Nermina Ferizovic | Health Economics Expert

I recently came across an exciting commentary on the economic sustainability of advanced therapy medicinal products (ATMPs). The authors describe four ‘provisions’ which contribute to the economic sustainability of these transformative therapies: 

(1) substantial and durable therapeutic benefit for most treated patients that surpasses that of any other treatment available, which establishes the patient and societal drug value in terms of sparing suffering, improving quality and duration of life, and limiting healthcare and caregiving costs; (2) fair return on investment (ROI) on the developers’ side; (3) financial affordability for healthcare systems (that is, payers); and (4) timely access to all patients who might benefit from such therapies, at least within certain jurisdictions.

The focus of the commentary is that of provision number (2). The discussions are insightful and thorough, and I really enjoyed reading it. The authors propose excellent solutions to concerns from developers: 

• Reduce all post marketing fees (for example, regulatory and pharmacovigilance) for GCTs for (ultra-)rare diseases and/or calculation of those fees as a percentage of the revenues generated by the product.
• Facilitate cross-border movement of patients for access to once-in-a-lifetime treatments for severe ultra-rare conditions, especially within the European Union but also across different jurisdictions.
• Introduce measures that can generate revenues not linked to the number of patients treated (such as the US priority review vouchers) — however, such incentives should be linked with a commitment to keep the product on the market for at least some years after approval.
• Implement pricing and reimbursement models based on a fixed subscription fee plus a per-patient price that reflects only the direct costs generated by the treatment of that specific patient. The subscription fee should allow the developer to cover at least the bulk of fixed costs incurred for product maintenance.

There are a couple of points that I would like to discuss further. For me, one of the key solutions missing relates to value assessment of ATMPs. This is a topic close to my heart and I was pleased to read that the authors refer to the ‘tremendous patient and societal values…’ that ATMPs can bring. However, there is evidence that current health technology assessment methods do not allow for the full assessment of value of ATMPs. I would propose the addition of ‘implement broader value elements to the enable the assessment of the full value of ATMPs’ or perhaps even go as far as to say ‘implement alternative value assessment frameworks’. Further, I would add ‘society’ to the list of perspectives which should be considered when determining economic sustainability of ATMPs, but this could be context dependent (i.e., private or public healthcare systems). Finally, an assessment of (potential) value for money, with the value component incorporating all relevant value attributes, should be undertaken by developers as early as possible to inform investment decisions.