OBJECTIVES

Successful adoption of Advanced Therapy Medicinal Products (ATMPs), with potentially curative effects after one treatment cycle, can provide significant clinical and economic benefits to multiple stakeholders. However, limited data at launch, challenges quantifying long-term benefits and high upfront (or total) costs lead to clinical and economic uncertainty for payers. The complexity is highlighted by five European ATMP market withdrawals between 2014- 2019.

This research aims to explore the feasibility of new sustainable funding models to drive patient access for high cost, high value ATMPs from global and market specific perspectives.

METHODS

An advisory meeting was held with EU and US HTA and budget-holding stakeholders and senior industry executives. Participants reviewed existing ATMP financing and reimbursement approaches and explored the challenges with incentivising their development and commercialisation, whilst ensuring sustainable funding and management of payer uncertainty. Feasible application of proposed solutions, depending on product type and market archetype have been explored in further detail with market specific analysis.

RESULTS

Successful models to align stakeholder requirements for ATMP launch and uptake will likely need to utilise a combination of financial and payment models, tailored for market and product characteristics. Advisors recommended individual ATMP manufacturers engage in early dialogue with payers/HTA bodies, to address administrative and implementation requirements and explore models for mitigating financial and clinical uncertainty. Participants also suggested inter-industry collaboration to align on a common framework to address ATMP challenges. Furthermore, industry and payers/HTA bodies could collaborate to develop a hierarchy of global unmet needs and clinical priorities, to inform the prioritisation of development, access and sustainable reimbursement for therapies with high societal value.

CONCLUSIONS

A cross-stakeholder collaboration is required to address uncertainties on the clinical and economic value of ATMPs at launch, whilst ensuring sustainable value-based reimbursement for high value curative therapies to address global unmet needs.