Guest Blog|Medicine revolution – getting ATMPs across the line
Guest blog by Fieldfisher
Cliodhna McDonough, Director
James Gallagher, Senior associate
Emily Lockey, Trainee solicitor
Advanced therapy medicinal products (ATMPs) is the buzzword that has been around the industry for a while now, with hopes to revolutionise the way medical treatments are done, hopefully moving away from 'traditional disease management' to cures. To overview what ATMPs are, they are a group of medicines for human use that are based on genes, tissues or cells which hold promise as treatments for a variety of previously untreatable and high-burden diseases. There are broadly three types; somatic cell therapy, gene therapy and tissue engineered. In addition, some ATMPs may be referred to as combined ATMPs.
So, if ATMPs are the future of medicine, why the delay? Although, there is much promise for ATMP treatment, there are tremendous challenges getting the products onto the market, with success being dependent on the use of science and risk-based approaches to their development and manufacture.
The number of new ATMP clinical trials has increased significantly over a 4-year period on a global scale, as shown in recent research carried out by the Alliance for Regenerative Medicine, particularly in North America and Asia, this trend was however not reflected in Europe. Why the slowdown in Europe though? The same research speculated that fragmentation of regulatory/ethical guidance and a lack of harmonisation on various other factors (e.g. donor testing requirements, patient information consent forms, contracting agreements, etc.) across European countries may also make for a less attractive environment for clinical trials. Furthermore, a possible cause of the relatively low number of gene therapy clinical trials in Europe compared to North America was also traced back to the classification of some of these therapies as GMOs, requiring specific approval by different national authorities, a step that adds complexity to the clinical trial authorisation process and often extends the time required for approval.
From a commercial point of view, ATMPs present a different scenario to that of pharmaceutical medicines in that there is not a classic supply and demand model largely because ATMP therapies are more likely have a patient pull to fulfil an unmet medical need. In addition, the clinical development of ATMPs does not typically follow conventional clinical trial phases unlike traditional medicine. It is often the case with rare disease indications, that ATMP clinical programs are compressed into one or two clinical studies, followed by conditional approval with post-marketing commitments. To this end, ATMPs often utilise Early Access Programs, which allow for supply to patients prior to marketing approval.
Furthermore, another barrier to the commercial viability for ATMPs is the supply chain, which contributes significantly to the overall cost of goods and is limited by infrastructure, temperature requirements and, of course, the time frame for transportation taking into consideration cell viability. Poor co-ordination of supply and logistic conditions have the potential to negatively affect the quality of ATMPs.
In brief we have outlined two of the major obstacles from a commercial and development perspective, however we cannot forget about restraints over compliance and thus the need for regulatory requirements and industry practices to catch up with ATMP development to allow a smoother process to market. Despite delays the future of medicine will become a reality.