Lately, the U.S. Food and Drug Administration seems to be doing a bit of re-branding: A new Patient Network site is anchoring a push for more patient engagement in the agency’s decision-making duties. There’s a good reason for that move, and it’s not just because everybody else is doing it.
“There’s this transition in thinking of the agency from a purely regulatory agency to embracing the public health mission of the agency,” said James Valentine, program manager with the FDA’s Office of Health and Constituent Affairs.
Since the 1990s, the FDA has recruited patient advocates to serve on advisory committees and as consultants to divisions that review new drugs and medical devices for approval. But Valentine said that over the past few years, patient engagement has become increasingly important.
Part of that has been driven by legislation: the Food and Drug Administration Safety and Innovation Act enacted last summer calls for expanded use of patients as consultants of review teams, and part of the Prescription Drug User Fee encourages patient-focused drug development practices.
But really, those are things that should be happening anyway. Traditional practices engage patient advocates when a drug or medical device reaches and advisory committee for evaluation. But the FDA sees an opportunity in bringing the patient voice into the conversation earlier, when they can provide input around the actual design of clinical trials. Patient input on unmet medical needs in a certain disease area, for example, could help determine what the most important clinical outcomes would be for a new drug being studied.
Then, once that scientific data is available, the agency needs patients to help interpret what a drug or medical device could, or could not do, for a certain disease community. Of course there are certain scientific requirements for a new drug or device, namely well-controlled clinical studies that demonstrate safety and efficacy, but Valentine said it has become more accepted that the benefit-risk judgment of a drug is truly that — a judgment.
“Are you at a point in a disease where you have no treatment and you just want anything, or is it something like in HIV/AIDS, where now the disease is manageable and you’re looking for a cure?” he said. “When it comes down to final decision, we want input on what risk patients are willing to tolerate to get the benefits.”
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In addition to boosting the patient representative program, the FDA also hopes its efforts will help it reach more of the less engaged patient population. Using the Patient Network site as a vehicle, it wants to do a better job of providing clinical trial information, FAQs and live interaction with FDA representatives to those who just have questions or are frustrated with their current treatments, so they can better understand the regulatory process and what their options are. (The site’s next virtual town hall meeting happens next Tuesday at 3 p.m.)
“The next steps are to keep our ears to the patient community and see where there are areas that (the site) could be further developed as we hear from patients in areas where they want more information,” Valentine said.
