EVENT RECAP – Life Science Lectures: Episode Two “Defining (High) Unmet Medical Need (HUMN)

On 7 July, EUCOPE – the European Confederation of Pharmaceutical Entrepreneurs – hosted the second episode of its Life Science Lectures webinar series.

The focus of the webinar, titled ‘’Defining (high) unmet medical need: will it steer innovation towards filling existing gaps?’’, was to dive deeper into the concepts of unmet medical need (UMN) and high unmet medical need (HUMN) outlined in the EU Pharmaceutical Package, with the intention to initiate a discussion on whether these concepts will steer innovation towards underserved areas and bridge existing gaps.

Over the past two decades, the European Union (EU) pharmaceutical legislation has played a crucial role in ensuring the safety, efficacy, and accessibility of innovative medicinal products. The evolving healthcare landscape requires the EU to keep pace with scientific research and medical knowledge advancements. The review of the EU pharmaceutical legislation is therefore crucial to promote innovation in the EU. While the revision holds great promise, it is vital to recognise that some diseases present unique and complex challenges. For instance, around 95% of rare diseases currently lack any treatment option. The proposal to introduce the concept of (High) Unmet Medical Need aims to address this issue, by linking (H)UMN to the incentive framework.

The discussion delved into the potential impact of (H)UMN definition on the development and accessibility of innovative therapies. MEP Tomislav Sokol, representing the European People’s Party (EPP), expressed his support for the modulation of incentives. Still, he cautioned against reducing the baseline period of incentives, emphasising the need for certainty and business clarity.

“The modulation of incentives is a good idea in principle, but it does not provide enough incentives for very innovative therapies. I don’t know if the reduction of the baseline is the ideal approach. More certainty, clarity and predictability of incentives are needed. It’s also important to get a clear definition of HUMN in a way that streamlines towards what it is needed. On the procedural part, we can be more ambitious and further simplify procedures and offer more tailored regulatory processes for needed therapies”.

The Patient Perspective

While Simone Boselli, Public Affairs Director at EURORDIS, also welcomes the modulation approach to drive investment towards underserve areas, he warns about the European Commission’s proposal. “We don’t have clarity on the definition of HUMN yet. Concepts such as ‘meaningful reduction of morbidity or mortality’ or ‘exceptional therapeutic advancement’ are unclear’’. Boselli also questioned the effectiveness of providing only an additional year of OME for addressing HUMN and advocated for stronger investments, infrastructure, and innovation support to fill the existing gaps in rare diseases. He also highlighted the importance of a comprehensive ecosystem involving patients.

“The consultation of patients in the definition of the HUMN is not strong or enough. We need to maintain a good level of inclusion of patients and healthcare professionals on how HUMN should be defined”.

Similarly, Claudia Louati, Head of Policy at the European Patients Forum (EPF), emphasized the crucial role of patient involvement in defining UMN and criticized the restrictive nature of the current definition.

“Unmet medical need is not always a yes-or-no question, and a definition must include criteria that matters to patients. The patients’ perspective is missing from the current definition, which is concerning”.

Louati called for a holistic approach that considers chronic diseases and the adequacy of existing treatments. She stressed the importance of patient perspectives in shaping UMN definition and decision-making processes.

The Industry Perspective

To date, a vast percentage of rare diseases remain without treatment options. The Pharmaceutical Package highlights the need to target those underserved areas where research is mostly needed and where investments are riskier. Alexander Natz, Secretary-General of EUCOPE, recognises the need for a modulation approach to steer innovation. However, he expressed concerns over the definition of (H)UMN.

“From the industry perspective, it is a very strict definition. We need predictability in the system, and so, ‘meaningful reduction of mortality or morbidity’ is not always easy to bring to the table at the early stage of a lifecycle of medicine when it is authorised by the European Medicines Agency (EMA). It may also be difficult to negotiate prices if you don’t have a ‘branding’ of UMN or HUMN from the EMA perspective”.

While the Commission proposed (H)UMN concept aims to improve the development of new therapies for very small patient populations that lack effective treatment options, it may present challenges for smaller companies and overlook other patients that would benefit from innovative therapies. On this topic, Diego Ardigò, Head of Research & Development of Global Rare Diseases at Chiesi, emphasized the distinction between “need” and “therapeutic advancement” within the (H)UMN definition, and he calls for a different system of incentives to support knowledge generation in ultra-rare diseases and more complex conditions.

“Until we have incentives that support knowledge of rare diseases where clinical research and medical knowledge is lacking, we will not steer innovation. There is still UMN where treatment options are available”. He also warned that downsizing incentives could hinder market development and lead to an innovation gap between the EU and the US. ‘’At the end, if you are sure to get that (H)UMN ‘label’, you probably pursue that, but if you think that this may not be the case, the risk is that even innovative companies will drive away from the EU”.

EUCOPE acknowledges the need for a modulated approach to support the development of rare disease therapies in underserved areas. Achieving this goal requires a predictable framework that encompasses patients’ perspectives, fosters innovation, and stimulates investment in R&D in the EU.

EUCOPE’s Life Science Lectures webinar series will continue to explore various aspects of the EU Pharmaceutical Package and its impact on access to innovative therapies and regulatory processes.

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Don’t miss out on the third episode of the Life Science Lectures on “EU HTA: How will the Pharmaceutical Package impact value assessment of therapies?”’ scheduled for 7 September 2023.

Save the date and register now to secure your spot. Stay tuned for updates and prepare for an enriching session with valuable insights and engaging discussions.