Regulatory Win to Commercial Disaster: Mind the Evidence Gap

(3 Minute Read - All sources cited below)

The Growing Evidence Gap: A Market Access Challenge

"We must base our recommendations on evidence, not on hope."

This declaration from Birgitte Klindt Poulsen of the Danish Medicines Council at ISPOR Barcelona captures a fundamental challenge facing breakthrough innovations today: the growing divide between regulatory approval and market access reality.

As regulatory pathways accelerate to meet patient needs, market access expectations grow increasingly complex. This divergence isn't merely procedural - it's creating material business risk for breakthrough innovations. After two decades of guiding companies through market access challenges, I've observed that recent developments in Europe illustrate this evolution most starkly.

European Evidence Shifts

While delays between regulatory and reimbursement approvals are not new, shifting evidence requirements and acceptable price points across markets creates acute challenges for breakthrough treatments. European Health Technology Assessment (HTA) regulations seek to bridge the evidence gap, and, starting in 2025, new cancer and advanced therapies will be the guinea pigs for early consultations that will extend to orphan drugs in 2028 and all medicines by 2030. While joint clinical assessment aims to align EMA and HTA body requirements, pricing decisions and patient access policies will remain firmly at the national level.

U.S. Market Transformation

Stateside, ICER’s growing influence and recent Inflation Reduction Act provisions for drug price negotiations are similarly driving a significant transformation in access dynamics. As healthcare costs rise and novel therapies seek premium prices, U.S. payers are increasingly adopting evaluation and negotiation frameworks that mirror European HTA bodies. Forward-thinking companies are taking aggressive steps to prepare for a future where comparative evidence requirements and public price negotiations will be routine in the U.S. as well as Europe.

A New Evidence Paradigm

This evolution transcends regional differences in evidence requirements and access regulations. We're seeing a fundamental shift in the expected demonstration of clinical value, particularly for breakthrough innovations. What was once a post-launch consideration is now an urgent commercial imperative. Comparative evidence must be considered and prioritized much earlier in development, boldly pursued to differentiate, and strongly resourced over the product’s lifecycle.

Leading Through Change

In my work developing Breakthrough Blueprints for leading pharmaceutical companies, I've seen how forward-thinking organizations are adapting. Their approach reveals a crucial insight: success in bringing breakthrough innovations to patients depends less on regulatory approval speed and more on how well companies understand and shape the evidence landscape from the start. Those leading this evolution are seeing dramatic reductions in approval timelines and significantly improved market uptake.

Strategic Imperatives for Success

Market leaders are already implementing three critical strategies to address these challenges:

1. Gather Insights from Stakeholders:
   Working closely with decision-makers and policy influencers across geographies early in clinical development to shape endpoints, identify real-world comparisons, and capture resource use measures that will resonate locally.
2. Integrate Payer Evidence Early:
   Embedding HTA requirements into Phase II trial design and creating parallel evidence streams to address gaps in efficacy data.
3. Collaborate with Patients:
   Engaging the patient community from the beginning of clinical development through post commercialization to gauge meaningful outcomes and comparisons, implement relevant, real-world evidence collection, and enhance responses to access challenges.

    

The Path Forward

The "hope-based" and “wait-and-see” era of market access is over. Our experience creating market shaping manuals shows that organizations proactively shaping the evidence landscape for multi-stakeholder acceptance gain a significant competitive advantage. Those that don't risk watching their innovations falter at the final hurdle.

The question isn't whether to adapt to this new reality, but how swiftly we can evolve our approach to ensure breakthrough innovations reach the patients who need them.

Betsy J. Lahue is Chief Executive Officer of Alkemi. She partners with pharmaceutical executives to transform regulatory success into appropriate market access for Breakthrough treatments globally.

To learn more about our comprehensive Breakthrough Blueprint to "Building Evidence Strategy for Breakthrough Success" and how to implement these strategies in your organization: Get in touch

Links:

Birgitte Klindt Poulsen: ISPOR BIO

Evidence Requirement: Apellis eye drug again turned back in Europe

Acceptable Price Points: England's NICE turns down cancer drug Enhertu—again—amid pricing stalemate with AZ, Daiichi Sankyo

Regulations: Implementing the EU Health Technology Assessment Regulation

Early Consultations: European Commission Joint Scientific Consultations

Joint Clinical Assessment: European Commission Joint Clinical Assessments

ICER’s Growing Influence: Payer Perspectives on the Use of ICER Evidence Reports Over the Past 24 Month

Inflation Reduction Act: Fact Sheet: Medicare Drug Price Negotiation Program Final Guidance for 2027 and Manufacturer Effectuation of the MFP in 2026 and 2027

Comparative Evidence: Lilly beats Novo in bellwether head-to-head weight loss study