Table 2 Identified key domains to address the challenge of uncertainty
Challenges with Uncertainty | ||||
|---|---|---|---|---|
Key Domains | Early and inclusive collaboration | Agreement on feasibility of RCT and acceptance of uncertainty | Alignment on closing evidence gaps | Capacity gaps |
Description | Collaboration should be enhanced especially at an early stage when designing the clinical development program. Continuous involvement of regulators, HTA bodies, clinicians, patients and industry is critical | Early exploration of feasibility of RCT should be agreed by all relevant stakeholders. If an RCT is considered not feasible alternative options of generation of comparative evidence should be explored and level of acceptable uncertainty should be agreed | Early agreement on comparative evidence gaps and how to generate data to appropriately address those gaps | Ensure sufficient capacities to allow for early and inclusive collaboration e.g., sufficient joint early advice opportunities |
Guiding Questions | How can EU HTA take into account evolving clinical research and development paradigms in oncology to be a driver not a bottleneck for patient access to new medicines while at the same time not reducing methodological rigor? How can stakeholder involvement have meaningful impact? | Is an RCT possible in this context? What other options for generation of comparative data exist? How can additional types of evidence and methods help increase certainty? What level of certainty can be achieved with small sample sizes e.g., in childhood cancer or in ATMPs? | How to approach a gradually evolving evidence body with limited certainty at time of launch? What are suitable and achievable comparative evidence requirements for Real World Data and New Clinical Trial Designs? | What is the expected number of new oncology drug applications / year and the related joint scientific advice capabilities of EMA/ EU HTA? What can be done to increase the number of available joint scientific advice slots? |