Having a clear understanding of patient priorities and an effective means to assess clinical relevance is critical, particularly in rare diseases where advances can be delayed due to a lack of precedent with the groups that must evaluate the efficacy of novel treatments.
Neurofibromatosis Therapeutic Acceleration Program (NTAP) consults with regulatory and other governmental agencies to ensure our initiatives will provide the data necessary to meet the rigorous requirements for drug approval and reimbursement support.
In targeting pNF, NTAP has interfaced extensively with the FDA as part of its role on the clinical study evaluating the small molecule inhibitor Selumetinib. In 2017, NTAP played a role in helping to establish the Response Evaluation in Neurofibromatosis & Schwannomatosis International Collaboration cNF working group , through which NTAP has regular interaction with representatives from the FDA to address the challenges in the clinical development of cNF therapies.
The US Food and Drug Administration (FDA)
The European Medicines Agency (EMA)
Office of Rare Diseases Research (ORDR-NCATS)