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"For people living with rare diseases, time is of the essence," said Joseph Scheeren, PharmD, C-Path president and chief executive officer. "By leveraging the rare disease community access and data of NORD and the data curation, aggregation, governance, and advanced analytics expertise of C-Path, we are poised to make a significant impact on rare disease drug development by providing quality data that will inform clinical trial design and accelerate the development of therapies."
"Currently, more than 25 million people in the United States are affected by one or more of the over 7,000 rare diseases," said Peter L. Saltonstall, NORD president and chief executive officer. "Drug development for these diseases is often impeded due to the low affected patient numbers and a limited understanding of how rare diseases progress or how to measure clinical improvements. FDA-approved treatments exist for only 10% of rare diseases; with this collaboration we can change that statistic for the better for our rare community."
The project between the organizations will kick off at a September launch meeting in Bethesda, MD, and will aim to reduce barriers for the development of new treatments and cures for rare diseases.
Last year, NORD announced a partnership with the FDA to help the agency's medical reviewers better understand the experiences of patients with rare diseases.
Under the partnership, the FDA and NORD are working together in identifying patient community representatives and coordinating listening sessions where patients with rare diseases can share their experiences with FDA review division staff.
The listening sessions are organized through the FDA's patient affairs staff in cooperation with NORD. The patient affairs staff is working with the review division to draft questions for the patients and with NORD to facilitate the discussion between the requesting review division and the patient community.
"These listening sessions will provide FDA review division staff with better insight into what is important to patients in managing their diseases and improving their quality of life,” said Saltonstall.
"It is important for FDA to understand, from the patient perspective, disease burden, management of symptoms, daily impact on quality of life, and patients' risk tolerance. Patients and caregivers bring a pragmatic, realistic perspective about what they are willing to deal with in terms of potential risks and benefits for new therapies," he said.
"Our goal is to facilitate the patient's voice being heard more directly in the regulatory decision-making process, an invaluable opportunity for the rare disease community as a whole to advance medical research and new product approvals,” he concluded.
