NCHR Testimony to the FDA on Transparency

I am pleased to have the opportunity to testify as president of the National Research Center for Women & Families. Our Center is dedicated to improving the health and safety of adults and children, and we do that by scrutinizing medical and scientific research to determine what is known and not known about specific treatments and programs, and to compare their safety and effectiveness.

In addition to my work at the National Research Center for Women & Families, I am a fellow at the University of Pennsylvania Center for Bioethics.

My doctorate is in clinical psychology and my post-doctoral training at Yale was in epidemiology and public health. Prior to my current position, I was on the faculty at Yale and Vassar, directed a multi-site longitudinal research project at Harvard, and worked in the U.S. Congress, the White House, and the Public Health Service. I have worked on FDA issues for 19 years. I am speaking from a public health perspective as a researcher and policy expert.

I was very glad to hear the FDA Commissioner and Deputy Commissioner talk about the need to refocus the FDA on its public health mission. This initiative on transparency is a great place to start.

I have scrutinized FDA policies, processes, and decisions as a Congressional investigator and in my current position. There is no doubt that I know more about the FDA than at least 99% of Americans and probably more than at least 95% of health policy experts. However, as an outsider who does not work at the FDA, I am still amazingly limited in terms of the information that I have access to. That’s why this conversation about transparency is so important.

I know how to go to the FDA web site to find industry data and FDA analyses for medical products that have been reviewed at public FDA Advisory Committee meetings since 1998, when that information was put online. I know how to use the FDA web site to find other information about those same medical products, such as approval decisions and recalls.

However, if I want to see data about other medical products – the vast majority of medical products that were not subject to public FDA Advisory Committee meetings – I am not able to get that information. The information available is the tip of the iceberg in terms of FDA’s data and decisions about prescription drugs and vaccines, and not even the tip of the iceberg for the thousands of medical devices cleared for market by the FDA every year.

In recent years, I have written testimony and articles about the safety and effectiveness of certain medical products reviewed at FDA meetings, based on FDA statistical analysis. In some cases, such as Neurostar, a medical device for the treatment of depression, the data presented at the public meeting clearly indicated that the product was not effective – in that case, Neurostar was shown to be equally effective whether it was turned on or off. In other words, it was no more effective than sham treatment. As one of the Advisory Committee members so eloquently summarized the situation: you can’t determine the risk-to-benefit ratio when the benefit is 0. And yet, about a year later the FDA quietly announced that they had cleared Neurostar for market, and that the data indicated it was effective. What happened to magically transform data indicating no effectiveness to data indicating it was effective? I don’t know. Those data are not available. However, published data suggest that post-hoc manipulation of the outcome measures produced the results the company needed for approval.

Similarly, data presented at FDA meetings on silicone gel breast implants indicated that women who got breast implants had lower self-esteem and lower quality of life two years after getting breast implants than they had before surgery. Those data were made available on the FDA web site. Since the manufacturers and the plastic surgeons claimed that increased self-esteem was a major benefit of breast implants, these scientific results should have been devastating for the application. However, despite the lowered self-esteem and quality of life, silicone breast implants were approved by the FDA, apparently over the objections of FDA scientists. As a condition of approval, FDA required that doctors provide free booklets to their patients prior to breast implant surgery, showing the risks and benefits as analyzed by the FDA, but the data in the patient booklets are substantially different from the data presented at the FDA meetings. Complication rates are lower, and data indicating lower self-esteem and quality of life have magically disappeared. How did those data change to make the products look more effective? We don’t know and there is no way to find out.

These are just two examples, but there are many more. For example, when medical devices are approved under the condition of continued longitudinal research, those future longitudinal data are almost never made public. They are not on the FDA web site and they are usually not published either. Are they not published because the product is found to be less safe or less effective than expected? Or are so many patients lost to follow-up that the studies are not publishable? We don’t know because we can’t see the data. In some cases, I know that key Members of Congress have requested the data from FDA regarding required long-term studies and have not received them.

These are examples of lack of transparency regarding medical devices, but the same is true for drugs and biologics. The data are rarely available, the data made available at Advisory Committee meetings sometimes mysteriously change behind closed doors, and the public has no way to know if those changes are a result of new data or of non-scientific decisions resulting from negotiation between a company and the FDA, or some other reason. We don’t have access to the data or to a clear explanation of how FDA made a decision – even when the decision seems suspicious.

The American public deserves better. Most people will not go on the FDA web site looking for data, but any scientist, journalist, or other interested person who wants to see the data on products that the FDA has approved or not, should be able to access that information, whether the studies were the basis of approval (or non-approval) or whether those studies were required in the post-market period.