The Impact of Meeting Orphan Criteria on Reimbursement Decisions and ICERs in Scotland
Author(s)
Marta Wilk, MSc1, Kamilia Ben Abdallah, MEng2, Lylia Chachoua, PhD2, Samuel Aballea, MSc, PhD3, Mondher Toumi, MD, MSc, PhD4.
1Clever-Access, Cracow, Poland, 2Clever-Access, Paris, France, 3InovIntell, Rotterdam, Netherlands, 4InovIntell, Paris, France.
1Clever-Access, Cracow, Poland, 2Clever-Access, Paris, France, 3InovIntell, Rotterdam, Netherlands, 4InovIntell, Paris, France.
OBJECTIVES: The Scottish Medicine Consortium (SMC) is willing to accept greater uncertainty in the economic case for orphan drugs, but rarity is not supposed to influence the accepted cost per QALY. This research aims to evaluate the impact of meeting orphan criteria (orphan vs. non-orphan), in general and within the specific area of oncology, on reimbursement decisions and incremental cost-effectiveness ratios (ICERs) in Scotland.
METHODS: Submission data, ICERs, and HTA outcomes (recommended without restriction, restricted, interim recommendation, not recommended) were extracted for SMC appraisals from the NaviHTA database. HTA outcomes and ICERs were compared between orphan drugs and others.
RESULTS: Of the 262 SMC reports identified, 145 (55%) concerned products that met SMC orphan criteria. The majority of oncological drugs (78%) met these criteria. The likelihood of receiving a positive recommendation (with and without restriction and interim recommendation) was similar for orphan and non-orphan drugs (90% vs. 94%, respectively). However, orphan-designated products were more likely to receive a recommendation without restriction (50%) compared to non-orphan products (37%). Among oncological drugs, these percentages were 56% and 60% with and without orphan designation, respectively. ICERs were available for 77% of recommended cases and ranged from £24 to £1,558,488/QALY. ICERs were generally higher for products meeting orphan criteria, with a median of £44,013/QALY, compared to £17,356/QALY for other products. Among oncological drugs, the median ICER was also higher for drugs meeting orphan criteria compared to others (£41,201 vs £34,541/QALY).
CONCLUSIONS: The findings indicate greater flexibility from SMC in assessing products meeting orphan criteria compared to non-orphan drugs, reflected by a higher proportion of recommendations without restriction and acceptance of higher ICERs. However, the influence of orphan criteria appears less pronounced in oncological indications, where full recommendations and ICERs show more alignment between orphan and non-orphan drugs.
METHODS: Submission data, ICERs, and HTA outcomes (recommended without restriction, restricted, interim recommendation, not recommended) were extracted for SMC appraisals from the NaviHTA database. HTA outcomes and ICERs were compared between orphan drugs and others.
RESULTS: Of the 262 SMC reports identified, 145 (55%) concerned products that met SMC orphan criteria. The majority of oncological drugs (78%) met these criteria. The likelihood of receiving a positive recommendation (with and without restriction and interim recommendation) was similar for orphan and non-orphan drugs (90% vs. 94%, respectively). However, orphan-designated products were more likely to receive a recommendation without restriction (50%) compared to non-orphan products (37%). Among oncological drugs, these percentages were 56% and 60% with and without orphan designation, respectively. ICERs were available for 77% of recommended cases and ranged from £24 to £1,558,488/QALY. ICERs were generally higher for products meeting orphan criteria, with a median of £44,013/QALY, compared to £17,356/QALY for other products. Among oncological drugs, the median ICER was also higher for drugs meeting orphan criteria compared to others (£41,201 vs £34,541/QALY).
CONCLUSIONS: The findings indicate greater flexibility from SMC in assessing products meeting orphan criteria compared to non-orphan drugs, reflected by a higher proportion of recommendations without restriction and acceptance of higher ICERs. However, the influence of orphan criteria appears less pronounced in oncological indications, where full recommendations and ICERs show more alignment between orphan and non-orphan drugs.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA19
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology, SDC: Rare & Orphan Diseases