The correlation between HTA recommendations and reimbursement status of orphan drugs in EuropeReport as inadecuate

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Orphanet Journal of Rare Diseases

, 11:122

Orphan Drugs


BackgroundThe aim of this study was to review and compare types of reimbursement recommendations for orphan drugs issued by eight European health technology assessment HTA agencies and the reimbursement status of these drugs in the corresponding countries. Separate calculations were also performed for three sub-groups: ultra-orphan drugs, oncology orphan drugs and other non-ultra, non-oncology orphan drugs.

ResultsWe reviewed drugs authorized by the European Medicine Agency EMA between 1 November 2002 and 30 September 2015. Among these, we identified 101 orphan drugs. Seventy-nine of them were assessed by eight European HTA agencies. The average rates of positive, conditional and negative reimbursement recommendations issued by these agencies were 55.7 %, 15.3 % and 29.0 %, respectively. On average, 21.2 % of EMA-authorized orphan drugs were reimbursed in the eight European countries studied: 49.0 % of those with positive, 53.6 % of those with conditional, and 16.0 % of those with negative reimbursement recommendations. In addition, 5.4 % of orphan drugs that had not been assessed by any of the eight HTA agencies were also reimbursed. The shares of oncology, ultra, and other orphan drugs that were assessed by HTA agencies were similar, with the lowest share observed in ultra-orphan drugs 72 % and the highest in other orphan drugs 80 %. In terms of reimbursement, 20 % of oncology orphan drugs, 25 % of ultra-orphan drugs and 21 % of other orphan drugs were reimbursed.

ConclusionsReimbursement of orphan drugs does not always correspond to the type of HTA recommendation. While the highest rate of reimbursement is observed unsurprisingly among drugs with positive or conditional recommendation, a high rate of reimbursement 11 % is also observed among ultra-orphan drugs that had never been assessed by any HTA agency.

KeywordsOrphan drugs Ultra-orphan drugs Oncology orphan drugs Drug reimbursement Reimbursement status Reimbursement decision Health technology assessment AbbreviationsAOTMiTAgencja oceny Technologii Medycznych i Taryfikacji Polish HTA Agency

AWMSGAll Wales Medicines Strategy Group

EMAEuropean Medicines Agency

EUEuropean Union

G-BAThe General Joint Committee of Germany Gemeinsamer Bundesausschuss

HASFrench National Authority for Health Haute Autorité de Santé

HTAHealth Technology Assessment

NICENational Institute for Health and Care Excellence

SMCScottish Medicines Consortium

TLVDental and Pharmaceutical Benefits Agency Tandvårds- och läkemedelsförmånsverket

ZINNational Health Care Institute Zorginstituut Nederland

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Author: Paweł Kawalec - Anna Sagan - Andrzej Pilc


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