Good morning. My name is Linda Golodner, President of the National Consumers League (NCL). It is a pleasure to be invited to speak at the 43rd annual Ohio Pharmaceutical Seminar. This is not the first time I have had the privilege of speaking to this group. In the past I gave a presentation about pharmaceutical care and its impact on consumers. Today, I am here to talk to you about another important issue that has a profound impact for all of us: FDA Reform -- the Food Drug Administration Modernization Act of 1997. Before I do this, however, let me give you some background on the National Consumers League, and our work to promote consumer safety and protection with the FDA and its predecessors.
The National Consumers League is a national, nonprofit membership organization with nearly 100 years of experience representing consumers in the workplace. In fact, next year we will celebrate our centennial anniversary. NCL is concerned with a range of consumer issues, from consumer fraud, fair labor standards, electricity deregulation, and child labor issues, to health care coverage, food safety, and of course, drugs safety. No matter what issue we work on, however, consumer rights are of paramount importance -- the right to information, the right to education, right to safety, right to choice, and the right to quality service. These consumer rights must be respected by those who deal with the public -- by the government, pharmaceutical manufacturers, and health care professionals. And with those rights come responsibilities, especially the responsibility to be more aware of one's own health, to question one's doctor and pharmacist about the drugs we take.
Since 1899, NCL has worked to ensure that the drugs available in the U.S. are safe and effective. The League's first national consumer campaign in the beginning of the century was inspired by a member of our Board of Directors, Dr. Harvey W. Wiley, Chief of the U.S. Department of Agriculture's Bureau of Chemistry (the original FDA), who sought legislation outlawing adulterated foods and ineffective or dangerous patent medicines.
Through exhibits at state and national fairs, articles in magazines and newspapers, the league presented Dr. Wiley's facts on poisons in drugs and foods. The result was the 1906 Food and Drug Act prohibiting adulteration and misbranding of patent medicines and foods. That campaign was just the beginning.
League members continued to monitor drug regulations and took the lead on strengthening the law. In 1938, after NCL fought many battles to keep amendments intent on weakening the laws from passing, the Federal Food, Drug, and Cosmetic Act was passed. The league continued to fight for safer drugs for consumers, and was instrumental in several other significant pieces of legislation.
The League worked on amendments to the Food, Drug, and Cosmetic Act, including the 1951 Durham-Humphrey Amendment, establishing the present system of two categories of medications: prescription and nonprescription. In 1962, NCL helped to pass amendments requiring the FDA to base approval of drugs on efficacy standards, and to deny or withdraw drugs that were ineffective.
In the late 1960's, NCL served on the advisory panels reviewing drugs. In 1972, when Over-The-Counter (OTC) Review Panels were formed to evaluate safety and efficacy of OTCs, the League was once again asked to serve as a consumer representative. Today, the League is a member of the FDA Consumer Consortium, a group of consumer and public health organizations that selects consumer representatives to sit on the FDA advisory committees.
As you can see, we have an important legacy to continue as we campaign for the safety and efficacy of drugs. Using research, education, and advocacy, NCL promotes the safe and effective development and uses of drugs. NCL produces brochures -- in English and Spanish--conducts surveys, submits comments on proposed rules and petitions concerning products, and sends inquiries to the FDA on a number of concerns about safe medication use. In addition, we closely follow any legislative proposals or proposed regulatory changes that would affect drug safety and consumer protections. Thus, the recent changes brought about by the FDA Modernization Act are of great interest to the League.
FDA is one of our nation's oldest consumer protection agencies. First and foremost, FDA is a public health agency, charged with protecting American consumers by enforcing the Federal Food, Drug, and Cosmetic Act and several public health related laws. It is FDA's job to see that the food we eat is safe and wholesome, the cosmetics we use won't hurt us, and the medicines and medical devices we use are safe and effective. With the recent ideological shift in Congress, there has been a growing anti-government sentiment. The passage of the recent FDA Modernization Act reflects this. It seems that recent Congressional actions are pushing the FDA in the wrong direction. Pro-industry and anti-regulatory political interests are now driving the decisions by congress, rather than the scientific and public health interests championed by the FDA.
FDA has more responsibility and an increased work load, especially in the areas of drug approval and food safety and inspection, while the budgets for these responsibilities are being scaled back dramatically. As Deputy Commissioner William Schultz says, "The fear of a lot of us at the agency is that the weight of the provisions and the workload that is demanded could have a very negative effect on our ability to do our work."1 Unless we are to believe that lawmakers and special interests are naive to the consequences of these actions, we must realize that there is a political agenda that seeks to diminish the agency's regulatory capacities.
The biggest criticism against the FDA--waged by patients' rights groups--mostly AIDS and cancer groups--as well as the pharmaceutical and device manufacturing industries--that it was too slow in reviewing new drug applications, has been addressed and corrected. The Prescription Drug User Fees Act (PDUFA) has allowed this process to be greatly streamlined and sped up, yet Congress forged ahead with other drastic changes that were clearly intended to weaken FDA and scale back its regulatory authority. Here are some of the major changes that will affect consumers:
Off-Label Promotion: the law abolishes the long-standing prohibition on dissemination by manufactures of information about unapproved use of drugs and medical devices. This will allow companies to promote drugs for purposes that have not been proven safe and effective by the FDA. This rolls back the law to pre Kefauver-Harris amendment (1962) when only safety was the standard. This could be detrimental for consumers since it will allow drugs to be used in ways they were not intended, and more importantly, where there has not been sufficient scientific and clinical testing.
On the other hand, however, the use of off-label promotion has allowed many drugs to be used for treating diseases and conditions where there is no alternative drug. Many cancer drugs were previously, and are currently, used off label. Furthermore, nearly all children's medications are off label, since there are very few clinical trials designed for children. While consumers accept the use of off label drugs, there must be caution, with a commitment for ongoing clinical testing, and an aggressive post-market surveillance.
The best example of Off-label use gone wrong, with serious health consequences for consumers is Fen-Phen. Fen-Phen was promoted off-label as a miracle diet drug combination to help people lose weight. It was prescribed by thousands of doctors to tens of thousands of people for several years. Recently, as you know, serious side effects have been found, most notably, incidences of defective heart valves. As a result, the FDA asked the manufacturers of fenfluramine--the "fen" in Fen-Phen, also known as Pondamin--and dexfenfluramine (Redux), to voluntarily withdraw these products from the market. The companies have agreed. The lesson learned here is that off-label promotion of drugs may have serious consequences since these uses are not being sufficiently tested under rigorous clinical and scientific trials.
The Act allows a firm to disseminate peer-reviewed journal articles about off-label indications, provided the company commits itself to file, within a specified time frame (three years), a supplemental application based on appropriate research to establish the safety and effectiveness of the unapproved use. The law states that if a company finds that a drug's off-label use is dangerous, it must stop marketing it for that off-label purpose, but physicians will still be allowed to prescribe it. As Arnold Relman, MD, editor emeritus of the New England Journal of Medicine, has pointed out, "publication of an article on the unapproved use of a drug in a peer-reviewed journal is no guarantee of safety or efficacy."2 We seriously question the practice of allowing companies to promote off-label use for three years without publishing serious clinical trials. After three years of marketing, it will be extremely difficult to get doctors to stop prescribing the drugs.
Use of Economic information (Pharmacoeconomics): the Act allows drug companies to provide economic information about their products to formulary committees, managed care organizations, and similar large-scale buyers of health-care products. The provision is intended to provide such entities with dependable facts about the economic consequences of their procurement decisions. This provision, like off-label promotion is a double-edged sword. Pharmacoeconomics is an important tool for all those involved in the procurement and distribution of pharmacy benefits, including managed care organizations, unions, and private businesses. The use of cost-benefit analysis is the foundation of the development of formulary lists, and is often necessary to ensure that coverage of some sort exists at all.
However, NCL is concerned that decisions could be made based on the costs of the drugs, rather than the effectiveness. Many drugs that are cheaper in the short-run, may actually turn out to cost more in terms of unintended complications, in the long run. For example, Premarin, which is the most widely prescribed drug in the world, and is used to alleviate post-menopausal symptoms, as well as helping to prevent osteoporosis, may cost 40 cents a day. A generic, which is similar to Premarin, but not identical--it contains different estrogens--may cost 30 cents a day, but has no effect in preventing osteoporosis. Thus, the short-term costs may be greatly outweighed if the patient develops osteoporosis and breaks a hip, which is an expensive condition to treat. If the economic data is stressed over and above the safety and efficacy data, then consumers will be hurt. Their drug choices may be limited--effective but expensive drugs may not be available, and less effective, but cheaper drugs may be substituted. This is an unacceptable situation.
Pharmacy Compounding: The Act creates a special exemption to ensure continued availability of compounded drug products prepared by pharmacists to provide patients with individualized therapies not available commercially. It also seeks to prevent large-scale manufacturing under the guise of compounding by establishing parameters. As William Schultz, FDA's Deputy Commissioner for Policy, states, "It is generally recognized that pharmacists can compound drugs, and in a sense, that's manufacturing, if on a small scale. But until now, there have been no attempts in law or regulating to draw that line. This bill recognizes that pharmacists can compound drugs for individual patients but not on such a broad scale that it could be construed as manufacturing."3 It allows pharmacists to use ingredients (either imported or domestic) that are not approved by the FDA or recognized with a monograph from USP, if these ingredients are on the list that is being developed by the newly created Pharmaceutical Compounding Advisory Board.
Fast Track: Expedited Drug Review: The Act codifies FDA's regulations and practice to increase patient access to experimental drugs and medical devices and to accelerate review of important new medications. What the law says is that if surrogate endpoints are likely to show a benefit, then the drug can be on a fast track for approval. Previously, drugs or devices had to show that they decreased morbidity and mortality (cured the disease and/or prolonged life). Now, it only has to have significant impact on the disease-state, and does not have to increase life expectancy. For example, new cancer drugs only have to show tumor shrinkage, not that the drug increases life expectancy.
while it is true that this will speed more life-savings drugs to consumers, it also can cause dangerous or unnecessary drugs to be allowed to reach consumers:
Many patient's rights groups support this provision because they are desperate--they are sick or dying and they want a cure. When the HIV/AIDS epidemic first reached significance, in the mid-to-late 1980's, many AIDS patients and advocates were calling for drugs to help cure them or extend their lives, and the government and pharmaceutical companies began to search for new drugs to do this. A host of products were rushed through the approval process and marketed to HIV/AIDS patients. Some of these drugs have since been found to be effective, while others were found to be quite dangerous. This is a difficult situation. We must balance the need and desire for new drugs to come to market to help save people's lives, with proper testing and evaluation of these drugs to ensure that they do not do more harm than good.
Medical Devices: the expedited review process eliminates the mandatory tracking and surveillance of high-risk medical devices by manufacturers. Now it is a voluntary system. And while it is in the best interest of manufacturers to track their devices in patients, not all will do so. One perfect example is the Shiley Heart Valves. These valves were proven dangerous (malfunction) and were recalled by FDA. The only problem was that the company wasn't tracking the device and was unable to locate all of the patients who had the device implanted. There are currently people walking around with this faulty device inside of them.
Prescription Drug User Fees Act (PDUFA): The act reauthorizes the user fees for 5 more years. These fees have allowed FDA to greatly speed up its evaluation and approval of new drugs, using fees paid the agency by industry. It has allowed FDA to reduce time of drug review from 30 months to 15 months. FDA has added 696 employees to the biologics and drugs programs while taking in $329 million in user fees from the pharmaceutical industry. While this has been beneficial to all parties involved, there are also some problems with not only the program, but the process. First of all, none of the PDUFA money can be used for post-market safety studies or monitoring. The increase in the user fees has resulted in a large increase in the number of approved drugs, but there have been no post-market studies to ensure safety and efficacy funded by these fees. Since PDUFA is popular and supported by groups on both sides, it was used to the advantage of those in Congress and other special interests who are anti-FDA and anti-regulation. As Deputy Commissioner Schultz notes, "in 1994, both houses of Congress became controlled by Republicans, who were much more willing to bring FDA reform legislation to a vote than were the Democrats."8 This political agenda was designed to erode consumer protection and erode FDA's regulatory authority, while increasing its workload and significantly decreasing its funding (funding was cut 40%).
The FDA reform debates were not very open to the public. As former Senator Howard Metzenbaum (D-OH) stated in an interview in JAMA, "[the Labor and Human Resources committee] did not hold a public hearing before reporting the bill favorably to the Senate. That was wrong. This bill is too important to slide through without a public hearing."9
Standards for Medical Products: Clinical Trials: The law codifies the agency's current practice of allowing, in certain circumstances, one clinical investigation as the basis for product approval. The act, however, does preserve the presumption that, as a general rule, two adequate and well-controlled studies are needed to prove the product's safety and effectiveness. As Larry Sasich, Pharm.D., of Public Citizen's Health Research Group states, "two studies are better than one because replication of results is vital to ensuring, with a high degree of statistical confidence, that the product is safe and effective."10 We are concerned that codifying into law the practice of allowing only one clinical trial will seriously erode the standards so necessary to protect consumers from dangerous or ineffective drugs.
Notice of Discontinuance: Patients will receive advance notice when a manufacturer plans to discontinue a drug on which they depend for life support or sustenance, or for a treatment of a serious or debilitating disease or condition. Companies are required to notify the patients and physicians six months prior to discontinuation. While this provision does nothing to address the problem of orphan drugs--the lack of incentives for manufacturers to produce drugs for very small segments of the population--it does allow patients advance warning so they can seek alternative treatments. Unfortunately, there is often only one drug, leaving patients with no treatment available if the manufacturer discontinues the drug. What are these patients to do? Do we send them to Canada or Mexico to get their drugs, if they are even available in those countries? Patients shouldn't have to be without drugs they need simply because a manufacturer, for one reason or another, is no longer interested in keeping a drug on the market.
Clinical Trials Database: The law provides for an expanded database on clinical trials which will be accessible by patients. With the sponsor's consent, the results of such clinical trials will be included in the database. This will help patients with serious or life-threatening diseases to gain more information about clinical trial results. As Deputy Commissioner Schultz states, "this is the kind of information that physicians and patients need when making decisions about whether to try experimental drugs."11 Many patient support and advocacy groups are in favor of this legislation.
As you can see, the FDA Modernization ACT of 1997 has serious implications and consequences for consumer protection and safety. While some of the new provisions I have mentioned are clearly intended to help consumers, there are others that may cause grave effects. We cannot allow Congress, the industry, or any other groups to erode the consumer protections that we have fought so hard to obtain. FDA is concerned about safeguarding the public's health and safety for food, drugs, and devices, yet the recent changes are restricting its ability to be an effective advocate for consumer protection.
It is not right to greatly increase a regulatory agency's responsibilities and not provide adequate funding. This, leads to overworked employees, a reduced ability to enforce regulations, and reduced ability to provide oversight and protection measures. This is what congress has done to the FDA. This reduced oversight authority hurts the consumers. The National Consumers League will continue to fight to preserve consumer protections and safety and efficacy standards for FDA. We will submit comments to FDA on rule-making and regulation proposals. We will work with Congress to ensure that sufficient funding and consumer protections are maintained. We invite you to join us in this important battle. Thank you.
2 Ibid.
11 JAMA, March 18, 1998--Vol. 279, No. 11. P. 816.
For more information, to schedule an interview with NCL staff, or to contact NCL's
Communications Department on this or any other release, call Director of
Communications Holly Anderson at (202) 835-3323.