The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe

Kawalec, Paweł; Sagan, Anna; Pilc, Andrzej

doi:10.1186/s13023-016-0501-4

Cited by 35 publications

(39 citation statements)

References 5 publications

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“…The National Institute for Health Research Horizon Scanning Research & Intelligence Centre has a remit to identify key emerging health technologies that may have a significant impact on patients or the provision of health services for NICE to appraise. In England, ODs are not evaluated using any special HTA or reimbursement considerations50 and are appraised under the standard technology appraisal program. NICE bases its recommendation on clinical effectiveness and an assessment of the ICER of a new technology and how this compares to their cost per QALY threshold 51.…”

Section: Resultsmentioning

confidence: 99%

Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category?

Adkins

Nicholson

Floyd

et al. 2017

CEOR

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Orphan drugs (ODs) are intended for the diagnosis, prevention, or treatment of rare diseases. Many cancer subtypes, including all childhood cancers, are defined as rare diseases, and over one-third of ODs are now intended to treat oncology indications. However, market access for oncology ODs is becoming increasingly challenging; ODs are prone to significant uncertainty around their cost-effectiveness, while payers must balance the need for these vital innovations with growing sensitivity to rising costs. The objective of this review was to evaluate different mechanisms that have been introduced to facilitate patient access to oncology ODs in five different countries (Australia, Canada, England, France, and Sweden), using eight oncology ODs and non-orphan oncology drugs as examples of their application. A targeted literature review of health technology assessment (HTA) agency websites was undertaken to identify country-specific guidance and HTA documentation for recently evaluated oncology ODs and non-orphan oncology drugs. None of these countries were found to have explicit HTA criteria for the assessment of ODs, and therefore, oncology ODs are assessed through the usual HTA process. However, distinct and additional processes are adopted to facilitate access to oncology ODs. Review of eight case-study drugs showed that these additional assessment processes were rarely used, and decisions were largely driven by proving cost-effectiveness using standard incremental cost-effectiveness ratio (ICER) thresholds. The predominant implication arising from this study is that application of standard HTA criteria to oncology ODs in many countries fails to take into account any uncertainties around their clinical- and cost-effectiveness, resulting in disparities in HTA reimbursement decisions based on differences in addressing or accepting uncertainty. In order to address this issue, HTA agencies should adopt a more flexible approach to cost-effectiveness, as typified by the Tandvårds-och Läkemedelsförmånsverket in Sweden, which takes into account the small patient numbers involved, limited budget impact, and high unmet medical needs.

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Section: Resultsmentioning

confidence: 99%

Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category?

Adkins

Nicholson

Floyd

et al. 2017

CEOR

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“…The present study is innovative, as we did not identify valid studies on the similar topic carried out for European countries. A study by Kawalec et al ( 2016 ) had a similar approach, but a revision of methods and an update of input data were necessary to provide the topical and valid review on the management of orphan drugs in the selected European countries. The study revealed that 21% of EMA-authorized orphan drugs were reimbursed in 8 European countries that were studied: 49% of those orphan drugs had positive reimbursement recommendations, 54% of those had conditional reimbursement recommendations, and 16% had negative reimbursement recommendations.…”

Section: Discussionmentioning

confidence: 99%

“…The shares of the orphan drugs for oncologic diseases, orphan drugs for ultrarare diseases and other orphan drugs that were assessed by HTA agencies were similar, with the lowest percentage observed in ultra-orphan drugs (72%) and the highest in other orphan drugs (80%). While the highest rate of reimbursement was observed among drugs with positive or conditional recommendation, a high rate of reimbursement (11%) was revealed among ultra-orphan drugs that had never been assessed by any HTA agency (Kawalec et al, 2016 ). Although methods used in the previous study by Kawalec et al ( 2016 ) seem quite similar, the results are unsuitable for direct comparisons.…”

Section: Discussionmentioning

confidence: 99%

Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment

et al. 2018

Self Cite

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Objective: To assess shares of reimbursed orphan drugs and agreement in reimbursement decision-making in different European Union member states as well as to define odds for reimbursement influenced by the presence of conditional approval or exceptional circumstances granted by the European Medicines Agency (EMA) or by type of the disease.Methods: The list of authorized drugs with current orphan designations was collected from the website of the EMA. For each drug, the information regarding conditional approval or approval under exceptional circumstances was collected. The reimbursement statuses were available on national reimbursement or HTA agencies websites. The agreement for reimbursement decisions between selected countries was assessed using the κ coefficient for the measurement of agreement. The impact of the EMA's conditional approval as well as approval under exceptional circumstances was assessed using the logistic regression and presented as odds ratio.Results: The percentage of reimbursed orphan drugs varied significantly from 27% in Poland to 88% in Denmark, with an average value of 51% (p < 0.0001). Regarding the reimbursement status, the highest, substantial agreement was observed between Spain and Italy, and the lowest agreement was observed between Germany and England, with κ of 0.64 and 0.01, respectively. Conditional approval status significantly decreased the chance for reimbursement in France, Italy, and Spain by 77–80%; however, approval granted under exceptional circumstances had significant impact only in Germany with 85% decrease in chances for reimbursement. The type of the disease (oncology or metabolic) was significantly associated with both conditional approval (p of 0.03—oncology drugs were more likely to be conditionally approved then the rest of analyzed drugs) and exceptional circumstances (p of 0.02—drugs for metabolic diseases were more likely to be approved under exceptional circumstances).Conclusions: Access to reimbursed orphan drugs varies significantly across EU countries. The highest, substantial agreement in reimbursement decisions was observed between Italy and Spain and the lowest between Germany and England. Conditional approval and approval under exceptional circumstances were significant negative predictors of reimbursement in some countries and they were significantly associated with the type of the disease (oncology or metabolic).

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“…Orphan drugs tend to be very expensive on an individual patient basis but provide significant health benefits to those treated [27]; whereas pharmacogenetic tests are inexpensive for individual patients, but expensive for populations and benefit only a small proportion of those tested. Because of their high costs, economic evaluations of orphan drugs often yield ICERs in the order of hundreds of thousands (if not millions) of pounds per QALY gained [28], far exceeding the cost-effectiveness threshold. Yet despite this, most orphan drugs are approved for use [29], often justified on the grounds of equity [30].…”

Section: Articlementioning

confidence: 99%

Rare Disease Prevention and Treatment: the Need for a Level Playing Field

Hughes

Plumpton

2018

Pharmacogenomics

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Pharmacogenetic tests are being used increasingly to prevent rare and potentially life-threatening adverse drug reactions. For many tests, however, cost-effectiveness is hard to demonstrate, and with the exception of a few cases, widespread implementation remains a distant prospect. Many orphan drugs for rare diseases are also not cost effective but are nonetheless normally reimbursed. In this article, we argue that the health technology assessment of pharmacogenetic tests aimed to prevent rare but severe adverse drug reactions should be on a level playing field with orphan drugs. This is supported by a number of arguments, concerning the severity, rarity and iatrogenic nature of adverse drug reactions, the distribution of benefits and costs and societal preference towards prevention over treatment.

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The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe

Cited by 35 publications

References 5 publications

Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category?

Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category?

Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment

Rare Disease Prevention and Treatment: the Need for a Level Playing Field

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