Belgium and the Netherlands lead push for need-driven research priorities involving patients [Advocacy Lab Content]

Belgium and the Netherlands are pushing for a need-driven research model prioritising society’s requirements, with an on emphasis addressing unmet medical needs. Belgium is setting the stage for future presidencies, with the Netherlands providing research insights.

Currently, private R&D investments are guided by expected returns, with regulatory incentives being supply-driven. The EU now has an opportunity to direct innovation towards unmet medical needs.

In April, the Federal Agency for Medicines and Health Products (FAMHP), the National Institute for Health and Disability Insurance (NIHDI), and the Belgian Health Care Knowledge Centre (KCE) convened a high-level conference on health-related needs driving healthcare policy and innovation.

This conference, held during Belgium’s EU Council Presidency, provided a platform to chart a collaborative path forward.

The Netherlands presented a study identifying 33 conditions with the highest disease burden, highlighting gaps in drug development.

During the conference Pedro Facon, CEO of NIHDI, stated, “Even small changes towards a need-driven approach will provide key results for the healthcare system. One percent of the budget better spent can make a huge difference for many patients.”

Ann Van den Bruel, General Director of KCE, emphasised in a press release that scientific evidence on unmet patient and societal needs is crucial for innovation and policy. 

Introducing the NEED Method

The KCE, in collaboration with Sciensano, introduced the NEED method at the conference, a systematic approach to identify and prioritise unmet health-related needs.

“The ultimate objective is to set up a database with scientific evidence on the unmet health-related needs in different diseases for use by different types of stakeholders, such as investors in R&D, regulators, pricing and reimbursement agencies, and policymakers,” Irina Cleemput, NEED project lead told Euractiv.

“We also tested the applicability of the NEED framework to rare diseases and performed in-depth case studies to provide a proof-of-concept.”

Cleemput emphasised that it is important to consider different types of interventions (not only pharmaceuticals) as possible solutions to unmet health-related needs in the movement towards more needs-driven innovation and policy, adding: “we stress the importance of looking at unmet patient needs as well as unmet societal needs.”

The investigation of the proposed revision of the pharma legislation was not part of the NEED project, Cleemput told Euractiv.

Concerns

RaDiOrg expressed reservations about the NEED project’s impact on rare diseases, noting that it aims to analyse 100 diseases in five years, insufficient given the 6,100+ known rare diseases. One of the concerns is the methodology not reflecting patient-level needs.

“It’s a very artificial methodology that has the advantage of being able to come up with ‘clean data’ but the disadvantage of not reflecting needs at patient level. Because in real life, diseases do not have needs, patients have needs. And the patients most in need are often not those with the textbook manifestation of a disease.”

Revision of pharmaceutical legislation

When the Commission published its pharma reform proposal in 2023, EURORDIS welcomed the emphasis on focusing research where it is most needed and modulating incentives to encourage essential research.

“The split between these two categories was certainly not the best way to ensure this,” said Eva Schoeters of RaDiOrg.

Schoeters advocated for early structured dialogue in a multi-stakeholder format, including patient representatives, clinicians, regulators, HTA experts, and payers, to address unmet needs effectively.

“Unfortunately, during our advocacy activities with the European Parliament, we understood that most MEPs wanted to keep this bilateral division. Therefore, our request is to ensure that the implementation of such legislation is feasible, that the EMA has sufficient freedom in drafting the guidelines on UMN, and that patient representatives are always involved,” said Schoeters.

The Working Group has conducted a preliminary reading of the incentives chapter, gathering positions and concerns from member states, with the goal of advancing negotiations and preparing for future presidencies through a potential options paper.

Detailed position of the Netherlands

The Netherlands is developing a detailed position on the definition of ‘unmet medical need’ in the revision of the pharmaceutical legislation, with a focus on ensuring flexibility in criteria application across legislative and non-legislative incentives.

Their approach stems from several considerations. Firstly, the Commission’s proposal entails a uniform definition serving as a prerequisite for accessing various incentives like PRIME, accelerated assessment, conditional marketing authorisation, and additional data protection.

These incentives are granted at distinct stages of medicinal product development, each characterised by varying levels of certainty regarding the product’s capacity to address the unmet need.

In addition, a standardised definition risks constraining future innovations.

“The Netherlands is therefore looking into solutions leading to a clear, high-level definition of unmet medical needs with different sets of criteria tailored towards the development stage of the product and the individual incentives,” a spokesperson for outgoing Medical Care Minister, Pia Dijkstra, told Euractiv.

[By Nicole Verbeeck, Christoph Schwaiger, Edited by Vasiliki Angouridi, Brian Maguire | Euractiv’s Advocacy Lab]

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