The Phantom Menace: How Consultations and Appeals During NICE HST Appraisals Contribute to Delays in Patient Access
Author(s)
Daniel Anzola, MD, Daniela Gonçalves-Bradley, PhD, Christine Worsley, PGC.
STRATENYM Inc., Toronto, ON, Canada.
STRATENYM Inc., Toronto, ON, Canada.
OBJECTIVES: NICE’s Highly Specialised Technology (HST) programme evaluates treatments for ultra-rare conditions, aiming to provide timely patient access. However, delays from consultations, appeals, and additional committee meetings can delay final guidance. This study evaluated the impact of these events on the time to final guidance in completed HST appraisals.
METHODS: Publicly available HST appraisal data (programme inception to May 2025) were extracted and analysed for appraisal duration (Final Scope to Final Evaluation Determination) and number of consultations, appeals, and committee meetings.
RESULTS: Of 30 published HST appraisals, 2 technologies were not recommended. Mean duration of recommended appraisals was 81 weeks (range: 39-318). Of the 28 appraisals that ultimately resulted in a positive recommendation, 4 had no consultations or appeals and 24 underwent ≥1 consultation. Four of these HST appraisals required 2 consultations before final guidance was issued. Appeals were less frequent; 3/24 appraisals had ≥1 appeal. Where reported, the number of evaluation committee meetings ranged from 2 to 5. The duration of the HST appraisal process was substantially longer when additional events such as consultations and appeals were held, when compared with HST appraisals for which no consultations or appeals were observed (mean [range] duration, 86 [39-318] vs. 51 [44-58] weeks).
CONCLUSIONS: Delays in the HST appraisal process can push back patient access to rare disease therapies by many months, sometimes years. For the submitting company, this can increase both operational costs and financial burdens from lost revenue. Our findings relating to delays in patient access in rare diseases support the case for better preparation for submission, which should include increased engagement with HTA bodies and the clinical and patient community, coupled with well-thought-out evidence collection and value messaging. These steps can mitigate risk and increase the chances of a smooth, timely appraisal that benefits all parties.
METHODS: Publicly available HST appraisal data (programme inception to May 2025) were extracted and analysed for appraisal duration (Final Scope to Final Evaluation Determination) and number of consultations, appeals, and committee meetings.
RESULTS: Of 30 published HST appraisals, 2 technologies were not recommended. Mean duration of recommended appraisals was 81 weeks (range: 39-318). Of the 28 appraisals that ultimately resulted in a positive recommendation, 4 had no consultations or appeals and 24 underwent ≥1 consultation. Four of these HST appraisals required 2 consultations before final guidance was issued. Appeals were less frequent; 3/24 appraisals had ≥1 appeal. Where reported, the number of evaluation committee meetings ranged from 2 to 5. The duration of the HST appraisal process was substantially longer when additional events such as consultations and appeals were held, when compared with HST appraisals for which no consultations or appeals were observed (mean [range] duration, 86 [39-318] vs. 51 [44-58] weeks).
CONCLUSIONS: Delays in the HST appraisal process can push back patient access to rare disease therapies by many months, sometimes years. For the submitting company, this can increase both operational costs and financial burdens from lost revenue. Our findings relating to delays in patient access in rare diseases support the case for better preparation for submission, which should include increased engagement with HTA bodies and the clinical and patient community, coupled with well-thought-out evidence collection and value messaging. These steps can mitigate risk and increase the chances of a smooth, timely appraisal that benefits all parties.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA326
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas