gress will not take the view that development of “cool tools” should be seen as a metric of success for CAN. It would be a success for NCATS, but for CAN, he said, “the success is cures, treatments, devices, therapies, behavioral interventions.” Goodman noted that he sees CAN’s responsibilities as including an examination of the process for product development so that CAN advances the field and not just an individual project, thus serving the public good. He noted that industry already has incentives in regard to development of cures, and it is in the scope of a federal program such as CAN to do more. Parikh added that there are areas where treatments are needed but are not being developed. Vertex Pharmaceuticals is an example of a company driven by patient advocates where there is a high need but not much interest in the pharmaceutical industry. Kristin Schneeman, Program Director, FasterCures, offered a potential semantic clarification. She suggested that CAN, as implied by its name, is accelerating cures, not necessarily producing them, which could be helpful in framing CAN’s metrics for success.

The discussion also touched on the role that CAN could play in helping partnerships of patient advocates, small companies, and academic researchers get high need treatments through the approval process. Goodman noted that CAN could require the developer of a product to have a plan for project management and engagement with FDA. NIH could partner with another group that could provide product developers with assistance in these areas.

Buckman-Garner added that, in this regard, CDER has been involved in an exploratory data submission program, which is designed to encourage early conversations about what is needed in product development. This program has led to meetings with groups from academia, industry, and NIH to have discussions about how to get through the regulatory process.

Neil said he could think of at least half a dozen cases of high need populations with no treatment alternatives where the problems are potentially tractable based on current scientific understanding. These cases may not be commercially attractive projects, but they offer excellent case studies of whether the drug development ecosystem could work in a different way to come up with an effective, safe, small-molecule treatment for a high need population.



The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement