The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe
The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe
Paweł Kawalec1*, Anna Sagan2,4 and Andrzej Pilc3
Orphanet Journal of Rare Diseases (2016) 11:122
The 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.
Results: We 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.
Conclusions: Reimbursement 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.
Keywords: Orphan drugs, Ultra-orphan drugs, Oncology orphan drugs, Drug reimbursement, Reimbursement status, Reimbursement decision, Health technology assessment Abbreviations: AOTMiT, Agencja oceny Technologii Medycznych i Taryfikacji (Polish HTA Agency); AWMSG, All Wales Medicines Strategy Group; EMA, European Medicines Agency; EU, European Union; G-BA, The General Joint Committee of Germany (Gemeinsamer Bundesausschuss); HAS, French National Authority for Health (Haute Autorité de Santé); HTA, Health Technology Assessment; NICE, National Institute for Health and Care Excellence; SMC, Scottish Medicines Consortium; TLV, Dental and Pharmaceutical Benefits Agency (Tandvårds- och läkemedelsförmånsverket); ZIN, National Health Care Institute (Zorginstituut Nederland)
