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Pink Sheet – US FDA Patient Affairs Office Could Accelerate Involvement With 'Central Entry Point'

Pink Sheet – US FDA Patient Affairs Office Could Accelerate Involvement With 'Central Entry Point'


The US FDA is considering elevating patient engagement to a more visible position by establishing a central office for the programs.


The agency said in a Federal Register notice expected to be published March 14 that it is seeking comments on its idea to create an Office of Patient Affairs. It is intended to provide a “more transparent, accessible and robust experience for patient communities,” the agency said in the notice, as well as handle patient engagement activities across FDA’s medical product centers and other offices.


FDA wants a “single, central entry point” for patients that also can help move their inquiries to the proper place. It also would host and maintain data management systems that would “incorporate and formalize knowledge shared with FDA by patient stakeholders” and create a “scalable and forward-looking platform for communicating with patient stakeholders, particularly online channels,” according to the notice.


Stakeholder comments on the idea will be due in mid-June.


Among the problems at FDA now is that with the increasing number of patient initiatives across the various product centers and other offices, there is often little communication between them, even though they have similar tasks, Paul Melmeyer, associate director of public policy at the National Organization for Rare Disorders, said in an interview. He said NORD hopes coordination would improve with a central office for patient issues.


Indeed, agency officials have been working to increase its outreach to patients over the last few years, in part through mandates in the 2012 FDA Safety and Innovation Act (FDASIA). FDA established its patient-focused drug development initiative in the wake of FDASIA’s enactment, which included scheduling more than 20 public meetings to solicit patient input on various diseases. They were intended to help FDA reviewers and other staff understand unmet needs and preferences to help inform benefit-risk decisions about new drug applications. (Also see “Patient-Focused Drug Development At 10: Where Does It Go From Here?” – Pink Sheet, 20 Oct, 2014.)


The meetings have largely been successful and have inspired patient groups to conduct their own meetings. (Also see “FDA’s Patient-Focused Drug Development Meetings Inspire Imitators” – Pink Sheet, 19 May, 2014.) One on Duchenne muscular dystrophy helped inform FDA guidance for drug development in the space. (Also see “Group Submits Duchenne Muscular Dystrophy Guidance, Hoping To Direct FDA Policy” – Pink Sheet, 7 Jul, 2014.)


And patients have seen their power to influence FDA grow in recent years as well. The most high-profile example is in the DMD space. Patients and advocates pushed hard for approval of Sarepta Therapeutics Inc.’s DMD treatment Exondys 51 (eteplirsen). They not only flooded the advisory committee meeting on the product, but a patient group also outlined its own data during Sarepta’s formal presentation, which was thought to be a first. (Also see “Duchenne Group’s Presentation Is Milestone For Patient Involvement” – Pink Sheet, 2 May, 2016.)


And despite FDA questions about Exondys 51’s efficacy, it was thought patient pressure factored into the decision by Center for Drug Evaluation and Research Director Janet Woodcock’s decision to overrule her subordinates and approve the product. (Also see “Sarepta’s Eteplirsen Approved After Contentious Internal Debate” – Pink Sheet, 19 Sep, 2016.)


However, there still have been complaints that some FDA staff have not been as accessible to patients as desired. (Also see “PDUFA VI: NORD Wants Reviewers To Be More Accessible” – Pink Sheet, 18 Aug, 2016.)


Symbolic And Practical Promotion

Creating an office dedicated to patient affairs would elevate patient engagement symbolically, as well as practically within FDA.


FDA now has a patient liaison team within its Office of Health and Constituent Affairs within the Office of the Commissioner that handles a number of patient inquires and includes liaisons for various medical products. There are other patient initiatives within the Center for Devices and Radiological Health and other centers.


Nancy Goodman, executive director of Kids v Cancer, told the Pink Sheet that she supported the idea of a patient affairs office and “any process the FDA establishes to increase and improve communication with patients and advocates can only improve public health.”


Jeff Allen, Friends of Cancer Research president and CEO, also said the office would be a valuable resource for the community.


Creating a new office would make the program much more visible to the public, even if it does not result in major changes to current practice. The office also would better illustrate that patients, FDA’s primary customer, and their input, are important to the agency.


FDA’s notice did not discuss where the new office would be located within the agency, its staffing needs, or a potential timetable for its creation, should it decide it is necessary.


The agency made a move analogous to the creation of the patient office a few years ago when it elevated the Office of Generic Drugs to “super office” status within CDER. Up until 2013, it had been part of the Office of Pharmaceutical Science, and its leader did not report directly to Woodcock. OGD was promoted in part because of its importance to public health: it approves drugs that make up the vast majority of prescriptions in the US. (Also see “FDA Generics Office Drinks Super Serum, Then Shrinks” – Pink Sheet, 16 Dec, 2013.)


A Venue For Structuring Evidence?
The patient affairs office might also coordinate FDA efforts to research and integrate patient data into its review process. As the agency has received more input on drug safety and efficacy from patients, it has been trying to determine how it can formally affect its approval decisions.


The next evolution of patient-focused drug development is expected to be determining how to standardize the data generated so it can be relied upon by reviewers. FDA has said it needs to be de-risked. (Also see “PDUFA VI: Price Tag For Patient Experience Data Validation May Go Up” – Pink Sheet, 18 Jun, 2015.) Industry groups have called for FDA to establish a structured process for patient-reported data. (Also see “Patient-Focused Drug Development High On Industry’s List Of PDUFA VI Tweaks” – Pink Sheet, 29 Sep, 2014.)…