Improving the availability, accessibility and affordability of innovative medicines for patients with cancer
In April 2023, the European Commission (EC) published its proposals, the first major overhaul to the EU’s medicines regulations in 20 years, to revise the following EU pharmaceuticals legislation:
- Regulation 726/2004 - authorisation and supervision of medicinal products for human use and creation of a European Medicines Agency (EMA);
- Regulation 141/2000 on Orphan Medicinal Products (OMPs) laying down criteria for designating certain medicinal products as orphan drugs;
- Regulation 1901/2006 on medicinal products for paediatric use;
- Regulation 1394/2007 on Advanced Therapy Medicinal Products (ATMPs);
- Regulation 536/2014 on clinical trials on medicinal products for human use;
- Directive 2001/83 on the Union code relating to medicinal products for human use.
The EC’s legislative proposal has an overarching goal of so-called ‘3 As’ - Affordability, Accessibility, Availability - and includes, amongst others, the following objectives:
- Putting patients at the centre - equal access and security of supply for patients across the EU;
- Driving innovation for the pharmaceutical industry through a number of incentives related to market exclusivity;
- Developing the criterion-based definition of unmet medical need (UMN) and scientific guidelines on the category of ‘orphan medicinal products’ addressing high unmet medical need (HUMN);
- Introducing structural changes to the European Medicines Agency (EMA), which includes greater representation of patients and healthcare professionals;
- Adopting a list of critical medicines that require coordinated EU-level action to prevent medicines shortages;
- Repurposing off-patent medicines;
- Greater transparency in public funding for research and development of medicines.
ESMO published its statement on the EC’s proposal welcoming the general objectives of the proposal and highlighting the need for the involvement of patients and healthcare professionals in the development of definitions of UMN and HUMN.
ESMO has developed its official position on the legislative proposal, which served as its response to a public consultation on the EC’s proposal, submitted in November 2023, and was shared with the European Parliament (EP) and the Council.
In April 2024 the EP adopted in plenary its reports on the Directive and on the Regulation. Ahead of the plenary vote, ESMO wrote to the Political Groups Coordinators on the EP’s Committee on the Environment, Public Health and Food Safety (ENVI) to welcome/comment on the following EP’s proposals included in the reports:
- ESMO welcomes the EP’s proposal introducing stronger wording to Article 162 of the draft Regulation, which reflects ESMO’s recommendation, that the European Medicines Agency (EMA) shall establish a consultation process with relevant stakeholders, including healthcare professionals and patients, on general issues of a scientific or technical nature related to the tasks of the EMA, in particular guidelines on UMN and the design of clinical trials.
- ESMO reiterated its support for plans to establish a criteria-based definition of UMN and scientific guidelines on the category of orphan medicinal products addressing HUMN and advised that further clarification will be required to explain how these criteria and scientific guidelines will be quantified/defined to ensure that they reflect the latest scientific developments.
- However, ESMO expressed its disappointment that the EP’s initial amendments of the definition of UMN, notably referring to quality of life, have not been included in the final version adopted by the ENVI Committee on 19 March 2024.
- Noting ESMO’s recommendation, previously communicated to the EC and the EP to establish a European network for adult rare cancers, ESMO also welcomes the EP’s proposal that the EC establish a European framework for rare diseases to better meet the unmet needs of people living with rare diseases, and their carers. Such a network - patient representatives, academics, medicines developers and investigators – will act as an invaluable advisory mechanism on specific rare cancer types and, ultimately, result in the improved treatment of patients with rare cancers.
The Council is currently developing its official position on the EC’s legislative proposal and, when finalised, Trilogue negotiations will begin with the EC and EP. Due to the EU elections (6-9 June 2024) the legislative process is expected to be significantly delayed, with the final text to be possibly adopted in 2026/2027.
Given that Europe’s cancer burden is expected to grow from over 4.4 million new cancer cases in 2022 to 5.32 million in 2040, ESMO is committed to working with the EU institutions to ensure that this revision is completed promptly and results in improved health outcomes to patients through the timely delivery of innovative cancer and rare cancer medicines.