Focus on Two Years of Economic Opinions on Orphan Drug: What Are the Specific Features?
Author(s)
Sambuc C1, Boussahoua M2, Tehard B2, Midy F2, Chevalier J2, Roze S3
1VYOO Agency, Paris, 75, France, 2Vyoo Agency, Paris, 75, France, 3Vyoo Agency, VILLEURBANNE, 69, France
Presentation Documents
OBJECTIVES: In France, the Commission for Economic and Public Health Evaluation (CEESP) provides an economic opinion of the incremental cost-effectiveness ratio (ICER) validity.
Orphan drugs occupy a special place in terms of technological innovation and are often awaited by patients who have reached a therapeutic impasse. At the same time, the availability of data to document the analyses associated with these treatments represents a real challenge. The aim is to analyze the CEESP opinions in orphan drug appraised over the last two years (2021-2022) to discuss how the CEESP stands in relation to this type of challenge.METHODS: Using Vyoo Agency efficiency database, all CEESP opinions on orphan drugs between January 1, 2021, and December 31, 2022, were reviewed.
RESULTS: Fourteen CEESP opinions concerned orphan drug on 12 different diseases. CEESP opinions included neither major objection nor global uncertainty for only 29% (4/14) of the dossiers. For these 4 dossiers, the average ICER is €777, 878/QALY and ranges from €7,392/QALY to €2,429,397/QALY.
Among the 14 economic opinions, 6 opinions were in onco-hematology, 3 of them have a validated ICER (50%). Only 1 economic opinion considered that the economic information provided by the model submitted by the industrial was informative in opinions covering all other therapeutic areas (1/8). In one opinion, the CEESP chose to postpone the results of one of the sensitivity analyses presented by the industrial, even though it invalidated the ICER of the base case.CONCLUSIONS: Over the two years considered for the analysis, CEESP has delivered 55 economic opinions, including 21 with a validated ICER (38%). Opinions on orphan drugs comprised 25% of all opinions appraised during this period and is characterized by a lower proportion of validated economic opinions (29%), especially concerning therapeutic areas other than onco-hematology (13%). Fulfilling the requirements of CEESP seems difficult for rare diseases.
Conference/Value in Health Info
Value in Health, Volume 26, Issue 11, S2 (December 2023)
Code
HTA247
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Cost-comparison, Effectiveness, Utility, Benefit Analysis, Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases