Understanding Reasons of Negative Reimbursement Decisions for Orphan Drugs in the Spanish National Health System (APRIL 2019-MAY 2023)

Author(s)

Gil A, Pérez Á, Badia X
Omakase Consulting S.L., Barcelona, B, Spain

OBJECTIVES: Since April 2019, the Spanish Interministerial Pricing Committee (CIPM) resolutions include the criteria behind P&R decisions. ODs are usually associated with limited data and perceived high prices, resulting in many cases in negative P&R decisions and limited access. A previous study (2022) showed that 57% of ODs assessed between April 2019 and June 2022 were not reimbursed at first P&R submission and 33% obtained P&R approval after allegations. Budget impact (BI) and uncertainty on relative clinical efficacy contribution represent the main criteria for unsuccessful P&R. This study aimed to update (until May 2023) the number and reasons behind negative P&R decisions for ODs in Spain.

METHODS: Review of CIPM decisions between April 2019 and May 2023 to identify ODs with a negative P&R resolution and the reasons according to Spanish legislation criteria.

RESULTS: 58 ODs were assessed by the CIPM during study period of which 37 (64%) received an initial negative P&R decision. BI (86%) and uncertainty on relative clinical benefit (46%) were most common criteria behind negative reimbursement decisions. Marketing Authorisation Holders (MAHs) submitted allegations to initial negative P&R resolutions for 31 (86%), of which 16 (52%) were rejected. Of these, in 9 cases (56%) the MAH withdrew from the process with the remaining 7 (44%) proceeding through a second round, resulting in a subsequent negative resolution in 5 (71%) of cases. Allegations to these negative resolutions were presented for 4 (80%), with 3 (75%) finally obtaining P&R approval.

CONCLUSIONS: 16 additional ODs have been evaluated in the last year. Compared to previous data, initial negative resolutions have increased from 57% to 64% (+7%) with a decrease in negative resolutions after allegations from 63% to 52% (-11%). Reasons behind decisions remain the same: BI and uncertainty on relative clinical benefit.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

EE394

Topic

Economic Evaluation, Health Policy & Regulatory

Topic Subcategory

Budget Impact Analysis, Reimbursement & Access Policy

Disease

Rare & Orphan Diseases

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