editorial that “fortification should be the long term goal” (Junod, 2006).

NEW PUBLIC RECOMMENDATION

In September 1992, the U.S. Public Health Service announced that women of child-bearing age should get 400 mcg of folic acid every day, a decision touted by the CDC but soft-pedaled by the FDA and the National Institutes of Health (Palca, 1992). The announcement did nothing to satisfy the two policy questions in the FDA’s lap: whether to allow food products to promote their folic acid levels on their labels and whether to require that certain foods be fortified with folic acid.

Amid safety concerns, the FDA’s folic acid advisory committee recommended against a folic acid health claim after a November 1992 meeting. In early 1993, the FDA, working under tight deadlines, adopted an NLEA rule that reflected this position and disallowed a health claim; however, the committee expressed an interest in fortification, and it reconvened in April 1993 to look at a variety of unresolved issues, including fortification. By October, the FDA had reversed its position, publishing a draft rule that would allow health claims for foods containing folic acid. There were many aspects of the rule, but its core was a provision that would allow health claims for foods that contained 40 mcg or more of folic acid per serving. On December 31, 1993, the rule became final.

The October 1993 draft rule had contained provisions about fortification, but fortification was not mandated as part of the New Year’s Eve ruling. The FDA and its folic acid advisory committee continued to struggle with a variety of implementation questions.

COST–BENEFIT ANALYSIS

Federal agencies are required to conduct regulatory impact analyses as part of rulemaking and starting in 1993 were required to assess expected costs and benefits of significant rules. FDA staff conducted a CBA and estimated that fortification with 140 mcg of folic acid per 100 grams of cereal grain products would prevent 116 NTD-affected births per year. This analysis tallied direct savings, notably medical care avoided, and estimated a savings of $5 million for each case averted, resulting in economic benefits of $651–786 million annually. The annual cost of fortification would be $27 million, and thus the annual net economic benefit would be $624–750 million (FDA, 1993).

In 1995, University of California researchers published a second CBA. They estimated that 304 NTD-associated births would be avoided through fortification. Using a different method that looked at lost productivity, they put the value of a case avoided at $342,500. The economic benefit came in at $121.5 million. From this, the researchers deducted not only the cost of fortification, which they put at $11 million, but the cost of adverse events—namely 500 cases of neurological damage annually at a cost of $16.4 million. Their calculations resulted in an estimated net benefit from fortification of $93.6 million (Romano, Waltzman, & Scheffler, 1995).

These two studies were before the advisory committee as it debated folic acid implementation, and they played a role in shaping the draft folic acid fortification rule published on March 5, 1996. A third analysis, published by the CDC that same year, was not influential. The CDC estimated 89 averted NTDs at a total benefit of $16.1 million annually. That was largely offset by the $11 million cost of fortification and an estimated $350,000 in health costs related to 89 cases of neurological damage. That left a net benefit of $4.7 million.

The FDA ultimately adopted a rule requiring fortification of cereal grain products with 140 mcg of folic acid for every 100 grams of grain. The rule went into effect on January 1, 1998. Between October 1998 and December 1999, the prevalence of reported cases of spina bifida declined 31%. Anencephaly declined 16%. Various studies over the years have put the total reduction of cases of spina bifida and anencephaly at 20–30%, much more than were reflected in the three ex ante economic analyses. (Not all of the improvement can be attributed to fortification, because women of childbearing age can expect to get only about a quarter of their recommended intake of 400 mcg through fortified grains, and public education campaigns continue to promote consumption of folic acid through vitamin pills.) Figure 12-1 shows the type of product labeling allowed with folate-fortified products.

images

Figure 12-1 Two labels (“nutrition facts” for foods and “supplement facts” for vitamin supplements) showing how regulations allow display of folate content.

Source: Courtesy of the Office on Women’s Health at the U.S. Department of Health and Human Services

THE RESULTS COME IN

Studies in Chile and Canada also reported the effectiveness of folate fortification programs. Canada mandated 150 mcg per 100 grams in 1998; Chile required 220 mcg per 100 grams in 2000. Three Canadian population-based studies showed reductions in the incidence of NTD-related births of 50%, 54%, and 43%, respectively. A single study in Chile included measures of increased folate blood levels from fortification and reported a 43% reduction in the NTD rate within 6 months. Differences among these studies included differences in fortification levels and differences in measurement and reporting regarding the inclusion or exclusion of stillbirths and terminated pregnancies.

The CDC published a before and after epidemiological study in 2004 that reported that surveillance-based population studies from 1995 and 1996 (prefortification) and 1999 and 2000 showed a reduction in the estimated number of NTD-affected pregnancies from 4,000 to 3,000. These results are summarized in Table 12-4. An editorial note in the Morbidity and Mortality Weekly Report (MMWR) noted that a 26% reduction was somewhat less than earlier studies had indicated and short of the national goal of a reduction of 50% (CDC, 2004).

In 2005, Grosse and colleagues published an ex post economic study of fortification in the United States. It estimated 520 averted cases of spina bifida and 92 cases of averted anencephaly annually, which led to economic benefits per case of $636,000 and $1,020,000, respectively. That translated into $425 million in economic benefits ($146 million in direct costs, mostly medical) against an annual cost of fortification of $3 million. The authors did not identify any documented adverse health effects from fortification.

This study used a 3% discount rate but noted that Office of Management and Budget guidelines called for comparisons using both 3% and 7% discount rates (at least one ex ante study had used a 5% rate). Of the $636,000 savings per spina bifida case avoided, the study reported, $279,000 was direct costs, mostly medical, with the rest apparently indirect costs for nonmedical caregiving. For the anencephaly cases, almost all of the $1,020,000 in costs were indirect. In addition to the required 3% and 7% discount rate comparisons, the authors performed sensitivity analyses with only 80% of the cases avoided attributed to folate fortification and with a doubling of the fortification costs. Then they developed a worst-case scenario that assumed only 80% of the observed benefits attributed to the intervention, a doubling of the cost of fortification, and a $25 million allowance for the potential effects of neurological damage from untreated anemia. This worst-case scenario still yielded annual net direct benefits of $88 million after expenditures of $6 million on fortification and an overall benefit of $312 million. The authors observed that the benefits were exceptionally large and noted that “few public health interventions beyond immunization and injury prevention are cost saving.”

Table 12-4 Estimated Average Annual Numbers of Spina Bifida and Anencephaly Cases Based on Prevalence per 10,000 Live Births from Surveillance Systems—United States 1995–1996 and 1999–2000

images

For systems with prenatal ascertainment, estimated total pregnancies included live births, stillbirths, prenatally diagnosed cases, and elective terminations. For systems without prenatal ascertainment*, estimates included live births, stillbirths, and fetal deaths through 20 weeks. Fetal deaths and elective terminations were calculated as difference between systems with and without prenatal ascertainment. The numbers of NTD-affected pregnancies and births were determined as prevalence multiplied by the average total number of U.S. births during the respective periods, as derived from the U.S National Vital Statistics System.

*Programs with prenatal ascertainment use specific case-finding techniques to identify prenatally diagnosed and electively terminated cases.

Source: Modified from: Centers for Disease Control and Prevention. (2004). Spina bifida and anencephaly before and after folic acid mandate: United States, 1995–1996 and 1999–2000. MMWR, 53, 362–365. Accessed December 11, 2013, at www.cdc.gov/mmwr/preview/mmwrhtml/mm5317a3.htm.

Hertkampf (2004) estimated that in Chile the fortification process cost approximately $280,000 annually. For spina bifida, she estimated the cost of surgery and rehabilitation for each of the 110 cases avoided annually at $100,000. She noted that bread is more of a staple of the Chilean diet. Commenting on the low priority given to folate fortification in most developing countries, she noted that prevalence data are lacking and that NTDs are not recognized as an important cause of morbidity and mortality.

A meta-analysis of eight population-based studies found that folic acid food fortification reduced the incidence of NTDs by 46% (Blencowe et al., 2010). The authors estimated that in low-income countries, fortification could reduce the number of neonatal deaths stemming from congenital abnormalities by 13%.

NEW CONCERNS ABOUT ADVERSE EFFECTS OF HIGH DOSAGES

The earlier concerns about the masking of B12 deficiencies in the elderly have not been supported with reported cases. New concerns, however, surfaced by 2007 about the impact of high doses of folates on colorectal cancer (CRC). Animal studies suggested that high doses led to two conflicting effects. Doses higher than those normally introduced by fortification protected against the onset of CRCs, but also seemed to stimulate the growth rates of existing neoplasms. These observations, reported by Mason et al. in 2007, were not followed up by randomized controlled trials, which led the British Scientific Advisory Council on Nutrition (SACN) to report in January 2008, “The evidence for an association between folic acid and increased or reduced cancer risk is equivocal.”

A 2009 report on two Norwegian clinical trials with high doses of folic acid and vitamin B12 in patients with ischemic heart disease showed significant increases in overall cancer rates (Ebbing et al., 2009). However, folic acid was considered to have a preventive effect toward heart disease and stroke (Wang et al., 2007).

Vollset and colleagues (2013) conducted a meta-analysis that examined 13 randomized trials of people taking folic acid supplements. The studies were all conducted before 2011 and combined they enrolled almost 50,000 subjects. No significant effects were found with regard to folic acid supplementation on the incidence of cancer of the large intestine, prostate, lung, breast, or any significant site. The authors noted that folic acid dosages in the study were an order of magnitude greater than intake from fortified food.

A CDC-sponsored study (Crider, Bailey, & Berry, 2011) that reviewed concerns about adverse effects found no evidence that fortification masked or exacerbated neuropathies related to anemia. It found no increase in cancer; no definitive studies showing adverse health effects from exposure to unmetabolized folic acid; and no evidence that folic acid was leading to epigenetic changes in human DNA. The researchers concluded, however, that future hypotheses addressing concerns about epigenetic changes would have to be explored. Crider and colleagues noted that careful monitoring of existing and proposed programs was needed to

allow the scientific community to determine the blood folate concentrations required for NTD prevention as well as to evaluate and respond appropriately to concerns that may arise.

FORTIFICATION IN OTHER COUNTIES

To date, more than 50 countries have adopted mandatory fortification, but many of the programs have yet to be implemented (Crider et al., 2011). A number of English-speaking countries are considering a mandate at the recommendation of food safety agencies, but for a variety of reasons only Australia has completed its implementation.

Australia and New Zealand

Mandatory fortification of bread began in Australia in September 2009. Voluntary fortification had been allowed for more than 10 years. Food Standards Australia New Zealand (FSANZ) recommended mandatory fortification of bread in 2007 following an extensive review begun in 2004. The recommendation triggered a public comment period followed by approval of the Australia and New Zealand Food Regulation Ministerial Council, which is made up of the food and health ministers of the two countries. A 12-month phase-in period was to follow. FSANZ (2009) anticipated that a mandate would avoid about 14–48 of the 300–350 NTD-affected pregnancies that occur annually in Australia. New Zealand, however, put the plan on hold after a media campaign against fortification by bakers increased public concern about the proposed fortification. In August 2012, the New Zealand government announced that fortification would remain voluntary, with the goal of half of all bread products to be fortified eventually.

Ireland’s Plans Derailed

In 1990, Ireland had one of the highest rates of NTDs in Europe, with 10–15 NTDs per 10,000 live births. Voluntary fortification and nutritional education were only marginally successful, so in 2002 the Department of Health turned to the Food Safety Authority of Ireland (FSAI). In 2003, FSAI recommended fortification, and the health minister established a National Committee on Folic Acid Food, which consulted the parties involved and in July 2006 recommended mandatory fortification. An Implementation Group on Folic Acid Fortification was formed with the expectation of final implementation. In 2008, however, the implementation group reported that folic acid intake by woman of childbearing age had increased 30% as a result of voluntary fortification, and the incidence of NTDs had been reduced to 9.3 per 10,000 live births. Although Ireland’s daily folic acid intake of 90 mcg is well below international targets and other countries are achieving a much lower rate of NTD-associated births (5–6 per 10,000 live births), the group concluded that mandatory fortification would be of limited public health benefit at that time, although the decision is subject to future reconsideration.

Meanwhile, Back in the U.K.

The U.K. Food Standards Agency (FSA) first discussed the issue of fortification in 2002 amid concerns about possible risks to the health of older people. Then the Scientific Advisory

Committee on Nutrition (SACN), an independent expert panel, reviewed the evidence and tracked the emerging science. SACN issued a November 2005 draft report (the final report was released in December 2006) that recommended mandatory fortification of flour, and in April 2006, the FSA agreed to consider four options for improving folate levels in young women:

• Do nothing.

• Increase efforts to encourage young women to change their diets and take supplements.

• Further encourage voluntary fortification of foods.

• Implement mandatory fortification of “the most appropriate food vehicle.”

The FSA consulted with consumers, stakeholders, and industry in early 2007. A SACN briefing paper issued in January 2008 recommended fortification of flour but also called for the establishment of baselines for folic acid intake and folate concentrations in the blood so future surveillance programs could identify trends. It also called for standardized testing protocols (SACN, 2008).

In 2009, SACN concluded that there was not sufficient data to support concerns about cancer, and it reconfirmed its recommendation in support of mandatory fortification. The four chief medical officers for the U.K. then recommended mandatory fortification to the four health ministers.

CONCLUSION

Mandatory fortification with folic acid is established practice in the United States and voluntary or mandatory fortification has been introduced in more than 50 countries worldwide as a strategy to help women of childbearing age increase their intake of folate. In countries where it has been implemented, it dramatically reduced NTD-associated births. So far, no European country has adopted mandatory folic acid fortification. Voluntary fortification is widely practiced in the European Union under regulation 1925/2006/EC, which allows the fortification of all foods except unprocessed foods and alcoholic beverages. Foods fortified with folic acid are widely available on the European market, except in Sweden, where fortification is not practiced, and in Denmark and Norway, which require approval. The range of product categories that are fortified on a voluntary basis includes dairy products, breakfast cereals, cereal bars, fruit juices, fat spreads, bread, and beverages. Maximum levels for the addition of folic acid to foods are not yet set in European regulations. The levels vary widely, with the highest levels added to spreads. Further use of fortification around the world, including in poorer nations, could dramatically reduce the global burden of NTDs.

Discussion Questions

1. Describe the authorizing environment for folic acid fortification in the United States.

2. Why would a decision based on strong British studies influence the U.S. decision to fortify cereal products but yield a delayed response in the U.K.? 3. Contrast and compare the variables and their values used in the before and after CBA studies. What conclusions would you draw from them about that approach in this case? In general?