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.

The US Food and Drug Administration (FDA)

The European Medicines Agency (EMA)

Office of Rare Diseases Research (ORDR-NCATS)

Drug Development Tools (DDTs) Qualification Program