Unmet medical need and market access: enabler or barrier to innovation?
Designed to help healthcare stakeholders identify more urgent health needs, the concept of “unmet medical need” is being increasingly applied formally and informally to set priorities during the development, assessment, and reimbursement of new drugs and medical devices. Perhaps counterintuitively, an “unmet need” does not refer to a disease or health condition but rather to any possible unresolved challenge with its rapid diagnosis, effective treatment, or management. As a result, unmet needs are not static but are instead “moving targets” that evolve with the rapidly changing landscape of available medical technologies and treatments. Therefore, when a healthcare innovation supersedes the best standard of care that is currently used by healthcare providers, it reveals an unmet need that nobody had previously considered.
In the UK, drugs and medical technologies that are perceived to address an unmet need enjoy higher priority during regulatory approval and health technology assessment (HTA). Knowing that a proposed innovation will benefit from this status reduces the risk involved in researching and developing it in the first place. However, whether regulatory and assessment agencies will grant that status is revealed later in the process, after developers have already had to invest substantial amounts of time and resources.
In this way, it is uncertain whether drugs or technologies addressing an unmet need will be prioritised for regulatory approval and reimbursement. Such uncertainty can act as a barrier to innovation as developers are likely to be less incentivised to invest their time and resources in the development of new interventions. This blog explores how this barrier may be removed by aligning various healthcare stakeholders, such as decision makers or analysts, around the definition of an unmet need.
Towards a definition of unmet medical need
Unmet need can be defined from the societal perspective as the interaction of three healthcare perspectives: (1) prioritising those who are the worst off, (2) ensuring equality of access, and (3) maximising overall population health (Figure 1). To make things more difficult, there is no standard consensus definition of unmet need. One review of grey literature identified 16 definitions. Of these, 100% considered currently available treatments, 38% (6 of 16) took into account disease severity or burden, and 6% (1 of 16) took into account the population size. This led the authors of the review to propose a definition of unmet need comprising of the interaction between alternative treatments, patient population and disease severity (Figure 2).
In addition to the lack of a standard definition of unmet need, various healthcare stakeholders weigh the priorities presented in Figure 2 differently. This presents another complication to defining unmet need as their priorities will then influence their decision-making. Aligning stakeholders around the criteria of unmet needs can strongly influence how they evaluate a proposed drug or technology as beneficial for individual patients, the greater society and the healthcare system as a whole.
Figure 1. Conceptualisation of an unmet medical need from a societal perspective as presented in a webinar by the Office of Health Economists (adapted)
Figure 2. Definition of unmet need based on a review of grey literature
Role of unmet need in healthcare intervention development
Unmet need is often used to signal that a proposed healthcare innovation should receive special priority at every step in its development, from initial discovery to pricing and reimbursement (Figure 3). Healthcare decision makers must strike a balance between enabling early patient access to novel technologies against the uncertainty around their benefits and harms. Understandably, reimbursement agencies differ in how they take unmet need into account in their decision-making. For example, the Scottish Medicines Consortium (SMC) allows for two fast-track decision categories, “interim accepted” and “accepted restricted advice”, in the case of healthcare interventions that fulfil an unmet need based on the scientific opinion of the Early Access to Medicines Scheme of the Medicines and Healthcare products Regulatory agency (MHRA). In this way, the SMC aligns with the regulator on the definition of unmet need and its role in market access. In contrast, the UK National Institute for Health and Care Excellence (NICE), merely state that their committee will decide whether a proposed intervention addresses an unmet need during the health technology assessment process, without describing how that decision is made or how it may influence reimbursement.
This heterogeneity in how reimbursement agencies deal with unmet need increases the risk and complexity the developers face when deciding to invest in the development of new drugs or medical devices. Aligning stakeholders’ perspectives around the definition of an unmet need and its implications for achieving healthcare goals may prove beneficial to the healthcare intervention evaluation processes.
Figure 3. Examples of how the concept of unmet need can accelerate development of healthcare interventions
Abbreviations: EAMS, Early access to medicines scheme; EMA, European Medicines Agency; FDA, U.S. Food and Drug Administration; HTA, Health Technology Agency; MHRA, Medicines Healthcare products Regulatory Agency; NICE, National Institute for Health and Care Excellence; PRIME, Priority Medicines; R&D, Research and Development; SMC, Scottish Medicines Consortium.
Unmet needs are always evolving. Defining an unmet need early on in the development of an intervention increases the chances of receiving preferential treatment by regulatory and HTA bodies. This can give manufacturers certainty and aid stakeholders in prioritising where to concentrate their efforts and push innovation. Although regulatory and HTA bodies have processes to prioritise innovations which address an unmet need, there is not a consistent definition of unmet need. This gives rise to confusion and acts as a barrier to innovation. An operational definition and framework for discussing unmet needs with healthcare stakeholders is required to remove this barrier.
How Symmetron can help
Across all our activities, our team at Symmetron is driven by the mission to increase the number of effective treatments available to patients. To achieve this, we always utilise the most effective tools and practices to help our clients secure official recommendation and reimbursement for their healthcare innovations. Let our extensive experience with successful submissions to NICE, SMC and other regulatory agencies around the world help you align your intervention with unmet needs from the earliest stages of research and development to ensure the strongest case for reimbursement and market access.
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