So what are ATMPs exactly and why all the fuss about these therapies and the impact they are having within the market? Inbeeo hosted a free to attend webinar looking into this topic focusing on some specific country challenges within the US and EU, with 60+ attendees contributing to the discussion during the Q&A.
ATMPs or Advanced Therapy Medicinal Products have been classified by the EMA into three main types:
- Gene therapy medicines
- Somatic-cell therapy medicines
- Tissue-engineered medicines
These therapies still have the same regulatory principles applied to them as other types of biotechnology products, such as biologics, to meet the safety, quality and efficacy requirements. The type and amount of the data for these assets to demonstrate these requirements however is highly specific.
ATMPs are of real concern to the payers of today as these therapies fall at the intersection of many of their key concerns, such as;
- Cost effectiveness of these potentially life-extending therapies and how to evolve current cost effectiveness frameworks
- Valuation of curative therapies
- Accelerated Access to Paediatric Treatments
- HTA framework for orphan-designated drugs – as is the case in Germany
- Reimbursement of products approved through an adaptive pathway or through conditional approval
- Access to breakthrough innovations
- New payment models for high cost therapies
Most payer reforms that we read and hear about may not be specifically mentioning ATMPs but as these reforms are being designed, we no doubt believe that payers will have ATMPs at the back of their mind and in their thinking as they progress with reforms over time.
ATMPs present challenges to our current thinking and approach to health technology assessment under usual health economic conditions. Limited clinical data packages, with data coming from single arm, no control and lack of comparator studies – there is limited appetite given to historical controls by HTA bodies.
Long term efficacy is another area where lack of balance between the duration of clinical observation of patients and the expected benefit over decades or over a lifetime creates more headaches for today’s health economists.
Start to couple this with the high initial, and often one-off, front-loaded acquisition costs of ATMPs, we start to see the assumptions that health economists are having to make and trade off. For example – modelling time horizon – how do you find a balance in encapsulating the benefit of the treatment and at the same time remaining credible for carrying forward the same benefit over 20+ years.
These are not just the challenges of ATMPs today – there are many more of these therapies on the way, with around 12% of all clinical studies focussing on ATMP therapies – some 250 assets due to be on the market between 2025 & 2030.
These challenges from a payer’s perspective, along with specific country examples are exactly the topics that we addressed during our recent webinar held on 31st March 2020. You can view the recording of the webinar and the answers we gave to questions raised by delegates by playing the video embedded at the top of this post.
Should you have a question about ATMPs or wish to explore the topic further, or would like our expert advice on how we can support you with your own ATMP asset, then please drop us a line at firstname.lastname@example.org