Expediting Patient Access to New Medicines: A Call to Action, London, January 26–27, 2010

Abstract

In the last 50 years the average life span has increased by 10 years, largely due to advancements in pharmaceutical drug development, primarily within the developed world. What will drive this over the next 50 years?

Improved patient access to drugs is a critical goal for all stakeholders in drug discovery, development, and delivery. However, drug access levels for patients continue to decrease, driven by, for example, stricter regulatory policies, lower R&D output, stronger payer cost pressures, and a complex legal environment. Given the importance of health and well-being, it is critical for stakeholders to better understand these hurdles and develop high-impact solutions that significantly increase patient access to new medicines. While pharmaceutical companies have had some limited success in overcoming R&D hurdles, a multistakeholder holistic approach is necessary to develop paradigm-changing solutions.

A meeting of 12 distinguished health care experts was convened on January 26–27, 2010, in London, UK, to address this issue. The participants included government regulators, health care policy experts, industry leaders, health economists, health care attorneys, patient advocates, and academics. The primary objectives of the meeting were to further understand the hurdles to patient access and to develop high-impact global solutions. Topics discussed included the following:

• The nature of the problem

• Initiatives currently underway

• Insights on the challenges and barriers that inhibit patient access to new and innovative medicines

• Prioritizing ideas to facilitate meaningful strategies for expediting patient access to new medicines

• A potential path forward for implementing the priority ideas from the meeting

Five of the ideas that were discussed are as follows:

• 1. A nonprofit, independent, patient-driven drug assessment agency to provide an appraisal of risks versus benefits of new drugs to both individuals and the society as a whole, unbiased by nonclinical measurements such as the quality adjusted life year (QALY)

• 2. An academic institute of regulatory science to drive research into the discipline of drug regulation

• 3. A new Asia-Pacific panregional regulatory agency to provide centralized regulatory support

• 4. Better sharing of data within the pharmaceutical industry (eg, safety databases, genomic and small-molecule libraries, negative outcomes) directly, or potentially through government bodies

• 5. Enhanced use of private-public partnerships in new product development building on learning from organizations such as the Bill and Melinda Gates Foundation, the FDA Critical Path Initiative, the Innovative Medicines Initiatives, and the Cardiac Safety Research Consortium.

The column reviews important discussion and insight emerging at the meeting, and is intended as a call to action highlighting the importance of improved global patient access to new medicines. The first two of the ideas just described were viewed as top priority based on feasibility and potential impact.