I have just attended a panel discussion at Bio Trinity 2019 and thought it was worth summarising the challenges the panellists described as this market evolves and matures. Whilst some of these issues, in part will be addressed by the market maturing and becoming more commercially-orientated, these are real issues that our panellists feel today.
What's New in Cell & Gene Therapy - Bio Trinity 2019
PANEL: Hartmut Tintrup - Lonza, Arthur Stril - Celluctis, Ryan Cawood - Oxford Genetics, Joe Dupere - Rexgenero and Ena Prosser - Fountain Healthcare Partners
- Scaling up manufacturing in particular, for autologous therapies. Up to now, there hasn't been the real world experience, the volumes or incentives to move from an essentially academic, small patient population perspective to a scaled-up, commercially viable approach to manufacturing.
- The overall logistics and supply chain is a major challenge for complex advanced therapeutics, linked to the above point on manufacture. Again, more challenging with autologous therapies, requiring a complex, often personalised, process to ensure appropriate care, stability and delivery of therapy to patients in desperate need of treatment. As the market matures and as more 'off-the-shelf' allogeneic therapies become available then some of these manufacturing and logistical challenges get easier.
- The complexity of point of care logistics, in particular, is challenging, especially with autologous therapies, where cells need to be processed in lower-control environments vs. typical manufacturing facilities. This is only exacerbated when working in the developing world, adding to the challenges of stability and consistency. The panel believes that the way forward here will be Point of Care, Centres of Excellence where manufacturing, processing and delivery can all be controlled within a specialist site analogous to how Nuclear Medicine is handled in specialist centres today.
- Evolving regulatory environment means uncertainty and challenge. Although regulators have been proactive, this still requires active consideration, recognising the differences for example between the approach of the FDA vs. EMA.
- Procurement of raw materials was noted as a key challenge by some panellists, especially considering viral-vectors and access to sufficient materials at a reasonable price.
- Acceptable pricing was noted by all and is not surprising given the challenges outlined above, and their impact on therapies with an inherently high cost of goods. Pricing and Reimbursement is becoming more challenging generally, as health care systems struggle to keep up with novel therapeutics often targeted at rare and orphan diseases but without volumes, without scale and whilst predominantly focused on individualised, autologous therapies, the costs of advanced, cell and gene therapies, will remain high.
- Lack of commercialisation experience was mentioned during the discussion, and personally, I think this point is often overlooked. Experienced biopharmaceutical professionals across multiple functions from medical, market access, regulatory, manufacture, marketing and sales have 'grown up' in a world of small molecules and monoclonal antibodies. Advanced Therapeutics pose a completely different set of challenges, a new business model with multi-dimensional issues that need to be thought through, especially as a company looks to launch and commercialisation.
- Sophisticated patient groups and a more educated, informed patient and/or carer was raised as a positive dynamic, however adding to the challenge for cell and gene therapy companies. Patients recognise that a 'wrong' decision with a cell or gene therapy can impact what subsequent options are available to them and may preclude them from a next-generation treatment.
The many challenges in translation posed by this class of products include aspects covering: manufacturing, non-clinical development plan as relevant to clinical trial, marketing authorization, and reimbursement.