NIH-NCATS Related Repurposing References
Austin, C.P. (2020) DIA Global Forum
Highlight: In order to break the logjam of repurposing, NCATS will need to consider a much broader research agenda than it has to date. Attendees were enthusiastic and cautiously optimistic that with a stepwise, data-driven, multi-sector, coordinated, and experimental but bold approach, the promise of off-patent repurposing for patient and public health can at last be realized.
Galson, S., Austin, C.P., Khandekar, E., Hudson, L.D., DiMasi, J.A., Califf, R., and Wagner, J.A. (2020) Nature
Highlight: More tools to stratify the patient population and assess treatment benefit, as well as more critical thinking on the selection of trial end points, could have made a difference in some trials. Sharing data from late-stage failures could promote the development of biomarkers, clinical outcome assessments and clinical trial simulation tools that would improve clinical trial design and consequently the likelihood of success. Early efforts to share clinical trial data, such as ClinicalStudyDataRequest, Project Data Sphere, SOAR, TransCelerate, Vivli, Critical Path for Alzheimer’s Disease and the YODA Project, are promising. However, competition and reputational impact remain barriers to information sharing when it comes to late-stage failures in drug development. Funders, academic researchers, sponsors, regulators, health systems and practicing physicians all have a role to play to increase success rates.
Colvis, C.M. and Austin, C.P. (2015) ASSAY and Drug Development Technologies
Highlight: I t has been 2 years since the National Center for Advancing Translational Sciences (NCATS) announced its intent to test a new strategy for establishing collaborations between pharmaceutical companies and academic partners to repurpose company agents (new molecular entities and biologics) that were phase 2a–ready, but had not been approved by the Food and Drug Administration (FDA) for licensure. The new strategy would use template agreements to allow an academic institution and pharmaceutical company to quickly reach an agreement about the terms of the collaboration. In May 2012, NCATS posted template collaborative research agreements and template confidential disclosure agreements for use by collaborating academic institutions and the pilot program’s first three pharmaceutical company partners, AstraZeneca, Eli Lilly and Company, and Pfizer. At the same time, NCATS published a request for information to get feedback on the template agreements. The initial response from academic institutions on the template agreements was not one of overwhelming enthusiasm. Although collaborations between academic institutions and pharmaceutical companies have been going on for years, the willingness of academic institutions to use template agreements that the NIH had drafted with pharmaceutical companies remained to be seen.
Colvis, C.M. and Austin, C.P. (2015) ASSAY and Drug Development Technologies
Highlight: I t has been 2 years since the National Center for Advancing Translational Sciences (NCATS) announced its intent to test a new strategy for establishing collaborations between pharmaceutical companies and academic partners to repurpose company agents (new molecular entities and biologics) that were phase 2a–ready, but had not been approved by the Food and Drug Administration (FDA) for licensure. The new strategy would use template agreements to allow an academic institution and pharmaceutical company to quickly reach an agreement about the terms of the collaboration. In May 2012, NCATS posted template collaborative research agreements and template confidential disclosure agreements for use by collaborating academic institutions and the pilot program’s first three pharmaceutical company partners, AstraZeneca, Eli Lilly and Company, and Pfizer. At the same time, NCATS published a request for information to get feedback on the template agreements. The initial response from academic institutions on the template agreements was not one of overwhelming enthusiasm. Although collaborations between academic institutions and pharmaceutical companies have been going on for years, the willingness of academic institutions to use template agreements that the NIH had drafted with pharmaceutical companies remained to be seen.
Pahud, D., Mitchell, L., Wilbanks, J., Stevens, M., Weir, S., McBride, J., Portilla, L., Pezalla, E., Nameth, M., O'Donoghue, B., Rai, A. K., Lappin, D., Ogle, M., and Howell, S. (2014) SSRN
Abstract: A diverse set of stakeholders within health care came together in the fall of 2013 to brainstorm potential new commercial paths for repurposed drugs for the treatment of rare diseases. This report highlights some of the identified solutions that potentially could create the right set of incentives for the efficient and accelerated development and delivery of needed therapies for patients suffering from rare diseases.