US FDA/NORD Partnership Will Further Increase Patient Involvement In Drug Development

Agency's agreement with rare disease advocacy group will facilitate patient meetings with review divisions, as well as enhance communication of safety information.

The US FDA is partnering with a prominent rare disease advocate to increase the patient voice in drug approval and development in what appears to be another evolution in the agency's patient-focused drug development efforts.

A new memorandum of understanding (MOU) with the National Organization for Rare Disorders will allow collaboration to plan workshops, training and listening sessions, as well as access to "patient clinical and medical product consumption experiences," according to the agreement, which FDA Commissioner Scott Gottlieb announced Feb. 26. The MOU went into effect Feb. 21 and will last three years unless otherwise terminated by either party.

Gottlieb said in a blog post that as part of the MOU, the agency and NORD have planned a pilot project with several listening sessions on rare diseases. FDA told the Pink Sheet that the goal is to have patients tell reviewers what is important for managing symptoms and improving quality of life. Topics would be contingent on the information needs of the review divisions, such as "disease burden, symptom management, impact on daily activities, and quality of life."

NORD is well-known to FDA because of its long-time advocacy for rare disease issues and its 260 member organizations will help the agency find patients that are needed for the listening sessions "because sometimes it can be challenging to identify rare disease patients and caregivers," the agency said. Indeed, the MOU says NORD has served as a "hub of the rare disease community" and led efforts to connect patients and groups with other stakeholders.

NORD told the Pink Sheet that FDA's Patient Affairs Staff will work with the organization to schedule the sessions to allow patients to share their thoughts with review division staff. FDA will create questions for patients and NORD will facilitate the discussion.

FDA created the Patient Affairs Staff to provide a central contact for patients to interact with the agency. (Also see "US FDA's Patient Affairs Office Is An Office … In Spirit" - Pink Sheet, 17 Nov, 2017.)

Gottlieb added that the MOU is intended to "enhance the incorporation of patient experience into regulatory discussions."

"We recognize that early and iterative engagement can improve clinical and regulatory understanding of diseases and conditions, provide a common understanding of the most urgent patient needs, and inform drug development programs," Gottlieb wrote.

FDA has been working to increase patient involvement in agency decisions for several years as part of its patient-focused drug development initiative. The agency held 24 disease-focused meetings to discuss treatments and unmet needs as part of the 2012 prescription drug user fee renewal.

But having fulfilled that commitment, the agency said it did not intend to maintain a regular meeting schedule going forward. (Also see "FDA Ends Regularly Scheduled Patient-Focused Drug Development Meetings" - Pink Sheet, 28 Sep, 2017.)

The listening sessions in the MOU may be a way for the agency to continue gathering patient opinions on various issues outside of the PFDD framework. FDA also wants to determine how best to collect patient experience data systematically so they can be used in drug development. (Also see "From Listening To Advising: The Maturation Of US FDA's Patient-Focused Drug Development Program" - Pink Sheet, 8 Jan, 2018.)

In addition, the agency has scheduled a May 9 public workshop to discuss orphan incentives and how they may apply to tissue agnostic therapies. Gottlieb has said the agency may consider changes to orphan designation policy to account for such therapies, including issuing guidance for gaining a designation based on molecular subtype. (Also see "Keytruda's New Tissue-Agnostic Indication Could Prompt Orphan Policy Changes" - Pink Sheet, 16 Oct, 2017.)

MOU Also Allows Safety Information Dissemination

The MOU also allows FDA to work with NORD to disseminate the agency's public health alerts through web links, provide content for patient informational, educational and training programs and broaden opportunities "to educate and communicate with rare disease patient communities about product safety issues and reporting of adverse events through FDA adverse-event reporting systems."

FDA may be attempting to find a way to ensure the best safety information about rare disease treatments, many of which are prescribed off-label, can get to patients, especially given the widespread use of internet forums and similar resources for those purposes.

NORD's reputation likely caries a lot of weight among rare disease patients, and using its distribution list and logo may better ensure a rare-disease-specific communication reaches the necessary patients and providers.

The MOU was announced during Rare Disease Week, which featured advocates lobbying on Capitol Hill. (See sidebar above.)

Orphan Designation Request Consult Process Improved

FDA also continues to tweak the orphan designation process to make it simpler and faster. A new inter-center consult process has been developed "to streamline and standardize our communications process," Gottlieb said in the blog post.

FDA said the consults are modeled after those established for combination products with established timelines and electronic forms that ensure all staff have the most updated information. Data collected over time will allow for process improvements and training for subsequent phases, the agency said.

"This iterative approach will ensure implementation of a robust consult request process that enables efficient, effective collaboration of orphan drug designations," the agency told the Pink Sheet. "We believe that this kind of nimble, adaptive cooperation reflects the future of medical product development and review."

The orphan products designation program had been suffering from a backlog in mid-2017, when Gottlieb directed staff to eliminate it and return to its goal of reviewing requests within 90 days. (Also see "FDA's Orphan Designation Backlog Plan Focuses On Efficiency, Not Staff" - Pink Sheet, 29 Jun, 2017.)

The agency is also piloting a new "fillable" form for orphan designation requests. The pdf document allows sponsors to type the necessary information directly into the form, which will simplify the process for sponsors and "make it more efficient for FDA to review," Gottlieb said.

However, sponsors still must copy the completed form and the necessary attachments onto a CD, include a signed cover letter, and send it via mail to the agency.

Direct entry of the requests into FDA's systems may make the process simpler and reduce review times. FDA recently began accepting meeting requests from generic sponsors interested in developing complex products via a web portal. (Also see "FDA Web Portal Eases Pre-ANDA Meeting Request Process" - Pink Sheet, 9 Oct, 2017.) But the agency still requires sponsors to email requests for a pre-assigned ANDA number. (Also see "FDA Web Portal Eases Pre-ANDA Meeting Request Process" - Pink Sheet, 9 Oct, 2017.)