Inka Heikkinen
DIA Senior Scientist

urrently, much development is being steered toward disease areas where there has been little movement in R&D and high unmet medical need remains. The concept of unmet medical need is one of the guiding elements of numerous regulatory and market incentive programmes throughout Europe, and was the crux of the DIA Europe 2018 DIAlogue session The Role of Unmet Need in Regulatory and Pricing Decision Making.

Key Takeaways

• Industry and sponsors who continue investing in medicines R&D for unmet medical needs would benefit from more predictability throughout the lifecycle of these products
• EMA, EUnetHTA, and payer organisations all recognize the challenge to increase the predictability of their decision outcomes, and unmet medical need is one of the elements of the EMA-EUnetHTA work plan for 2018-2020.
• European-level legislation that establishes the EMA’s remit and responsibilities does not fully align with national reimbursement policy in some countries, which forces these decision makers to use different weighing and criteria; better alignment would help regulators more efficiently allocate their limited resources
• Industry experts note that, while the benefit of the first-in-class therapy may not be high, such innovation often paves the way for subsequent products which might be more effective; at the same time, they have expressed concerns about the appropriateness of using quality-adjusted life-year (QALY) to measure whether or how an unmet medical need is addressed
• Demonstrating to the EMA that a medicine meets an unmet need won’t by default convince HTA assessors, payers, or other decision makers later in the value chain.

Different Questions Lead to Different Answers

Industry, HTA, regulatory, and payer organizations employ the concept of unmet medical need to ask and answer different questions. EMA refers to the concept of unmet medical need when choosing eligible medicines for increased development support, faster assessment, or early access programmes.

Industry uses this same concept of unmet medical need to steer their own priorities, as do HTA bodies and payers. However, proving to the EMA that a medicine meets an unmet need won’t by default convince HTA assessors, payers, or other decision makers later in the value chain. While they agree on the value of the concept, how these different stakeholders employ this concept remains unaligned.

Evidence demonstrating the degree of benefit is the key consideration for HTA bodies in their work to quantify a product’s benefit in quality-adjusted life years (QALYs). From the payer perspective, defining a product’s patient population is the most important step. Payers must also consider the product’s impact on their budget; some European countries increase the QALY threshold for products which provide high economic benefit (surplus). Many of these decision makers still consider QALY as the gold standard but also recognize that certain cases may present other ways of assessing a product’s benefit. Too much of a technical or scientific assessment might not fully consider a products’ other beneficial aspects.

Asking regulators to accelerate assessment or provide additional support to development programmes for medicines that patients may not be even access later only waste resources in the medicines approval system due to the opportunity cost for prioritizing another product instead. Even so, the EMA also recognizes that, given the diversity of this concept, they shouldn’t be the body assessing or advising on this concept outside of their own remit, decisions, or processes.

Better Alignment in Process

EMA and EUnetHTA are already progressing with their alignment. Payers and industry must build mutual trust but, due to the fragmentation and complexities of their respective landscapes, common ground has been difficult for them to find. Industry seeks an earlier commitment from payers, with the mutual understanding for adjustment as the evidence package becomes more complete, in order to continue innovating and incentivizing. It is important for all stakeholders, including taxpayers, to work together to find solutions to these issues, and that the patients of today and tomorrow remain at the center of these discussions.