Overcoming the barriers to cell and gene therapies

By Annie Hubert, Senior Director, European Public Policy at Alliance for Regenerative Medicine

ATMPs are varied and complex, ranging from potential one-off curative treatments to treatments tailored to a specific individual. As such, they differ significantly from ‘traditional’ medicinal treatments, something which has been recognised by regulators in Europe: the European Parliament introduced ATMPs as a new class in 2007 and that same year saw the establishment of the European Medicines Agency’s Committee for Advanced Therapies (CAT) to address the specific demands of this growing class of medicines.

Whilst regulatory frameworks have been implemented to enable the approval of ATMPs, payors and health technology assessment bodies haven’t yet been able to adapt their processes to adequately capture the full benefits of ATMPs. As a result, there are many barriers hindering ATMPs from reaching patients in a timely manner – patients whom, in many cases, have few or no alternative treatment options.

One key question is this – how do you measure the value of a cell or gene therapy treatment? ATMP treatments are at the forefront of scientific innovation and aim to address the underlying cause of diseases in a profound and durable manner. It is necessary to factor in the long-term value to patients.  ATMPs offer the possibility of treatment in just one or a few administrations, which do require high upfront costs compared to traditional treatments. Yet these potentially provide benefits for a patient’s entire lifetime.

The issue of value is particularly evident when applying existing Health Technology Assessment (HTA) frameworks to ATMPs. At the Alliance for Regenerative Medicine, we are convinced that such frameworks need adapting. HTA bodies need to evolve to evaluate new kinds of evidence, beyond traditional randomised controlled trials, and adapt their economic models to extrapolate longer-term benefit based on the data available at the time of approval.

It seems logical that innovative treatments require new payment models. Conditional reimbursement schemes, for instance, would be one way to address uncertainty on the durative effect of cell and gene therapies, as clarity on this point may be unavailable when an ATMP is first approved. Other payment models, such as pay-for-performance, annuity payments, and the creation of specialist funds could offer other further flexibility for the sector.

Collaboration is also key. Pan-European initiatives would enable a more streamlined process to providing ATMPs to patients. Firstly, the development of a European-wide Real-World Evidence infrastructure would help support the generation of high-quality, more robust, long-term evidence. Secondly, there is a need to invest in the creation of more opportunities for early dialogues, to offer developers early insight on HTA and payors’ needs and reduce uncertainties on product development at an early stage. Finally, whilst there is legislation on cross-border healthcare in Europe, much more could be done to help European nations coordinate and improve funding for patients who need to travel to a different country or region to get their ATMP treatment in a center of excellence.

We at the Alliance for Regenerative Medicine are working closely with developers, industry bodies and regulators to try and address the barriers to ATMP adoption. New models and refreshed frameworks are urgently needed if we are to broaden the access of these transformational therapies to the patients who need them.

Register for the UK Bioscience Forum 2019 to hear more about this topic during the panel discussion ‘Overcoming barriers to cell and gene therapies’. We will be joined by Annie Hubert – ARM, Dr Rubina Ahmed - Fight for Sight, Dr Claire Roddie - University College Hospital London, Nina Pinwill - NHS England, and Christopher Vann – Autolus.