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ESMO’s Statement on the Release of the Proposal to Revise the EU’s Pharmaceuticals Legislation

The publication of the European Commission’s proposal to revise the EU’s pharmaceuticals legislation is a once in a generation opportunity to improve the availability, accessibility and affordability of medicines in the EU

27 Apr 2023

“ESMO welcomes the main objectives of the revision,” says Jean-Yves Blay, ESMO Director of Public Policy. The objectives include placing the patients at the center, securing the timely delivery of innovative and affordable medicines across the EU, including cancer and rare cancer medicines, while addressing both Unmet Medical Need (UMN) and High Unmet Medical Need (HUMN).

“As cancer cases are set to increase by 24% by 2035, making it the leading cause of death in the EU unless decisive action is taken, ESMO is committed to working with the EU institutions to ensure that this revision is completed in a timely fashion, with tangible legislative measures to deliver improved health outcomes to patients,” says Blay.

ESMO is also pleased to note the proposed measures to revise the Orphan Medicinal Product Regulation: it aims at improving the therapeutic landscape through foreseen scientific guidelines on the category of orphan medicinal products addressing a HUMN. It also foresees to establish a criteria-based definition of an UMN, which reflects scientific and technological developments of precision medicine, to stimulate innovation in the areas of neurodegenerative and rare diseases and paediatric cancers. "We therefore appreciate the inclusion of rare diseases under the definition of a HUMN, which demonstrates the recognition of rare cancers as a major health challenge that needs to be urgently tackled.”

It is pivotal that patients and healthcare professionals will be included in the development of the future scientific guidelines and criteria and setting priorities for research and development. It is also important to have a holistic approach across legislation, including the HTA to promote equitable access, and further clarifications are needed for the methodologies.

The proposed simplification of the European Medicines Agency’s (EMA) structure is a step in the right direction; the greater involvement of patients and health care professionals across the organisation and providing an enhanced scientific and regulatory support if a product addresses an UMN or an orphan product addresses a HUM are two very concrete measures. ESMO also welcomes the proposed measures to tackle medicines shortages, including the adoption of a list of critical medicines that require a coordinated EU-level action and the obligation on market authorisation holders to provide shortage prevention plans, as only a supranational EU solution can mitigate shortages of inexpensive and essential medicines.

“The proposed measure on repurposing of medicinal products, combined with the use of off-label medicines is also to be welcomed, as this can provide significant opportunities for treating cancer and

rare cancers,” notes Blay. However, some issues around health technology assessment (HTA), methodology and access to patients will require further elaboration. Off-label use of old inexpensive medicines is an opportunity for the treatment of cancer patients: we are also pleased to note the proposal that entities not engaged in an economic activity can submit substantive non-clinical or clinical evidence for a new therapeutic indication that is expected to address a UMN.

ESMO also welcomes the proposed measures to increase transparency on the contribution of public funding for research and development of medicinal products whether successful or unsuccessful which have the potential to improve access to affordable medicines.

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