Baltimore, MD (March 15, 2013)
Neurofibromatosis Therapeutic Acceleration Program (NTAP) today announced a request for proposals (RFP) for the development of Patient Reported Outcome (PRO) measures that can be used to support clinical development of therapies against plexiform neurofibromas (pNFs), one of the most common tumors seen in individuals with neurofibromatosis type 1 (NF1).
PROs are reports generated directly by patients identifying what is most important to them about their condition and its treatment. There are currently no PRO measures that have been validated specifically for use in patients with pNFs.
NTAP will fund investigators to develop well-defined and reliable PRO measures that assess the human impact of NF1-associated pNFs. The program aims to identify and validate PROs that can be used as primary or secondary endpoints in clinical trials and product labeling applications.
“Being able to assess potential therapies using PROs is vital to ensure new treatments have an impact that matters to patients,” said Amanda Bergner, Director of Operations for NTAP who is leading the PRO initiative. “PROs are valuable research tools and apply across a broad range of clinical symptoms. We are delighted to partner with the FDA on this project through the Drug Development Tools (DDT) Qualification Program in Clinical Outcome Assessment. This partnership will allow NTAP to support the co-creation of powerful PRO measures that are pre-approved for use in clinical trials, which accelerates the progress toward therapeutics.”
NTAP is seeking proposals for projects of 24 to 30 months in duration that will create and validate PROs to assess the effect of therapies against pNFs. Potential applications will be accepted from independent investigators at US and international institutions, including public or private biotechnology companies, pharmaceutical companies, and not-for-profit organizations such as universities, hospitals, and eligible government agencies.