EMA-HTA Parallel Scientific Advice: Challenges and Opportunities: Conducting an EMA-HTA Parallel Scientific Advice for a Development Product between Clinical Phase I and Phase III

Compared to regulatory agencies - focusing on the benefit/risk assessment of a product, typically evaluated in the strictly controlled setting of randomized controlled trials with close attention to internal validity, safety, efficacy and manufacturing - HTA bodies have different remits and, therefore, additional evidence requirements. Criteria for reimbursement decisions vary across countries but can include unmet medical needs, the relative effectiveness and safety of the drug, drug price, budget impact and cost-effectiveness. Designing a study suitable for regulatory approval might not always translate into data suitable for reimbursement decisions, creating an evidence gap between the regulatory and reimbursement submission and posing a hurdle to patient access to new drugs in some countries. Indeed, even when a marketing authorization is obtained, HTABs and decisions on drug pricing and reimbursement often delay access to medicines for patients at a national or even regional level. For the development of new and innovative medicines it is therefore essential that discussion is initiated between industry, EMA as well as reimbursement agencies at an early stage.