Can Regulators and HTA Bodies Create Synergies for Patient Access?

Chris M. Slawecki
DIA Senior Digital Copyeditor

ealthcare systems in economies of every size continue to struggle with finding ways to ensure access to safe and efficacious healthcare products for their patients. These struggles have had one positive impact: Stakeholders throughout the product development lifecycle now agree that discussing access issues after a product has received regulatory and marketing approval is too inefficient, too unpredictable, and most of all, too late.

Key Takeaways

• Access decisions must be based on evidence, not emotion
  • Simultaneously and mutually define evidence required for access decisions with evidence required for regulatory and marketing decisions
  • This initiates access conversations among all stakeholders and enables prospective planning of evidence required downstream
  • Long-term collection of data, while complex, is essential for conditional approval and other regulatory accelerated access pathways
• Collaborations beyond individual product pathways must continue
  • More clearly define treatment guidelines and patient desired outcomes
  • Design and implement registers (registries) for data collection and sharing
  • Horizon scanning to prospectively identify and plan for financial, clinical, or organizational impacts on healthcare systems
• Companies developing advanced therapy medical products have been highly receptive to and encouraged by the EMA’s scientific advice and parallel advice tools.

Why this is important: While scientifically and clinically promising, the emergence of personalized medicine and immune-oncology, combined with the impact of more people living with more chronic illness for longer lifespans, are straining the capacity and resources of healthcare systems in nearly every nation.

“The overall goal of the track was to really advance the conversation that we have had now, for a number of years, between all stakeholders about how we can ensure together timely access for patients,” explained Jordi Llinares Garcia, MD, Head of Scientific & Regulatory Management, European Medicines Agency.

European Patient Registries

Patient registries have proven to be an important tool in all phases of clinical trials throughout Europe, from patient input shaping clinical trial feasibility/design studies to pharmacoepidemiology and pharmacoeconomic studies designed to determine if/how a product’s efficacy in its clinical trial is translating into real-world effectiveness. Register data can also be integrated into health technology assessments to ensure novel medicines reach the patients for whom they were designed and developed.

Launch of “Parallel Consultation”

“There was also agreement across different sessions how scientific advice can really be the tool that we offer to really have good discussions on planning of data generation, and in particular for advanced products,” Dr. Garcia explained.

Experience has already shown that parallel scientific advice can help align the evidence required by regulatory and HTA bodies; close collaboration between EMA and EUnetHTA continuously facilitates such dialogue. Although regulatory and HTA bodies must answer different questions, and have different requirements in terms of evidence, aligning these evidence generation plans (including patient populations, comparators, study design, endpoints, and post-licensing requirements) allows for synergy (or at the very least reduces duplication and other inefficiencies) between the two.

While stakeholders have focused so far on defining evidence needed for market entry, they also agree that more collaborative engagement is needed to determine post-licensing evidence requirements.

PRIME Goal and Scope

One of the EMA’s most visible tools to facilitate early patient access to medicines is the PRIME scheme, built upon existing regulatory frameworks and tools such as accelerated assessment and scientific advice. Now entering its third year, this voluntary scheme is based on early and enhanced regulatory-industry dialogue to optimize development plans and speed up evaluation so these medicines can reach patients earlier.


PRIME improves clinical trial designs so that patients participate in trials that generate data suitable for evaluating a marketing application, making the best use of precious patient resources; and optimizes the generation of robust data from these trials to determine a product’s benefit and risk, enabling accelerated assessment of these applications to patients’ benefit.

This article summarizes key takeaways from sessions in “Topic A: Can Regulators and HTA Bodies Create Synergies for Patient Access?”