TY - JOUR
T1 - The TREAT-NMD advisory committee for therapeutics (TACT)
T2 - An innovative de-risking model to foster orphan drug development
AU - Heslop, Emma
AU - Csimma, Cristina
AU - Straub, Volker
AU - McCall, John
AU - Nagaraju, Kanneboyina
AU - Wagner, Kathryn R.
AU - Caizergues, Didier
AU - Korinthenberg, Rudolf
AU - Flanigan, Kevin M.
AU - Kaufmann, Petra
AU - McNeil, Elizabeth
AU - Mendell, Jerry
AU - Hesterlee, Sharon
AU - Wells, Dominic J.
AU - Bushby, Kate
N1 - Funding Information:
We acknowledge funding support for TREAT-NMD from the European Commission through FP6 (contract number 036825) and the Second Public Health Programme (contract number 2012 3307), as well as support from the Medical Research Council (grant number MR/K501074/1), US department of defence (Award W81XWH-11-1-0782 K Nagaraju, CNMC, USA), Parent Project Muscular Dystrophy and Cure Duchenne. Support from patient organisations - Parent Project Muscular Dystrophy (PPMD), Cure Duchenne, Association Française contre les Myopathies (AFM), Jesses Journey, Muscular Dystrophy Campaign (MDC), Muscular Dystrophy Association (MDA), Action Duchenne, Cure CMD, and Foundation to Eradicate Duchenne (FED).
Publisher Copyright:
© 2015 Heslop et al.; licensee BioMed Central.
PY - 2015/4/23
Y1 - 2015/4/23
N2 - Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials. The ability to prioritise drug development according to objective criteria is particularly critical in rare diseases with large unmet needs and a limited numbers of patients who can be enrolled into clinical trials. TREAT-NMD Advisory Committee for Therapeutics (TACT) was established to provide independent and objective guidance on the preclinical and development pathway of potential therapies (whether novel or repurposed) for NMD. We present our experience in the establishment and operation of the TACT. TACT provides a unique resource of recognized experts from multiple disciplines. The goal of each TACT review is to help the sponsor to position the candidate compound along a realistic and well-informed plan to clinical trials, and eventual registration. The reviews and subsequent recommendations are focused on generating meaningful and rigorous data that can enable clear go/no-go decisions and facilitate longer term funding or partnering opportunities. The review process thereby acts to comment on viability, de-risking the process of proceeding on a development programme. To date TACT has held 10 review meeting and reviewed 29 program applications in several rare neuromuscular diseases: Of the 29 programs reviewed, 19 were from industry and 10 were from academia; 15 were for novel compounds and 14 were for repurposed drugs; 16 were small molecules and 13 were biologics; 14 were preclinical stage applications and 15 were clinical stage applications. 3 had received Orphan drug designation from European Medicines Agency and 3 from Food and Drug Administration. A number of recurrent themes emerged over the course of the reviews and we found that applicants frequently require advice and education on issues concerned with preclinical standard operating procedures, interactions with regulatory agencies, formulation, repurposing, clinical trial design, manufacturing and ethics. Over the 5 years since its establishment TACT has amassed a body of experience that can be extrapolated to other groups of rare diseases to improve the community's chances of successfully bringing new rare disease drugs to registration and ultimately to market.
AB - Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials. The ability to prioritise drug development according to objective criteria is particularly critical in rare diseases with large unmet needs and a limited numbers of patients who can be enrolled into clinical trials. TREAT-NMD Advisory Committee for Therapeutics (TACT) was established to provide independent and objective guidance on the preclinical and development pathway of potential therapies (whether novel or repurposed) for NMD. We present our experience in the establishment and operation of the TACT. TACT provides a unique resource of recognized experts from multiple disciplines. The goal of each TACT review is to help the sponsor to position the candidate compound along a realistic and well-informed plan to clinical trials, and eventual registration. The reviews and subsequent recommendations are focused on generating meaningful and rigorous data that can enable clear go/no-go decisions and facilitate longer term funding or partnering opportunities. The review process thereby acts to comment on viability, de-risking the process of proceeding on a development programme. To date TACT has held 10 review meeting and reviewed 29 program applications in several rare neuromuscular diseases: Of the 29 programs reviewed, 19 were from industry and 10 were from academia; 15 were for novel compounds and 14 were for repurposed drugs; 16 were small molecules and 13 were biologics; 14 were preclinical stage applications and 15 were clinical stage applications. 3 had received Orphan drug designation from European Medicines Agency and 3 from Food and Drug Administration. A number of recurrent themes emerged over the course of the reviews and we found that applicants frequently require advice and education on issues concerned with preclinical standard operating procedures, interactions with regulatory agencies, formulation, repurposing, clinical trial design, manufacturing and ethics. Over the 5 years since its establishment TACT has amassed a body of experience that can be extrapolated to other groups of rare diseases to improve the community's chances of successfully bringing new rare disease drugs to registration and ultimately to market.
KW - De-risking
KW - Drug development
KW - Neuromuscular disease
KW - Rare disease
KW - Review
UR - http://www.scopus.com/inward/record.url?scp=84928633900&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84928633900&partnerID=8YFLogxK
U2 - 10.1186/s13023-015-0258-1
DO - 10.1186/s13023-015-0258-1
M3 - Article
C2 - 25902795
AN - SCOPUS:84928633900
SN - 1750-1172
VL - 10
JO - Orphanet journal of rare diseases
JF - Orphanet journal of rare diseases
IS - 1
M1 - 49
ER -