By NANCI BOMPEY
The National Health Council has been meeting with FDA on proposals for implementing FDA Safety and Innovation Act provisions intended to provide a structured framework for including input from patients
and patient organizations throughout the agency’s decision-making process. The group, which represents close to 50 different patient organizations, is pushing FDA to distinguish between input from consumers, patients and organizations that represent them, and where each group should be factored in during the drug development process.
Myrl Weinberg, president of the National Health Council, said the group has been meeting with FDA to implement user fee provisions increasing the agency’s use of patient perspectives in its decision-making.
The group secured measures in the FDA-industry user fee agreement mandating FDA develop a plan to further develop and implement a structured benefit-risk assessment in the new drug approval process, and hold a series of public meetings on specific disease areas that could benefit from a more systematic and expansive approach to obtaining the patient perspective.
Further, the group also championed user fee measures to expand the use of patient-reported outcomes and surrogate endpoints, and enhance the agency’s rare disease program. The group said it worked to get the provisions into the industry-agency negotiated user fee agreement — as opposed to the accompanying legislation — to ensure fee money would go toward these initiatives.
Weinberg said the group has support for its plan — which it has outlined in public meetings, letters to FDA and other communications — from its membership and other patient groups, adding FDA has also been very receptive to its ideas.
Factoring patient benefit and risk into FDA decision-making was highlighted by a recent agency decision allowing limited use of the Stryker Wingspan brain stent system. FDA noted it took into account patient benefit in its recent decision, but Weinberg said it is unclear if the agency actually engaged with patients or organizations in a meaningful way. Further, she said issues with the product could possibly have been resolved prior to approval if patients had been included in the entire development and approval process.
Weinberg said it is important to “not just ask them, but ask them up front, from the beginning, what is important as they evaluate these stents.”
Public Citizen also slammed FDA for the recent decision, saying it is not clear the benefits of using the device outweigh the risks even for the limited population the agency identified. “This is further evidence that the agency is not making decisions in an evidence-based manner but, rather, stating that it believes the device will be beneficial, despite evidence to the contrary,” Sidney Wolfe, director of Public Citizen’s health research group, said in a statement. Wolfe has also cautioned against the agency moving away from a science-based approach, saying desperation shouldn’t outweigh the issue of public health (see related story).
The NHC is pushing FDA to make a distinction between patients and consumers, as well as the organizations that represent them, as it moves toward integrating public input on benefit-risk, saying perspectives on benefit-risk differ significantly between these groups. Weinberg said FDA needs to recognize and distinguish the different perspectives it may receive from consumers, who may be healthy; patients, who suffer from a disease; and individual patients and patient organizations with differing perspectives of a disease.
The NHC also believes FDA needs to determine where in the regulatory review process input from these groups would be sought, adding that the agency should engage with groups throughout the regulatory review process rather than at one point in time.
“This would enable FDA to better accommodate the dynamic nature of benefit-risk assessments, and to integrate new findings from clinical investigations, the evolution of the treatment landscape, or changes in patient preferences and risk tolerances,” the group wrote in a letter to FDA.
Weinberg added the NHC believes the patient perspective should be considered throughout the drug life cycle, including development, approval and post-market surveillance.
The group said this would include providing input on risk-benefit assessments during drug development, serving on advisory committees and providing insight on patient-focused factors for consideration in assessments of benefit-risk during drug approval, and offering assessments of new information in weighing benefit-risk during post-market activities. Further, the group also proposed a framework for ensuring patient engagement throughout the research and development process when setting the research agenda, developing research questions, selecting outcomes and comparators, recruiting subjects, and translating and disseminating findings.
The group said FDA should also consider different modalities for soliciting input and how these can be enhanced to ensure patient participation or if they inadvertently hinder participation by certain populations, including managing conflict of interest — a hotly debated topic during user fee negotiations. The group said “potential or perceived conflicts of interest should not be a de facto standard for disqualification from participation.”
Further, the NHC is also proposing a framework for FDA to use in grouping and prioritizing disease areas it will focus on in 20 public meetings with the patient community on benefit-risk considerations. The proposed framework would consolidate and adapt two existing approaches: the National Institutes of Health model used in its National Library of Medicine framework and the “Bridges to Health” model.
The NHC proposes to modify the NIH/NLM list of specific conditions to come to eight broad categories, to which FDA can add certain diseases or conditions that may affect multiple body systems. The “Bridges to Health” model would help stratify the population affected by these conditions into eight groups.
Weinberg said once more specifics are in place she would expect the agency to issue guidance or another form of communication that will “make it abundantly clear” when and how patients, consumers and organizations would be involved in the FDA process and what types of training that might require.