On 26 April 2023 the European Commission published its proposal to revise the EU’s general pharmaceutical legislation, which includes one Regulation and one Directive, with the following objectives:
- putting patients in the centre – equal access and security of supply for patients
- developing the criterion-based definition of an unmet medical need (UMN) and scientific guidelines on the category of ‘orphan medicinal products addressing a high unmet medical need (HUMN)
- introducing structural changes to the European Medicines Agency (EMA), which includes greater representation of patients and health care professionals
- adopting a list of critical medicines that require coordinated EU-level action to prevent medicines shortages
- repurposing off-patent medicines by granting a new therapeutic indication, not previously authorised in the EU, in order to increase the availability of medicines with a significant clinical benefit in comparison with existing therapies,
- ensuring greater transparency of public funding for research and development of medicines.
Following on from the publication of the legislative proposal, ESMO published its statement welcoming the aforementioned objectives and recently submitted a response to the public consultation which includes, amongst other, the following recommendations:
- approval of medicines based on head-to-head randomised controlled trials and, when feasible, between investigational therapies and state of the art standard of care
- patient-centered, hard or validated surrogate trial endpoints for regulatory and reimbursement decisions should be used
- implementation of a rigorous definition of equivalence for biosimilars is needed in order to ensure the highest possible level of patient safety vis a vis these products
- further clarifications are needed on how criteria-based definition of UMN and scientific guidelines on the category of ‘orphan medicinal products addressing HUMN will be quantified/defined to ensure that they reflect the latest scientific developments, considering the lack of access to appropriate therapies and commercial feasibility in developing new therapies for rare cancers
- further clarification is also required with regard to the criteria/methodology and the standardised/scientific base, which also applies to orphan medicinal products addressing HUMN
- the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) should be used as a reference tool to assist in the identification of those criteria which provide ‘no satisfactory/significant benefit’ while defining rare/and orphan conditions
- the definition of rare cancers and orphan medicinal products should be updatable, as common tumours may be fragmented in distinct, moleculary-defined rare clinicobiologic disease entities.
- for UMN the incidence of disease should be <6/100000/year and for HUMN when <1/10e6/yea
- patients and healthcare professionals to be included in the development of future scientific guidelines and criteria, that they have a co-designing and co-deciding role, and are also involved in the setting of priorities for research and development
- the involvement of the rare cancers’ community, including ESMO’s Rare Cancers Working Group – be sought in order to ensure that the appropriate expertise is properly utilised
Early October, the European Parliament published its draft reports on the proposed Regulation and Directive which include additional measures to establish a criteria-based definition of UMN and strengthening the proposed consultation process by obliging the European Medicines Agency to involve patients, medicine developers, healthcare professionals, industries or other relevant stakeholders. The Regulation draft report also proposes to establish a new EU agency called ‘European Medicines Facility’ (EMF) to led R&D projects focusing on health priorities including, amongst others, medicinal products addressing HUMN and UMN which have not been sufficiently addressed by the private sector and where the private R&D pipeline is unlikely to deliver on medicinal products and therapies.
The publication of the aforementioned reports launches an official debate within the EP to adopt its common position to which ESMO recently contributed by sending its suggested amendments and recommendations to the draft reports and continues engaging with the EU institutions to secure that the future EU’s pharmaceutical legislation reflects ESMO’s asks to ensure the availability, accessibility and affordability of medicines for cancer and rare cancer medicines in the EU.