Unmet Medical Needs
Addressing Unmet Medical Needs (UMN) is a cornerstone of pharmaceutical innovation as we aim to develop medicinal products that will improve and extend patients’ lives. UMN is a relevant concept throughout the whole value chain from medicine discovery to patient access.[1]
For the first time in EU legislation, there is a proposal to define UMN and High UMN for orphans in order to direct pharmaceutical R&D investments. Given the diversity of stakeholders and incentives involved, aligning on a common definition to assess those needs is an important and, at the same time, challenging exercise. We understand an unmet medical need as “a condition that is not adequately prevented, treated or diagnosed by authorized interventions”.[2]
Incentives for underserved areas are needed. However, in order for these to work, they need to be effective and predictable, ensuring the right patient-centric framework that can support innovation and not exclude certain patient populations or fail to encompass future diseases. Based on the proposal from the European Commission on the revision of the EU’s General Pharmaceutical Legislation, issued in April 2023, we aim to contribute to the ongoing debate on defining UMN with concrete insights:
- Limiting UMN focus to a life-threatening disease, or seriously debilitating disease with remaining high morbidity or mortality, risks further deprioritizing conditions such as migraine in investment and priority-setting decisions by a range of stakeholders. Migraine is already overlooked by policymakers while it affects 41 million adults in Europe and is a disease with an enormous social and economic impact. It’s the most common cause of disability in young women. The annual per capita cost for migraine amounts to € 1,177. Although 93% of this is attributed to indirect cost, it still results at € 48 billion per year. To put this into perspective, the EU Health Emergency Preparedness and Response Authority (HERA) budget is roughly € 1 billion per year.
- The development of medicines takes many years. It is, however, impossible to know in advance whether a particular investment in a given project will eventually address a specific UMN. Given the long and risky development timelines, it is possible that the remaining UMN can change during this period – for instance, a company may be disincentivised to pursue a candidate in an area where there is already a product at an advanced development stage. If what is considered an UMN can change after the investment in R&D has been made, or is defined very narrowly, this would lead to a lack of predictability of the incentive, weakening its impact and therefore reducing or removing the stimulus for companies to invest in R&D into relevant treatments to meet patients’ needs.
For these and other reasons, a definition of UMN needs to take the evolution of science and patients’ needs as a starting point.
Finally, having distinct standards for UMN is challenging, not least because it raises ethical concerns: this implies that some UMN are of less importance, either to patients or society, which would be inappropriate. While we believe additional incentives to encourage further investment in areas with specific additional challenges are needed, this should not be understood as an attempt to define a scale or gradation of different levels of UMN.
With these principles in mind, Pfizer remains committed to partnering with European policymakers to ensure any definition of UMN is patient-centric, inclusive, future-proof and fostering a predictable incentive framework for the development of life-changing medicines.