Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU

Vokinger, Kerstin Noëlle; Kesselheim, Aaron S.

doi:10.1136/bmjopen-2018-028634

Cited by 27 publications

(23 citation statements)

References 27 publications

(35 reference statements)

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“…Future efforts in histology-agnostic drug development should ensure that patients receive therapeutic agents targeting a true driver mutation and acknowledge co-occurring alterations in known resistance pathways [ 68 , 71 ]. The evaluation of potential tissue agnostic treatments in oncology raises a number of issues for consideration of orphan drug designation such as what factors should be considered in defining a tissue agnostic disease or condition at the time of request for orphan drug designation, how FDA and EMA should consider orphan drug designation requests of the same product for a tissue agnostic disease or condition and also for a disease based on traditional disease nomenclature [ 72 ].…”

Section: Discussion: Different Challenges and Opportunitiesmentioning

confidence: 99%

Towards Better Pharmaceutical Provision in Europe—Who Decides the Future?

et al. 2022

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Significant progress has been achieved in human health in the European Union in recent years. New medicines, vaccines, and treatments have been developed to tackle some of the leading causes of disease and life-threatening illnesses. It is clear that investment in research and development (R&D) for innovative medicines and treatments is essential for making progress in preventing and treating diseases. Ahead of the legislative process, which should begin by the end of 2022, discussions focus on how Europe can best promote the huge potential benefits of new science and technology within the regulatory framework. The challenges in European healthcare were spelled out by the panellists at the roundtable organised by European Alliance for Personalised Medicine (EAPM). Outcomes from panellists’ discussions have been summarized and re-arranged in this paper under five headings: innovation, unmet medical need, access, security of supply, adapting to progress, and efficiency. Some of the conclusions that emerged from the panel are a call for a better overall holistic vision of the future of pharmaceuticals and health in Europe and a collaborative effort among all stakeholders, seeing the delivery of medicines as part of a broader picture of healthcare.

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Section: Discussion: Different Challenges and Opportunitiesmentioning

confidence: 99%

Towards Better Pharmaceutical Provision in Europe—Who Decides the Future?

et al. 2022

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“…In particular, for non-melanoma skin cancers, the activity of ICIs is outstanding, and cemiplimab was recently approved for advanced squamous cell histology in both the US and European countries. In other rare tumours, the efficacy of ICI therapy cannot be fully ascertained because of the small sample sizes and non-randomised design of clinical trials ( 78 , 79 ).…”

Section: Discussionmentioning

confidence: 99%

Efficacy of Immune Checkpoint Inhibitors in Rare Tumours: A Systematic Review

et al. 2021

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BackgroundRare cancers, as defined by the European Union, occur in fewer than 15 out of 100,000 people each year. The International Rare Cancer Consortium defines rare cancer incidence as less than six per 100,000 per year. There is a growing number of reports of the efficacy of immune checkpoint inhibitor (ICI) therapy in patients with rare tumours, and hence, we conducted a comprehensive review to summarise and analyse the available literature.MethodsA literature search of PubMed was performed on January 31, 2021, using the following ICI names as keywords: ipilimumab, tremelimumab, cemiplimab, nivolumab, pembrolizumab, avelumab, atezolizumab, and durvalumab. Studies on patients with rare tumours who were being treated with ICIs were included. We plotted the overall response rate against the corresponding median survival across a variety of cancer types using linear regression.ResultsFrom 1,255 publications retrieved during the primary search, 62 publications were selected (with a total of 4,620 patients). Only four were randomised trials. A minority were first-line studies, while the remaining were studies in which ICIs were delivered as salvage therapy in pretreated patients. There was a good correlation between response rate and overall survival (Spearman R2 >0.9) in skin cancers, mesothelioma, and sarcomas.ConclusionsTreatment of advanced-stage rare tumours with ICI therapy was found to be associated with significant activity in some orphan diseases (e.g., Merkel cell carcinoma) and hepatocellular carcinoma. Several ongoing prospective clinical trials will expand the knowledge on the safety and efficacy of ICI therapy in patients with these rare cancers.

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“…An average increase in public expenditures on the reimbursement was 68%, as compared with an increase of only 8.5% in the total GDP and of 9.3% in GDP per capita. The factors influencing the increase could be associated with an increased number of reimbursed drugs, subject that was covered extensively by Vokinger and Kesselheim [11] as well as changes in public budget expenditures or in reimbursement policy. Changes in pricing could also result in fluctuations in expenditures.…”

Section: Discussionmentioning

confidence: 99%

Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe

Malinowski

Kawalec

Trąbka

et al. 2020

Orphanet J Rare Dis

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Background The reimbursement of orphan drugs (OD) is an increasingly important for country policymakers, and still insufficiently understood, especially in Central and Eastern Europe. The aim of this research was to provide a comprehensive description of country-specific health technology assessment (HTA) policies as well as evaluate the percentage of HTA recommendations and reimbursement decisions for oncology OD. In addition, the study was designed to elucidate the impact of reimbursement of these drugs on the public budget and the agreement between HTA recommendations and reimbursement decisions in the analysed countries. A questionnaire survey was used to collect data on the reimbursement status, HTA recommendation, marketing authorisation, and public expenses on reimbursement in 2014, 2015, and 2016 for all oncology drugs with an orphan designation by the European Medicine Agency in 2017 in Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, and Slovakia. The agreement between the HTA recommendation and reimbursement status was assessed using the kappa coefficient. The Pearson’s correlation was used to analyse the relationship between gross domestic product (GDP) and GDP per capita and reimbursement expenses. Results A total of 36 drugs were analysed (25% conditionally approved; 5.56% approved under exceptional circumstances). The share of reimbursed drugs ranged from 11.11% in Latvia to 41.67% in Poland. The highest share of positive recommendations was observed for Bulgaria and Estonia (36.11%), and the lowest, for Latvia (11.11%). The agreement varied from 0.4 for Poland to 1 for Latvia, Hungary, and Slovakia. Expenses were correlated with GDP (0.95 [0.81–0.99]), and not with GDP per capita (0.54 [− 0.136 to 0.873]). Expenses per capita were not correlated with GDP per capita (0.52 [− 0.15 to 0.87]). Conclusions In Hungary, Latvia, and Slovakia, a positive recommendation was associated with a reimbursement, and a negative one, with the lack of reimbursement. The reimbursement of oncology OD is associated with a growing burden for public budget, and the expenses are correlated with the total GDP. The highest share of drugs with any recommendation was observed in Poland, and the lowest, in Latvia and Romania. The share of reimbursed drugs was the lowest in Latvia and the highest in Poland.

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Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU

Cited by 27 publications

References 27 publications

Towards Better Pharmaceutical Provision in Europe—Who Decides the Future?

Towards Better Pharmaceutical Provision in Europe—Who Decides the Future?

Efficacy of Immune Checkpoint Inhibitors in Rare Tumours: A Systematic Review

Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe

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