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 assessed critical data gaps and recommended evidence generation strategies 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 stricter scrutiny over evidence quality; addressing gaps early is critical.
- Validated models and well-conducted literature reviews increase credibility and reduce the risk of rejection.
- Engaging stakeholders (experts, patient groups, payers) improves alignment with clinical reality and HTA expectations.
- Agility ensures that emerging data or stakeholder feedback can be incorporated to strengthen the submission.
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
Lisa Cox
Executive Director
Lisa has over 16 years of experience in HEOR consulting and has led the strategy, planning, and execution of various global, multi-disciplinary programs. From early HEOR and HTA landscaping to global reimbursement programs, Lisa has successfully contributed to over 100 submissions, including NICE STAs, FTAs, HSTs, and MTAs.
Sabine Gaugris
Executive Director
Sabine brings experience in HEOR across multiple disease areas within the pharma industry and consulting. Over the past 20 years, she has led successful HTA submissions in the UK and managed outcomes research studies, including network meta-analyses, survival analyses, and observational studies. She has developed global value dossiers, covering core health economics components from systematic reviews to economic models.
Romita Das
Associate Director
Romita has over 15 years of experience in health economics and outcomes research, specializing in rare diseases and challenging HTA submissions. She has worked across a wide range of therapy areas, delivering national market access solutions for major pharmaceutical companies like AstraZeneca and Merck Sharp & Dohme.
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.