Medscape: According to the Cures Act, clinical experience could come from observational studies, clinical registries, ongoing safety surveillance, and patient-centered outcomes research activities. Do you think using this additional clinical experience data to support the approval of a new drug indication or to satisfy post approval studies is a good idea?
Dr Vose: Unfortunately, the postmarketing information that is captured is very minimal and patient reporting is very sketchy. So, I think trying to capture that information in the real world is very important. ASCO is conducting the CancerLinQ project, through which we are trying to capture data in the real world, for patients not on clinical trials. I think this will probably be good for hypothesis-generation [in terms] of looking for new indications for current drugs or for potentially new options for patients.
I think it is important to look at the data and put them in the context of the real world. Clinical trials, unfortunately, are very stringent, so we need to get additional information to see what really happens in the real world. Collecting all of the data is probably a way to improve safety.
[The Act] will help us alter the standard of evidence to help ensure that the FDA can maintain its focus on efficacy and safety of clinical trials, but it will give us additional information. As long as we collect information that is accurate and correct, I think it is a good use of resources.
Dr D’Amato: Overall, the idea is to make drug development and approval more efficient. It is important to constantly evaluate whether our regulatory regime is nimble enough to respond to technological advances. The proposals take steps to move beyond a rigid two placebo-controlled studies for approval to something that is more dynamic. New endpoints allow for a better focus on outcomes that are meaningful for patients, not just what is meaningful to the FDA.
Dr Zuckerman: I believe the Act will allow unsafe and ineffective drugs to market. Clinical experience should be used to supplement and help understand scientific data from clinical trials, not to replace such data. Clinical experience should be used to support or reject applications. Unfortunately, I’ve seen many examples at FDA meetings where companies use patient and doctor experiences to support their application for approval, and dismiss clinical experiences as meaningless anecdotes when they show terrible side effects.
The definition of “clinical experience” includes everything and the kitchen sink in the bill, and our suggestion that the definition be improved was ignored. I’m an epidemiologist by training. I am a strong supporter of analyzing big data from medical records, but those analyses need to be conducted in a careful and unbiased manner. That’s very different from clinical experience defined as one doctor’s experience with a few patients, for example, or one patient’s experience with a drug.
Any scientist worth anything can tell you that the experience of a few doctors or patients does not predict the safety or effectiveness of a drug or device. And anyone who understands the FDA approval process—which unfortunately, many doctors and Hill staffers do not—realizes that even if a drug is already approved, it doesn’t mean it’s safe or effective for other uses. FDA approval is supposed to be based on whether the benefits outweigh the risks for a particular indication. To know whether that same drug has benefits that outweigh the risks for any other indication, or for the same use by other types of patients, requires scientific analysis.
On the other hand, a patient’s experience can help us understand the benefits and risks of a drug. Patients and family members can help us understand the devastating impact of side effects, such as gastrointestinal perforations, for example, or how the benefits of a cancer drug enabled them to live at home instead of a hospital during their final weeks of life.
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