Unmet medical need as a policy making tool
The concept of unmet medical need (UMN) is meant to help the research and healthcare communities distinguish more pressing patient and societal health needs from the myriad of other health needs.
It plays an important role in investment and priority-setting decisions by a range of stakeholders, including Regulators, HTA Agencies, Payers, academics and the pharmaceutical industry. Identifying a particular condition or disease area as an UMN is intended to signal its health policy significance, stimulate research activities and incentivise the development of innovative treatments, diagnoses or health technologies in these areas. Incentives associated with the identification of an UMN can take the form of preferential access to public research funds, access to alternative or accelerated regulatory pathways, consideration of UMN as a value element in HTA, and financial incentives or innovative payment models in reimbursing the health benefits a new treatment delivers.
Finding a narrow definition of unmet need is problematic
Defining unmet need is personal, patients living with chronic conditions will define their unmet needs very differently to people facing a life-limiting disease. For long-term conditions, how medicines fit in to daily life is critical, including reduced side-effects, whereas if you are facing a terminal illness you may value extension of life above all else, others may prioritise freedom from pain. Health systems may value treatments that reduce the burden on secondary care more than treatments delivered in hospital. It underlines the need to ensure that a broad range of stakeholders and particularly the patients’ voice is included in any process to define and use unmet need as a policy tool.
These cases show examples of unmet medical need from different stakeholder perspectives
Why a narrow definition of UMN hampers research and development
Attempting to limit incentives only for treatments that fit a certain narrow definition of unmet need risks excluding the development of important therapies for patients. You have to incentivise broad avenues of scientific discovery, then follow the promising science to make breakthroughs that can be applied across a number of potential diseases areas. Narrowing the definition of unmet need to focus on one particular disease area would stifle rather than accelerate the development of new medicines. Understanding the nature of innovation, how and where it happens, is critical to addressing unmet needs. There is no better example than the use of mRNA in COVID-19 vaccines. Originally researched as a potential oncology treatment, mRNA was studied for decades with little or no clinical applications, however, only through that research did we have an asset that could be used to protect citizens against the coronavirus.
Narrowing the definition of unmet need would lead to the unintended consequence of disincentivising companies to invest in R&D that may have addressed patients’ unmet medical needs and reduces the overall predictability. The development of medicines takes many years and is always done with the ambition to address an UMN. It is however impossible to know in advance whether a particular investment in R&D will eventually address a specific UMN. Given the long and risky development timelines, it is possible that a definition of an UMN can change during this period of development. If what is considered an UMN can change after the investment in R&D has been made, or is defined very narrowly, this leads 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. Moreover, R&D aimed at addressing a certain disease area could lead to positive developments, and even breakthrough innovation, elsewhere; limiting incentives to certain defined categories therefore disregards the reality of science. Any proposed linkage between RDP protection and development of medicines for UMN would therefore be artificial and would not spur R&D investments. In fact, the reduction of the baseline RDP period and/or the reduction of RDP for products that do not address “agreed UMN” would only diminish the effectiveness of the incentive system, which has allowed Europe to research and develop over 200 innovative products for rare disease patients who had no other alternatives previously, boost research into paediatric medicines, and cause a flourishing of biotech SMEs throughout the region.
Driving R&D to meet societal needs: Perspective matters, inclusivity is crucial
Taking the evolution of science and patient needs as a starting point, we understand an unmet medical need as a condition that is not adequately prevented, treated or diagnosed by authorised interventions. This is a context-neutral understanding as UMN has different meanings depending on different stakeholders’ perspectives and decision-making level and a broad understanding of UMN should recognise and tackle the many ways in which UMNs manifest. Within the context-neutral understanding of UMN, certain areas can be incentivised based on the principles of distributive justice.
Therefore, EFPIA proposes for the UMN definition to apply to pathways that currently rely on the concept of UMN (PRIME, conditional approval, accelerated assessment). Eligibility criteria for incentives and rewards must be tailored to the areas of UMN that they aim to address. Considering that the current system already aligns innovation to UMN, existing incentives effectively direct R&D towards areas of UMN and existing rewards from innovation are skewed towards medicines that offer the greatest value.