Role of Technical Engagement in NICE Process Under Proportionate Approach to Technology Appraisals
Author(s)
Ewa Rupniewska, PhD1, Carina M. Behr, PhD2, Stuart Gaines, PhD3, Ayesha R. Qureshi, MSc4, Ben Kearns, PhD3, Sallie Latimer, BSc3.
1Lumanity, London, United Kingdom, 2Lumanity, Utrecht, Netherlands, 3Lumanity, Sheffield, United Kingdom, 4Lumanity, Manchester, United Kingdom.
1Lumanity, London, United Kingdom, 2Lumanity, Utrecht, Netherlands, 3Lumanity, Sheffield, United Kingdom, 4Lumanity, Manchester, United Kingdom.
OBJECTIVES: On 24 April 2023, NICE formally introduced its proportionate approach to technology appraisals (PATT), making Technical Engagement (TE) optional when “NICE considers that it is appropriate, helpful and proportionate, taking into account whether the TE process is likely to resolve key issues before the committee meeting”. We aimed to evaluate the frequency of TE inclusion since it became optional, the characteristics of topics selected for TE, and the success of TE in facilitating committee decision-making.
METHODS: We screened all NICE Technology Appraisals (TAs) published between 1 October 2023 and 30 April 2025 to identify and characterize Single Technology Appraisals (STAs) with TE and Invitation to Participate (ITP) after 24 April 2023.
RESULTS: Of 139 TAs screened, 50 were STAs that reached a conclusion and had ITPs after PATT introduction, of which only three (6%) involved TE. All three TE STAs were for orphan drugs with complex evidence bases (single-arm studies with limited follow-up); two were for a gene therapy (exagamglogene autotemcel). TE in TA1012 enabled the company to submit new clinical evidence (longer follow-up), resolve some key issues and align its base case with External Assessment Group preferences, leading to positive recommendation within one committee meeting. TE in TA1044 enabled an independent review of the company's economic model by Decision Support Unit, and submission of a revised model that was used for decision-making. However, two committee meetings were required to reach a managed access recommendation. TE in TA1003 appeared unproductive (no new evidence; no substantial changes to the economic model; no major issues resolved), but managed access recommendation was reached in one committee meeting.
CONCLUSIONS: NICE’s use of TE is now extremely uncommon, seemingly reserved for rare diseases with complex evidence bases. TE was clearly successful in one case, while its impact in the other two cases is less evident.
METHODS: We screened all NICE Technology Appraisals (TAs) published between 1 October 2023 and 30 April 2025 to identify and characterize Single Technology Appraisals (STAs) with TE and Invitation to Participate (ITP) after 24 April 2023.
RESULTS: Of 139 TAs screened, 50 were STAs that reached a conclusion and had ITPs after PATT introduction, of which only three (6%) involved TE. All three TE STAs were for orphan drugs with complex evidence bases (single-arm studies with limited follow-up); two were for a gene therapy (exagamglogene autotemcel). TE in TA1012 enabled the company to submit new clinical evidence (longer follow-up), resolve some key issues and align its base case with External Assessment Group preferences, leading to positive recommendation within one committee meeting. TE in TA1044 enabled an independent review of the company's economic model by Decision Support Unit, and submission of a revised model that was used for decision-making. However, two committee meetings were required to reach a managed access recommendation. TE in TA1003 appeared unproductive (no new evidence; no substantial changes to the economic model; no major issues resolved), but managed access recommendation was reached in one committee meeting.
CONCLUSIONS: NICE’s use of TE is now extremely uncommon, seemingly reserved for rare diseases with complex evidence bases. TE was clearly successful in one case, while its impact in the other two cases is less evident.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA294
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas