Support for FDA Patient Engagement Plans
Eighty-eight patient- and disease-focused organizations say they support FDA for recognizing the need for a central Office of Patient Affairs to coordinate opportunities for patient involvement in medical product development and regulation at the agency. While the groups say the new office will be essential to success of efforts under the user fee agreements, 21st Century Cures Act, and the FDA Safety and Innovation Act, there are ways in which it can be substantially improved by harnessing patient engagement capabilities already in the Office of the Commissioner, and by ensuring that the new organization isn’t a barrier to existing relationships.
In their comment letter, the organizations say the new Office of Patient Affairs should not act as a gatekeeper, but rather should be a facilitator. “If implemented properly,” they say, “this office would level the playing field, providing an opportunity for the entire patient community to benefit from the level of involvement that some within the community already enjoy.” The implementation strategy the groups recommend involves removing the current Office of Health and Constituent Affairs from the Office of External Affairs, positioning it in the Office of Medical Products and Tobacco, and making it the new Office of Patient Affairs.
Areas in which such an Office of Patient Affairs could elevate and strategically enhance patient engagement at FDA, the groups say, include:
- confusion on the agency’s role in single-patient expanded requests;
- adverse conflict-of-interest determinations; and
- lack of transparency on use of patient representatives.
“We believe this office could greatly improve patient involvement within FDA, and we are hopeful for its creation,” the letter concludes. “However, rather than creating an additional office that could confuse patients and interfere with existing relationships, we believe that repositioning an existing centralized patient office that already administers many of these functions, and has extensive relationships with the patient community, would be the most efficient and effective method for improving patient engagement.”
In its letter, Johnson & Johnson says it is critical that creation of an Office of Patient Affairs does not inadvertently impede existing programs in the medical product centers. The company recommends saying that the new unit exists to promote, enhance, accelerate, and streamline patient-focused medical product development (rather than drug development as in early FDA writings on the subject. It also says that a key function of the new office would be to serve as the extra-agency interface between FDA, industry, and patient communities to raise the profile of issues of concern to patient communities. Areas that should be considered and clarified, it says, include:
- the office’s primary activities and how they differ from existing FDA efforts on patient engagement and drug and medical device development;
- coordination of the new unit with the medical product Centers;
- staffing;
- role of industry;
- metrics;
- no change in expanded access processes; and
- ways to leverage ongoing initiatives.
Merck says the Office of Patient Affairs “could engage in transactional activities such as connecting patients to information sources, gathering and disseminating information, and serving as the centralized place for ongoing relationship-building and engagement with a diverse patient community.” It asks for more clarity from FDA around the scope of the new office and mechanisms to allow for sharing information derived from patient engagement activities with other FDA constituencies to the extent appropriate and relevant.