Ensuring access to medicines: How to stimulate innovation to meet patients' needs?

This policy brief is one of a series on addressing market and policy failures in the pharmaceutical sector that was prepared for the Austrian EU Presidency.

It aims to inform discussions about stimulating more meaningful productivity in terms of R&D. More specifically, it explores how R&D efforts can be steered to areas of unmet clinical needs and how efficiency in the R&D process can be increased. It also explicitly considers concrete options for strengthening cooperation between EU Member States in this context.

The brief shows how only a comprehensive approach that combines initiatives to guarantee funding for R&D, optimize evidence generation and align regulatory requirements can effectively tackle innovation deficits. An overall vision with greater policy coherence and backed by strong political commitment and transparency is needed.

The brief’s key messages are:

• The development of innovative medicines is essential for making progress in preventing and treating diseases. However, the high price tags put on new medicines do not always reflect the value added for patients. Also, persisting unmet clinical need in the population suggests a misalignment of pharmaceutical research and development efforts.
• There is a growing consensus that existing policies need to be rethought and new approaches need to be found to strike the delicate balance between stimulating true innovation, particularly towards addressing unmet needs, and ensuring both financial sustainability for health systems and accessibility for patients.
• While small and medium-sized enterprises (SMEs), academia and public institutions play a major role in driving innovation and enriching the industry’s pipeline, the commercialization of new products is almost exclusively in the hands of large companies, often as a result of mergers and acquisitions. This is a concern for the sustainability of pharmaceutical research and development (R&D) infrastructure.
• The impact of public funding on pharmaceutical innovation cannot be underestimated. A stronger implementation of public interest provisions along the life cycle of pharmaceuticals, including “fair return of investment”, is required.
• For public finance of R&D to facilitate better alignment of innovation with unmet clinical needs, more discrimination could be introduced in the reward system. Through limiting the risks, reducing R&D costs and/or increasing the innovation potential, incentives can be created for industry to re-embrace revolutionary – or even disruptive – innovation.
• More can also be done to improve coordination and priority-setting across R&D efforts, ideally globally, but with further refinement from an EU level to reflect regional priorities. In this respect, the creation of an entity to monitor clinical need, in conjunction with inequalities in access to essential medicines, merits consideration.
• Improving the efficiency of evidence generation in clinical research is not only good for driving down the costs of clinical trials, it can also help to remediate some of the related technical and ethical challenges, such as the fragmentation and duplication that unnecessarily expose patients to risk; the lack of comparative effectiveness data; the evidence gaps regarding specific patient groups and therapeutic areas; or the perceived conflicts of interest and related publication bias.
• Raising the bar for market entry by requiring that a new product demonstrate its superiority or equivalence to existing alternatives could encourage manufacturers to focus more on areas with limited treatment options and facilitate increased alignment with specifications applied in post-marketing evaluations for pricing and/or reimbursement (for example, Health Technology Assessment). Increased collaboration and alignment on evidentiary requirements between and within EU Member States are likely to simplify evidence generation for manufacturers as well as increase efficiency on the evaluators’ side.
• Only a comprehensive approach that combines initiatives to guarantee funding, optimize evidence generation and align regulatory requirements can effectively tackle innovation deficits. An overall vision with greater policy coherence and backed by strong political commitment and transparency is needed.