Frueh concluded with several provocative thoughts emphasizing the role of payers:
• Comparative-effectiveness evaluations will become increasingly required by payers because of the need to demonstrate that a new treatment is better than the standard of care.
• What if payers were to cover a drug only if it actually works?
• Payers will progressively move toward employing coverage with evidence development.
• Can payers act to encourage patients to participate in clinical trials or even help in recruitment?
• What if payers were to co-sponsor clinical trials or provide pharmacy, lab, and outcome data for research?
• Could payers partner with industry to develop more personalized medicines faster?
Over the next 5 years, Frueh said, the answers to these questions could reshape relationships in the drug discovery and development system. For example, if payers across the board were to embrace the coverage-with-evidence-development paradigm, “that would really change the way that we’d be looking at drugs and diagnostics.”
The NCATS Pharmaceutical Collection (NPC) is a comprehensive resource of 3,800 approved and investigational medicines that was designed to facilitate the repurposing of medicines by the scientific community.1 As a recent paper states, the NPC is “a definitive, complete, and non-redundant list of all approved molecular entities as a freely available electronic resource and a physical collection of small molecules amenable to high-throughput screening” (Huang et al., 2011).
Christopher Austin of NCATS at NIH demonstrated how the NPC can be used. Drugs approved in different jurisdictions throughout the world can be accessed. Searches can look for indication, target, drug name, and so on. A search on “migraine,” for example, returned 14 drugs that are approved worldwide. Clicking on a particular drug gives the mechanism of action, known targets, the regulatory status in different countries, and other information.
To demonstrate the utility of the collection for drug repurposement, Austin cited a recent example of successfully identifying a drug that could potentially be used for the treatment of chronic lymphocytic leukemia (CLL),