Key drivers in today’s biopharma market include the shifting priorities and information requirements of major stakeholders, focused on the following:
  • Safety concerns, including a heightened need to better understand and manage safety signals and risk-benefit in diverse populations and health care settings after a product is launched. To meet this need, the US Food and Drug Administration is considering creating a new “super office” of drug safety within the Center for Drug Evaluation and Research to oversee FDA’s multiple drug safety initiatives.
  • Comparative effectiveness, focused on proof of clinical benefit versus the standard of care in a naturalistic environment outside of randomized clinical trials (RCTs).
  • Real-world value, entailing a demonstration of incremental economic impact to stakeholders compared with existing therapies.
  • Regulatory acceleration, with a call for more efficient approaches to approval and a faster time to market, especially in areas of acute unmet medical need. Taking cancer as an example, to date, FDA has granted accelerated approvals to cancer drugs 49 times. Of these, 27 have completed postmarketing studies verifying benefit, four have completed studies and await an FDA decision, five failed to confirm benefits in studies, six have been on the market for more than 2 years and have not completed studies, and seven have been on the market for less than 2 years.
These concerns about safety, value, and outcomes are driving demand for more robust evidence about biopharmaceuticals and other medical interventions. Data generated by traditional premarket clinical development typically do not provide a comprehensive view of a product’s ultimate performance to allay these stakeholders’ concerns. This de facto data shortfall limits market access. Consequently, a new paradigm of complementing and integrating real world research with RCTs is on the rise to provide a holistic view of biopharmaceuticals.