Lightning Health’s recently published research in ATMPs further explores the key themes discussed at the virtual ISPOR Europe 2020

Eleanor Butler, Consultant at Lightning Health attended the virtual ISPOR Europe 2020 and has provided a round up on the meeting.

Financial sustainability and risk sharing

Innovation is progressing faster than health technology assessment (HTA) processes and we are seeing a greater number of ”one-time” therapies with a big impact on public health spending (ATMPs) in addition to more combination therapies. These therapeutic paradigms result in more financial and clinical uncertainty and adaptive reimbursement models with alternative funding/access solutions with real world protocols are required.

Solutions to key (common) issues should be discussed globally in a common space and assume a whole eco-system perspective. Countries should share best practices and learn from each other. Collaboration requires initiative and leadership and should be supported by HTA agencies. Need to incentivise products that provide the greatest offsets on cost to society

Patient and public involvement and readiness for advanced therapies

Patient and public involvement in healthcare is an increasingly important component of the healthcare system. “Patients” (patients, experts and advocates) participate in advice procedures across the medicine’s lifecycle in all disease areas and adequate training to provide them with leadership and communication capacity to foster a transparent, positive decision-making framework is required. Patients have access to unique data that is valuable to industry (a greater proportion of the budget should be spent on patient preference data) and a global standard for generation and use of this patient experience data should be developed collaboratively. Funding of patient advocacy groups (PAG) can be subject to conflict of interest and in order to avoid this, multiple manufacturers/stakeholders should be approached to collaboratively fund the organisations.

Is Europe ready for the next medical frontier?

Novel advanced therapies present unique challenges to healthcare systems globally including high upfront costs and unclear long-term effects. We must find a mechanism to adequately and fairly evaluate these innovative therapies. Many stakeholders believe a new framework may not be required, instead an adaptation of existing frameworks such as the Quality Adjusted Life Year (QALY) with adjusted cost-effectiveness thresholds may be a starting point (advanced therapies have already demonstrated generation of a high number of QALYs).

Despite the high upfront expenditure, the average cost of a gene therapy is less than that of a chronic therapy over a 10-year period. In order to support this transition from a chronic to one-off treatment paradigm, we need novel reimbursement strategies to share risk and mitigate the initial payer uncertainty; conditional reimbursement and annuity payments are the most likely candidates for quick adoption. Other innovative access mechanisms were also discussed such as dedicated funds for ATMPs to allow health systems to invest in advanced therapies and pan-European initiatives to ensure timely and effective access to cross-border healthcare for patients.

Current HTA assessments of advanced therapies commonly criticise the short-term study designs (72% of reviews) and indirect or historical comparisons (49%). To improve market access of advanced therapies, closer cooperation between payers, healthcare providers, industry and policy makers is crucial to enable wider application of innovative payment/financial models (expansion of opportunities for collaboration and early dialogue could be supported by increased European funding). Furthermore, HTA methods need to incorporate more RWE and improved infrastructure to collect and use high-quality RWD is required. Post-launch studies to support innovative funding models are essential for approved advanced therapies and currently Strimvelis, Kymriah, Luxturna, Zynteglo and Zolgensma have registered studies with follow up durations of 15 years.