Government agencies, industry, and patient advocacy groups all agree that a patient-centered perspective is vital during the processes of drug development, clinical trials, benefit/risk assessment, and regulatory review. However, legal barriers and proprietary constraints complicate when patients can be brought to the table and integrate their input.
At the FDA’s annual Patient Network meeting, “Demystifying FDA: An Exploration in Drug Development”, there was a concerted effort among all stakeholders to increase collaboration. The FDA Safety and Innovation Act (FDASIA) requires the FDA to implement strategies that will take the patient viewpoint into account throughout medical product development and regulatory discussions. One example of this initiative is the Patient-Focused Drug Development Initiative, which consists of 20 public meetings targeted toward specific diseases within a five-year time frame. Results from the patient-focused meetings will be shared with FDA reviewers and posted online.
A challenge for the FDA and medical research community, where a patient-centered approach is of high value, is a collection of natural history data. Natural history narrowly defines what corporations want to pursue during product development; furthermore, the lack of natural history data within clinical development for rare diseases can affect efficacy.
In order for the FDA, patient advocacy groups, and industry to achieve meaningful patient-centered participation, communication empowered with action is the key. Patients, physicians, and FDA reviewers have different perspectives on endpoints and outcomes; therefore, patient involvement should be thoughtfully woven into developmental processes and not implemented haphazardly.
The Patient Network meeting and Patient Focused Drug development program are great starting points to facilitate this discussion, but stakeholders will need to ensure that patients’ voices are not only heard, but are also in the forefront during critical development decisions.
Leleah Robinson is a National Urban Fellow based in NORD’s Washington DC office.
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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