Written by Gianluca Quaglio and Nada Alkhayat,
Advanced therapy medicinal products constitute a heterogeneous group of biopharmaceuticals encompassing gene therapy and somatic cell therapy medicinal products, as well as tissue-engineered and combined products (tissues or cells associated with a device). Nanomedicine comprises applications of nanotechnology for the diagnosis and treatment of biological systems. Research into the delivery of pharmaceutical and diagnostic agents is at the forefront of projects in nanomedicine.
The workshop on ‘Therapies for the future’, held at the European Parliament on 11 October 2017, reported on the status of nanomaterial-based and advanced therapy medicinal products (ATMPs), as well as on possible options for the future development of these new therapies in Europe, supporting patient access and transparent information across Europe. Paul Rübig, MEP and First STOA Vice-Chair, began the session by underlining that nanomedicine and ATMPs have the potential to transform medicinal practice, reshaping the treatment of a wide range of clinical conditions.
Vytenis Andriukaitis, European Commissioner for Health and Food Safety, in his keynote speech, stressed that, in order to promote progress, the EU needs to do more together. He also argued that it is the EU’s duty and responsibility to guarantee that the regulatory frameworks facilitate the emergence of new therapies and better diagnostic tools, while also ensuring patient safety.
ATMPs: Need for ‘innovation in the process as much as in the therapy’
Beatrice Lorenzin, the Italian Minister of Health, joined the event via video. Lorenzin noted that many considerations come into play when assessing the value of new therapies, such as the durability of the treatment, the benefits for the patients and the overall cost-effectiveness of the medicines themselves.
In the EU, ATMPs are governed by Regulation 1394/2007 (ATMP Regulation). The cornerstone of the regulation is that marketing authorisation must be obtained prior to the marketing of ATMPs. The regulation has certainly protected patients from unsound treatments, however, many experts recognised shortcomings arising from the legislation and, hence, suggested actions to translate scientific progress into medicinal products available to patients. Guido Rasi, Executive Director of the European Medicines Agency (EMA), underlined that, if ATMPs are to fulfil their promise of providing innovative treatments for patients, then regulators must nurture a regulatory environment that encourages innovation, safeguards public health and, ultimately, facilitates timely patient access to new therapies.
Andrea Chiesi, Vice-President of the European BioPharmaceutical Enterprises (EBE), added that, since the entry into force of the ATMP Regulation, only eight ATMPs have been authorised. Indeed, despite the unified ATMP framework in Europe, variations in national legislations relate to such crucial issues as, for instance, clinical trials, good manufacturing practices, market access and ethical oversight. This is detrimental for European patients, as well as posing a major challenge for the future of the ATMP sector in Europe. Europe, Chiesi concluded, needs to find a path that ensures a more consistent and transparent approach in this field.
Building on the points made by the previous speakers, Yan Le Cam, Chief Executive Officer of EURORDIS, which represents rare disease patients in Europe, suggested four major actions for innovation in the regulatory process and the therapy development:
- a new R&D model, with, among other things, more flexible clinical trials, more innovative statistical methods, a more patient-relevant approach and use of biomarkers;
- early dialogue with those paying (in principle national and regional health services), early approval and increased European cooperation on value assessment;
- the need for a new healthcare delivery system, with more specialty medicines in specialised centres of expertise, associated with data collection in pre- and post-market authorisation phases;
- the portfolios of companies could be enlarged, with more products per company, but at lower prices.
Le Cam appealed for a better consideration, at European level, of inter-relations between the value at market authorisation time, the additional evidence generated in the following years and the volume of patients treated.
With the Horizon 2020 research programme, the European Commission made major efforts to support research and innovation aiming at bringing ATMPs into the clinic and to the market. At present, 23 projects have been or are being funded through an EU contribution, noted Bernard Mulligan, Deputy Head of Unit for Innovative tools, technologies and concepts in health research at the European Commission Directorate-General for Research & Innovation.
Nanotechnology: regulatory demands, clinical application and market opportunities
Nanotechnology is considered a key technology for the implementation of personalised medicine. It offers the means to adopt a more patient-centred approach, which becomes even more important for diseases with a poor prognosis. Susanne Bremer-Hoffmann, from the JRC‘s Directorate for Health, Consumers and Reference Materials, underlined that the translation of nanomedicines from the laboratory into clinical application requires the anticipation of regulatory demands, which must be satisfied to enable a smooth transition to the market.
A major limitation of today’s medicine is the lack of precision in targeting patients, organs and diseased cells. For the precision medicine of tomorrow, the currently evolving paradigms of receptor-specific targeting of nanomaterial-based drug carriers, and a predictive understanding of individual patient responses is needed. Professor Patrick Hunziker, President of the International Society for Nanomedicine, discussed the major obstacles that hinder the translation of new ideas through basic research to clinical and industrial applications.
Research in nanomedicine is extremely time-consuming and any novel technology needs up to 30–35 years to reach the market. Beat Loeffler, Chief Executive Officer of the European Foundation for Clinical Nanomedicine (CLINAM), discussed the market opportunities, restraints and drivers for nanomedicine, and proposals for an acceleration of the nanomedicine market in Europe.
Advancing these advanced therapies
There was common agreement that the EU is committed to supporting the development of advanced therapies. A crucial aspect will be to ensure that the regulatory framework supports and does not hinder development of these therapies. Moreover, an improved regulatory framework will also contribute to promoting innovation, investment and the competitiveness of the EU biotechnology sector, whilst striving to ensure patient access. The implementation of the actions suggested by the speakers will hopefully increase the treatment opportunities for patients in the future.