Chapter 11. Harnessing the Potential of Early Access: Advancing Timely Patient Access to Innovative Medicines in a Sustainable Way

Abstract

The way medicines are developed, regulated and priced determines how quickly patients have access to safe, effective and affordable medicines. Patients are increasingly requesting access to investigational medicines and regulators are developing guidelines for additional data collection outside the clinical trial setting. Payors and healthcare policy makers worry about the rising prices of new medicines and are searching for effective policy instruments. Developing effective pharmaceutical policy requires a thorough understanding of the working of the medicine development ecosystem. Currently the smaller players are the drivers of discovery and innovation but are unable to compete in the approved medicine market as they lack the financial capacity to progress to the commercial phase, which limits competition and pricing pressure. Experimenting with innovative funding models for advanced therapy medicinal products, personalized treatments and medicines targeting (progressive) orphan diseases is expected to occur more frequently. One such model that supports smaller medicine developers, and helps them survive transitioning to the commercial stage, is the funding of early access programs. Countries with universal healthcare systems are well suited to experiment with an adaptive funding model for early access that includes pay-for-performance elements based on evolving real-world data and evaluation. Such an approach has the potential to contribute to universal access to new treatments in a sustainable way.