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By Dr Ward Rommel, Chair, Access to Medicines Task Force of Association of European Cancer Leagues (ECL)  –  @CancerLeagues & @WardRommel | #LetsTalkAccess

In Europe, around 2.7 million people are diagnosed with cancer every year – and this number is set to grow.  Many of these cancer patients have unmet medical needs, meaning that there are no targeted or only limited treatment options for them. Fortunately, innovative therapies, such as the advanced therapy medicinal products (ATMPs), offer ground-breaking new opportunities for the treatment of their disease.

Currently, the only feasible way to bring ATMPs to patients is via a commercial route which often brings the unwillingness to invest if a return on investment is not ensured. Therefore, there is a clear role for academia in the development of personalised treatments and therapies to address unmet medical needs of all cancer patients.

However, as pointed out in the recent paper of the Association of European Cancer Leagues (ECL), not-for-profit developers of ATMPs face many challenges:

• Manufacturing of ATMPs is difficult as it is not easy to create a structure that complies fully with good manufacturing practice within a standard hospital environment that has sufficient capacity for phase III trials or clinical practice.
• Collection of evidence on the long-term safety and benefits of ATMPs is not easy as evidence provided by academic research does not always meet the requirements of the authorities responsible for the authorisation or reimbursement of such treatments.
• Acquiring authorisation is complicated as procedures are geared to the resources and capabilities of the industry and are not accessible to academics.

We put forward several policy recommendations to ensure that safe and efficacious ATMPs developed by not-for-profit entities become available to patient groups with high unmet needs. The proposals for a  new directive and a new regulation take steps in the right direction and address some of the challenges outlined above. For example, the legislative proposals foresee a support scheme composed of regulatory, procedural and administrative support, and a reduction, deferral or waiver of fees that not-for-profit entities will benefit from. The scheme should cover the various steps involved in pre-authorisation procedures, such as scientific advice, the submission of the marketing authorisation application, and post-authorisation procedures. In addition, we welcome a proposal to harmonise the application of the Hospital Exemption (HE), a crucial and indispensable tool used for personalised treatments or niche ATMPs for ultra-rare diseases, across the EU. HE allows medical professionals to administer an ATMP without market authorisation under strict conditions – it must be used on a non-routine basis, for a custom-made product for an individual patient. The competent national authorities need to give authorisation and ensure that an ATMP complies with the requirements of good manufacturing practice. As EU Member States currently implement HE differently, this creates inequality among European patients. The proposal in the new directive would allow for more harmonious implementation by setting up a system to collect, report and review data on the use, safety, and efficacy of ATMPs. The European Medicines Agency would be tasked with reporting on the implementation of the HE across the EU to the Commission. However, to create a level playing field across the EU, further steps need to be taken, for example, by providing guidance on the interpretation of the concepts such as “non-routine” and “custom-made”. Currently, the HE is tied to each Member State. However, ATMPs that are safe and effective, should reach every European patient despite the lack of commercial viability beyond national borders. It is not clear how the proposed legislation will make this a reality.

The proposed revision of the pharmaceutical legislation recognises the importance and the role of not-for-profit, including academic, development of innovative therapies. It is to be seen how well the co-legislators in the European Parliament and the Council will develop the Commission’s proposals ensuring that every patient with high unmet medical needs has access to potentially valuable therapies.