Patient Advocacy and Changing Paradigm in Drug Access

Background: In the process of drug development and drug approval, the pharmaceutical industry is challenged with increasing cost constraints, a growing need for real-world evidence and heavier regulatory scrutiny. Patient advocacy groups can be a valuable partner to the pharmaceutical industry in speeding up drug access. Patient advocates possess practical rather than purely scientific knowledge that is valuable for both drug development and drug approval. It was recognized quite early the patient advocacy groups and the pharmaceutical industry have similar goals. This understanding resulted in supporting patient involvement across the entire process of drug development, from discovery to USFDA approval for marketing. Support received by the patient advocacy groups include fundraising for research and development, organizing patients to expedite clinical trials, lobbying government and payer organizations to create public awareness, building support for research funding, and so on. Patient advocacy groups also back research for “orphan” diseases where there is a desperate need for new therapies. The recent success of Vertex and Sprout Pharmaceutical highlights the power of the patient voice in healthcare decision-making especially for orphan diseases. Conclusion: Working with patient groups can thus lead to a win-win situation for both the FDA and pharmaceutical industry.