Challenging opening comments by Greg Bonfiglio to initiate this discussion session with a bang. A series of complications have hit the industry in recent days: failure of the Athersys phase two trial, federal reimbursement for wound healing Apligraf (Organogenesis) cut. The question posed is, ‘where is the industry now and are these events predicative of the future?’
Alain Vertes, Managing director at NxR Biotechnologies, likens the current industry state to the beginning of monoclonals, and reaffirms that there will be bumps along the road. Investment is healthy (e.g. the recent £55M Cell Therapy Catapult facility grant), and still being inputted into diverse areas. Additionally, there are also positives to be taken from the Athersys clinical trial. Timothy Allsopp, head of external research at Neusentis, Pfizer Ltd, reminds us that safety was demonstrated in the studies, and in addition we are still waiting for the 16 week, repeated dose data.
This leads the discussion on to big pharma. The formula for all clinical trials are based off big pharma design, with their associated financial backing. Cell therapies cannot afford these costs, and consequently this logic and regulation needs to evolve.
The next question posed by Greg: ‘Following on from Michael May’s (CEO at the CCRM)discussion of quality-adjusted life year-based reimbursement overvaluation of acute myocardial infarction’s commercial potential, could this drive us towards a two tier healthcare system?’ Similar to the concierge medical systems now in California and New York, this would result in split access to technology and therapies dependent of patients’ spending capacity.
Michael responded by highlighting the current trend (in pharma as well as cell therapies) towards personalised medicines. ‘The scenario will work if you have diagnostics that identify patients effectively, that allow patient specific healthcare.’ Arnold expanded upon this point: ‘to identify both non-responders and patients that will react positively is a huge challenge, but there is big market potential.’ Most drugs from big pharma have around a 70% drug response, so the economics will be driven by capacity for healthcare providers to identify non responders. A further market for this information would be to identify inclusion criteria for clinical trials, which coincides well with Susan Solomon’s research automating iPS cell derivation and differentiation at the New York Stem Cell Foundation.
In summary, several key issues where touched upon: the current regenerative market has faced fluctuations, but investment is still present and following a similar trend to mAbs. Arnold Caplan noted that the industry should be judge success based on clinical data, not market fluctuations. Complication with reimbursement are tied in to economies of scale and cost of goods. Scaling therapies effectively will be discussed in further detail in Kim Warren and Alexander Vos‘s talks.
Ben Weil for Total BioPharma at the World Stem Cell and Regenerative Medicine Congress
Ben is an EngD research student at University College London. His work, in collaboration with SpheriTech Ltd., is in designing a novel purification step to isolate rare cell types derived through human pluripotent cells.