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How to achieve HTA submission success for rare disease therapies
The challenge
A company pursuing reimbursement for a rare disease therapy faced significant HTA hurdles following a prior submission that resulted in a non‑recommendation. The program needed to demonstrate alignment between clinical evidence and economic analyses, address key evidence gaps, and ensure relevance to UK clinical practice, common challenges in rare disease assessments where data are limited, and uncertainty is high.
To achieve HTA submission success, the team required a focused, evidence‑driven approach that could strengthen the submission, mitigate uncertainty, and resonate with HTA bodies and payers, without unnecessary complexity or delay.
The Acumetis approach
Acumetis partnered closely with the client to deliver an integrated, evidence‑led HTA strategy tailored to the realities of rare disease assessment. Our work focused on connecting clinical evidence, health economics, and stakeholder insight into a coherent, decision‑ready submission.
Key elements of the approach included:
HTA critical appraisal and strategic planning
We conducted a detailed review of the previous HTA submission and appraisal outcomes to identify the drivers of non‑recommendation, prioritize key uncertainties, and define a targeted remediation strategy.
Systematic literature review (SLR)
We performed SLR using validated Scottish Intercollegiate Guidelines Network (SIGN) and Evidence Assessment Group (EAG) filters to ensure all relevant evidence is identified.
Comparative effectiveness and uncertainty management
Where head‑to‑head data were unavailable, we developed a robust indirect treatment comparison aligned with HTA methodological expectations, with explicit exploration of uncertainty and scenario testing.
Comparative effectiveness
We ran a de novo indirect treatment comparison (ITC) using novel methodology fully aligned with NICE Decision Support Unit (DSU) guidance, enabling a robust assessment of comparative effectiveness in the absence of head‑to‑head data while transparently addressing assumptions and uncertainty.
Economic modelling
We developed a de novo patient‑level cost‑effectiveness model (CEM) aligned to the clinical disease pathway, using established surrogate outcomes to project multiple long‑term clinical and economic outcomes.
Practical agility
Throughout the engagement, the team operated with practical agility, working in close collaboration with the client to iterate rapidly as new insights emerged. This lean, flexible approach enabled efficient execution, timely decision‑making, and focused use of resources, ensuring the submission remained aligned to HTA expectations without unnecessary complexity.
Key elements of the approach included:
Gap analysis
We assessed key data gaps and developed targeted evidence‑generation recommendations to address them in line with HTA expectations.
Delphi Panel
We conducted Delphi panels with international experts to support structured consensus development in areas with limited evidence.
External validation
With patient groups and KOL stakeholders, we align the submission and economic model structure to UK clinical practice, ensuring that the model structure and results are valid, resonate, and reflect real-world use.
The outcome
The strengthened HTA submission incorporated a clearer alignment of clinical evidence and economic analysis, with key data gaps addressed through systematic review, modelling, and external validation. By ensuring that assumptions and outputs reflected real‑world clinical practice, the submission was positioned to meet HTA expectations and improve the likelihood of a successful assessment for a rare disease therapy.
Acumetis global value & access team
Acumetis helps biopharma teams demonstrate value and enable access, even in the most complex rare disease settings. By connecting evidence strategy, health economics, and market access expertise, we help teams demonstrate value and enable access in the most complex rare disease settings. By connecting evidence strategy, health economics, and payer insights, we de‑risk the decisions that shape HTA outcomes and support submissions that are clear, relevant, and aligned to global and local requirements.
To learn how Acumetis Global Value & Access can support your HTA strategy, contact us to start the conversation.
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FAQs on HTA submission success for rare disease therapies
1. What makes HTA submissions for rare disease therapies particularly challenging?
2. How does Acumetis help address evidence gaps in rare disease HTA submissions?
3. Why is alignment with clinical practice important for HTA success?
4. How does Acumetis reduce uncertainty in HTA decision‑making?