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Patient Engagement Collaborative expected to invite stakeholders to discuss how patient interaction with FDA can improve.

collaborating stakeholders discuss patient interaction 


The US FDA continues to invite patients to help it better open its doors, potentially further enhancing their influence over drug reviews and clinical trial design.


Andrea Furia-Helms, health programs coordinator in the Office of Health and Constituent Affairs, said May 12 that a new group called the Patient Engagement Collaborative is in development where stakeholders can attend "regular meetings with FDA to talk about can we enhance patient engagement at FDA."


"It seems to have been an explosion over recent years of patient engagement, which has been fantastic, but we sort of want to make sure we're going in the right direction and that maybe there's some things we could be doing better," she said during an FDA workshop on patient and advocate engagement with the Center for Drug Evaluation and Research.


FDA is working with the Clinical Trials Transformation Initiative to create the group, which will allow exchange of information, ideas and experiences related to patient engagement, according to FDA's website. It is modeled after the European Medicines Agency's Patients' and Consumers' Working Party.


FDA is expected to issue a Federal Register notice soon with eligibility requirements and calling for membership nominations. Furia-Helms said that the group has been in development since the summer of 2016.


The presidential transition process as well as confirmation of a new agency commissioner slowed the group's creation, in part because the review process for Federal Register notices took longer to than usual.


FDA's volume of guidance documents, as well as Federal Register notices, has decreased since the Trump Administration arrived. The agency issued dozens of documents in the waning days of the Obama Administration, in part to ensure they were not delayed. (Also see "FDA's Document Dump: Guidance Release Skyrockets Ahead Of Trump's Arrival" - Pink Sheet, 22 Jan, 2017.)



Patient Influence Still Growing

The new group seems poised to provide another avenue for patient influence within FDA beyond the usual public settings, including into the pre-application stages, where clinical trials are planned and endpoints discussed.


Patients and advocates have held voting seats for several years on agency advisory committees that recommend approval or denial of pending applications.


In recent years, the agency also has looked to expand patients' roles in the drug review process through its patient-focused drug development initiative. It will conduct more than 20 meetings on various diseases to hear patient views on the types of treatments that are needed and the potential improvements that mean most to them.


Comments from the meetings are condensed into a Voice of the Patient report, which serves as a resource for reviewers and FDA staff. (Also see "Patient-Focused Drug Development At 10: Where Does It Go From Here?" - Pink Sheet, 20 Oct, 2014.)


The idea became so popular that the agency now is encouraging stakeholder groups to conduct their own meetings on diseases the agency cannot cover. FDA has pledged to send its staff when possible and will consider the resulting reports. (Also see "FDA’s Patient-Focused Drug Development Meetings Inspire Imitators" - Pink Sheet, 19 May, 2014.)


Several external meetings already have taken place. One covered Duchenne muscular dystrophy (DMD) and the agency used the resulting report to write guidance for drug development in the disease. (Also see "Group Submits Duchenne Muscular Dystrophy Guidance, Hoping To Direct FDA Policy" - Pink Sheet, 7 Jul, 2014.)


FDA reviewers also will consult patients for views on issues related to a specific drug under consideration. (Also see "FDA Quietly Consults Patients To Understand Vimizim's Six-Minute Walk Significance" - Pink Sheet, 1 Dec, 2014.)


In addition, the agency and industry agreed as part of the upcoming prescription drug user fee reauthorization to look for ways to make patient-reported data collection more systematic. (Also see "PDUFA VI: Price Tag For Patient Experience Data Validation May Go Up" - Pink Sheet, 18 Jun, 2015.)


Patient involvement at FDA reached a new milestone in 2016 when a patient group gave its thoughts during a portion of Sarepta Therapeutics Inc.'s formal presentation time during an advisory committee meeting on its DMD treatment Exondys 51 (eteplirsen). (Also see "Duchenne Group's Presentation Is Milestone For Patient Involvement" - Pink Sheet, 2 May, 2016.)


But some at FDA may be leery about patients gaining more access. Staffers said patient pressure during the Exondys 51 review at times was problematic. (Also see "Patient Advocacy With FDA Review Staff Will Be Tougher Post-Sarepta" - Pink Sheet, 23 Sep, 2016.)


Indeed, the new group may be an attempt to find better ways of controlling patient access within the agency without overly limiting it.


Some stakeholders still complain that reviewers should be made more available to the public. (Also see "PDUFA VI: NORD Wants Reviewers To Be More Accessible" - Pink Sheet, 18 Aug, 2016.)



No Update On Patient Affairs Office

Internally, FDA also is growing its patient involvement efforts.


Furia-Helms said an internal cross-agency working group on patient engagement has evolved into a Patient Council, so staff can "better inform each other" as well as "leverage off each other when we have patient-engagement activities and really to know what's up and coming."


The agency also in March floated an idea to centralize all patient engagement activities in a new Office of Patient Affairs. It would be intended to provide a more visible and accessible entry point for patients and advocates and help ensure inquiries are directed properly. (Also see "US FDA Patient Affairs Office Could Accelerate Involvement With 'Central Entry Point'" - Pink Sheet, 14 Mar, 2017.)

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