In response to the lack of sufficient information on market access processes for cell and gene therapies in the major European markets, the Cell and Gene Therapy Catapult Health Economics and Market Access team has compiled a set of reimbursement roadmaps and accompanying explanatory presentations covering the Big5 EU: France, Germany, Italy, Spain and the UK.
The challenge
Information on European routes to market access for cell and gene therapies tend to be limited. Existing subject matter does not address considerations such as regulatory status, size of target population, requirements for accompanying interventional procedures, challenging service delivery and, most importantly, claims of how long-term benefits impact market access routes, evaluation processes and funding options.
Unlike typical pharmaceuticals, cell and gene therapies fall under various regulatory categories which impact pricing, reimbursement and market access. These include:
- Advanced Therapy Medicinal Product (ATMP)
- ATMP not intended for licensing/hospital exemptions/specials
- Minimally manipulated cell or tissue therapy
- Unlicensed ATMP
Additionally depending on size of target population, funding routes may vary from individual funding requests at local hospital level (e.g. for cell and gene therapies targeting diseases with single digit incidence), to formal product evaluations at national and/or regional level (where larger patient populations are concerned).
Furthermore the vast majority of cell and gene therapies are hospital-only products (often accompanied by one or more interventional procedures) and are costly. Autologous therapies in particular present additional challenges for implementation as they have the potential to disrupt existing treatment algorithms and necessitate relocation of healthcare resources.
Our expertise
Through extensive secondary and primary research with key regulatory and market access stakeholders (at national, regional and local level in France, Germany, Italy, Spain and the UK), we have explored how the following factors can impact market access routes, evaluation processes and funding options for cell and gene therapies at a national, regional and local level:
- the regulatory status and the size of the target population;
- the early access and temporary authorisation schemes available;
- the availability, selection, elimination and prioritisation criteria of HTA methodologies and their impact of therapy adoption;
- types of health economics assessments and their impact on the decision-making of key market access stakeholders; and
- availability of funding mechanisms for expensive innovative treatments and the criteria and processes for obtaining such funding.
Outcome
The individual reimbursement roadmaps can be accessed below:
Roadmap to market access for England and Wales
Roadmap to market access for Spain
Roadmap to market access for Italy