Enhancing Development Strategies Through Early Scientific Advice from HTA Agencies-Experiences, Expectations and Best...

Area of Science:

• Health Technology Assessment (HTA)
• Pharmaceutical Policy
• Drug Development

Background:

• Early health technology assessment (HTA) advice aids pharmaceutical companies in drug development by identifying data gaps and refining study designs.
• Understanding HTA agencies' evidentiary requirements is crucial for successful drug development pathways.

Purpose of the Study:

• To evaluate the landscape, benefits, and challenges of early HTA advice.
• Focus on European agencies and global practices during the EU HTA Regulation transition.

Main Methods:

• A perception survey of 12 pharmaceutical companies on engagement with HTA agencies.
• A forum with 22 company representatives discussing best practices, Joint Scientific Consultations (JSCs), real-world evidence (RWE), and patient-reported outcomes (PROs).

Main Results:

• Active engagement noted with NICE, G-BA, and EUnetHTA Joint Actions.
• NICE advice valued for cost-effectiveness; Brexit impacted European collaboration.
• JSCs under EU HTA Regulation are critical but face resource and availability challenges.
• Companies prioritize comparator choices, outcomes, and RWE, facing internal resource barriers.
• Limited uptake of advice services outside Europe highlights potential for lifecycle approaches.

Conclusions:

• Early HTA advice is essential for pharmaceutical development.
• Enhancing JSC capacity, stakeholder engagement, and feedback mechanisms are key.
• Strengthening alignment between companies and HTA agencies fosters evidence-based decisions and improves health outcomes.