Last Updated: October 24th, 2025
We drove Health Technology Assessment (HTA) submission success for rare disease therapies by combining critical appraisals, systematic literature reviews, economic modeling, and stakeholder validation, while proactively addressing data gaps.
What Is HTA Submission Success for Rare Diseases?
HTA submission success for rare disease therapies means providing evidence and analyses that meet review standards and resonate with HTA bodies and payers. It involves aligning clinical data and economic modeling to UK practice and addressing key evidence gaps. Approaches are then validated with patient groups and key opinion leaders to ensure relevance and credibility.
How Our Technology Delivers Results
We combine technical expertise with deep healthcare strategy and clinical insight on every project. Our work includes:
- HTA Critical Appraisal: We reviewed a previous NICE submission that had resulted in a non-recommendation.
- Systematic Literature Review: We conducted a systematic literature review (SLR) using validated Scottish Intercollegiate Guidelines Network (SIGN) and Evidence Assessment Group (EAG) filters to capture all relevant evidence.
- Comparative Effectiveness: We performed a de novo indirect treatment comparison (ITC) following methodology fully aligned with NICE Decision Support Unit (DSU) guidelines.
- Economic Modeling: We developed a de novo patient-level model tailored to the clinical disease, utilizing established surrogate outcomes to project multiple long-term outcomes.
How Our Practical Agility Keeps Teams Ahead
We are practically agile, operating with a lean structure that enables seamless teamwork and rapid resource deployment. Our process ensures efficient project completion without unnecessary bureaucracy. Key elements include:
Gap Analysis: We identified critical data gaps and recommended strategies to generate evidence to address them.
Delphi Panel: We conducted Delphi panels with international experts to help fill evidence gaps.
External Validation: Working with patient groups and key opinion leaders (KOLs), we align the submission and economic model structure to UK clinical practice. Ensuring the model and its results are valid, relevant, and reflective of real-world use.
Customer Testimonies
Why This Matters for Rare Disease Therapies
Rare disease therapies often face heightened scrutiny around evidence quality, making it essential to identify and address gaps early in development. Using validated models and well-designed literature reviews strengthens credibility and helps reduce the risk of unfavorable HTA outcomes. Early and ongoing engagement with key stakeholders, including clinical experts, patient groups, and payers, supports alignment with real-world clinical practice and evolving HTA expectations. Maintaining agility throughout the process allows emerging data and stakeholder feedback to be incorporated, strengthening the overall submission and its likelihood of success.
A Step Forward in Rare Disease HTA Submissions
By combining deep technical expertise with practical agility, companies can strengthen rare disease HTA submissions. In rare diseases, clinical trials are often smaller and data more limited. It is essential to align evidence and modeling with real-world clinical practice. Companies must identify and address key data gaps and validate approaches with patient groups and key opinion leaders. This targeted, evidence-driven approach creates credible, actionable submissions that support reimbursement success and improve patient access.
Ready to strengthen your rare disease HTA submission?
Meet our HTA Team
FAQs on HTA Submission: Rare Disease Therapies
- What is involved in a “critical appraisal”?
Reviewing past failed or borderline HTA submissions to understand what issues led to non-recommendation so that they can be avoided in future submissions. - How does the systematic literature review improve a submission?
It ensures comprehensive evidence capture, using validated filters to find all relevant studies, strengthening the HTA dossier’s foundation. - Why is external validation important?
It confirms that models and assumptions reflect real-world use and clinical practice, which helps HTA bodies accept the submission. - What is a Delphi Panel and why use one?
A Delphi Panel is a structured method for gaining consensus from international experts on uncertain evidence areas; it’s useful for filling gaps when data are sparse. - When should a company start this process?
It is best to start early in development (before pivotal trials) so critical appraisals, evidence generation, modeling, and stakeholder alignment can inform strategy and avoid late-stage surprises.