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Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries.

ABSTRACT: Objective: To review the reimbursement recommendations issued by selected European health technology assessment agencies for orphan drugs and the reimbursement status of these drugs; to assess the relationship between the type of recommendation and reimbursement status. Methods: The list of orphan drugs to be included in the analysis was obtained from the European Medicines Agency and Orphanet. Seven European states were included in the analysis: Belgium, England, France, Germany, Poland, Scotland, and Spain. For all identified orphan drugs, relevant data on the reimbursement status and type of recommendation were collected for each country. The relationship between the type of recommendation and reimbursement status was evaluated separately for each considered country, using Cohen's kappa coefficient for the measurement of agreement; sub-analyses for oncology and metabolic drugs were performed. Results: Most reimbursement recommendations for orphan drugs were positive (71%), while approximately 17% were negative and almost 13% were conditional. The highest percentage of positive reimbursement recommendations was observed in Spain (97%) and France (95%) and the highest percentage of negative reimbursement recommendations was revealed for Poland (49%). On average, 65% of the 163 analyzed orphan drugs were reimbursed from public funds. The highest number of reimbursed orphan drugs was observed in Germany (n = 148), while the lowest, in Poland (n = 41). Considering all analyzed drugs, the highest agreement between recommendations and reimbursement status was observed for Spain (value of 1), and the lowest, for Germany (? = -0.03). Conclusions: On average, more than 60% of identified orphan drugs were reimbursed from public funds in the included countries, and the majority of reimbursement recommendations were found to be positive. The agreement between reimbursement recommendations and reimbursement status differed between the countries, but overall, it did not show any patterns, as it ranged from -0.03 to 1 (? coefficient).

SUBMITTER: Stawowczyk E

PROVIDER: S-EPMC6890830 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

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Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries.

Stawowczyk Ewa E Malinowski Krzysztof Piotr KP Kawalec Paweł P Bobiński Rafał R Siwiec Jacek J Panteli Dimitra D Eckhardt Helene H Simoens Steven S Agusti Antònia A Dooms Marc M Pilc Andrzej A

Frontiers in pharmacology 20191127

<b>Objective:</b> To review the reimbursement recommendations issued by selected European health technology assessment agencies for orphan drugs and the reimbursement status of these drugs; to assess the relationship between the type of recommendation and reimbursement status. <b>Methods:</b> The list of orphan drugs to be included in the analysis was obtained from the European Medicines Agency and Orphanet. Seven European states were included in the analysis: Belgium, England, France, Germany, ...[more]

PMID: 31827433

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Project description:Objectives: The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications. Materials and methods: A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries). A descriptive and statistical analysis was conducted to determine statistical significance, correlations, between the drug or country characteristic and the positive recommendation or reimbursement of ODs. Results: The proportion of reimbursed orphan drugs differed between countries, ranging from 17.7% in Estonia to 49.6% in Hungary (p < 0.001). The odds that ODs were reimbursed were reduced in countries with a "strong" level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), the presence of other additional specific clinical aspects (e.g., genomic data) considered in the reimbursement decision (p < 0.001) and mandatory (without exception) safety assessments (p=0.004). The probability that ODs were reimbursed was increased in countries with a "moderate" level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), when reimbursement decisions are dependent on the EMA registration status and orphan drug designation (p < 0.001), the presence of the "positive HTA recommendation guarantees reimbursement" policy (p < 0.001), higher GDP per inhabitant (p=0.003), and higher healthcare expenditure (p < 0.001). Conclusion: We found that there are differences among CEE countries in the reimbursement of orphan drugs, and we identified aspects that may influence these differences. Safety, efficacy, and specific clinical aspect issues significantly influenced reimbursement decisions. Antineoplastic and immunomodulating agents drugs were the largest group of ODs and increased the chance of getting a positive recommendation. The higher GDP per inhabitant and healthcare expenditures per inhabitant were positively linked to the chance that an OD receives reimbursement.

Project description:BackgroundThe Canadian government has committed to developing a national strategy for drugs for rare diseases starting in 2022. Considering this announcement, we conducted a comparative analysis to examine patient access to therapies for rare disease in Canada relative to Europe and the U.S.MethodsGiven its similarity to the Canadian health care system, we used Europe as the reference point to analyze all of the therapies with an orphan drug designation approved by the European Medicine Agency (EMA) from 1 January 2015 to 31 March 2020. We then contrasted access to these drugs in Canada (Health Canada) and the U.S. (Food and Drug Administration, FDA). We focused on: (1) the number of therapies for rare diseases entering the Canadian market; (2) the percentage of these therapies that are publicly available to Canadians; and (3) the timelines for patients to access these therapies in Canada.ResultsSixty-three approved therapies with an orphan drug designation from the EMA were identified. Fifty-three (84%) of these drugs had also been submitted to the FDA for approval, and 41 (65%) were submitted to Health Canada for approval. In Europe, Germany, Denmark, and the U.K. had the highest percentage of publicly reimbursed orphan drugs (84%, 70%, 68%, respectively). In comparison, Ontario (32%), Quebec (25%), and Alberta (25%) had the highest percentage of drugs reimbursed among the Canadian provinces. The shortest median duration (in months) from EMA approval to jurisdictional decision on reimbursement was in Austria (3.2), followed by Germany (4.1), and Finland (6.0). In Canada, the shortest median duration (in months) from regulatory approval to reimbursement was in British Columbia (17.3), Quebec (19.6) and Manitoba (19.6), while the longest duration was in P.E.I (38.5), followed by Nova Scotia (25.9), and Newfoundland (25.1).ConclusionsOur comparative analysis found that relative to the EU Canadians had less frequent and timely access to therapies for rare diseases. This highlights the need for a rare disease strategy in Canada that allows for clear identification and transparent tracking of the pathway for rare disease drugs, and ultimately optimizes the number of patients with access to these therapies.

Project description:BackgroundThe prospective reimbursement of hospitals through the grouping of patients into a finite number of categories (Diagnosis Related Groups, DRGs), is common to many European countries. However, the specific categories used vary greatly across countries, using different characteristics to define group boundaries and thus those characteristics which result in different payments for treatment. In order to assist in the construction and modification of national DRG systems, this study analyses the DRG systems of 10 European countries.AimsTo compare the characteristics used to categorise patients receiving a coronary artery bypass graft (CABG) surgery into DRGs. Further, to compare the structure into which DRGs are placed and the relative price paid for patients across Europe.MethodPatients with a procedure of CABG surgery are analysed from Austria, England, Estonia, Finland, France, Germany, Ireland, Poland, Spain and Sweden. Diagrammatic algorithms of DRG structures are presented for each country. The price in Euros of seven typical case vignettes, each made up of a set of a hypothetical patient's characteristics, is also analysed for each country. In order to enable comparisons across countries the simplest case (index vignette) is taken as baseline and relative price levels are calculated for the other six vignettes, each representing patients with different combinations of procedures and comorbidities.ResultsEuropean DRG payment structures for CABG surgery vary in terms of the number of different DRGs used and the types of distinctions which define patient categorisation. Based on the payments given to hospitals in different countries, the most resource intensive patient, relative to the index vignette, ranges in magnitude from 1.37 in Poland to 2.82 in Ireland. There is also considerable variation in how much different systems pay for particular circumstances, such as the occurrence of catheterisation or presence of comorbidity.ConclusionPast experience of the construction of DRG systems for CABG patients demonstrates the variety of options available. It also highlights the importance of updating systems as frequently as possible, to incentivise best practice.

Project description:ObjectivesWe analysed the information on current national physical activity recommendations in all EU Member States provided by governments in a joint EU/WHO survey on the implementation status of the EU Council Recommendation on Health-Enhancing Physical Activity across Sectors.DesignCross-sectional survey.ParticipantsThe representatives of the 28 EU Member State governments to the EU Physical Activity Focal Point Network.Outcome measuresNational recommendations on: (A) minimum frequency, duration, intensity and lengths of bouts of physical activity, (B) preventing inactivity or sedentary behaviour and (C) further recommendations for additional health benefits, obesity prevention and specific types of activity.ResultsAn official document could be located for 23 of the 28 EU Member States, while four are currently developing recommendations. For children and adolescents, most countries follow the 2010 WHO Global Recommendations for Physical Activity, but there are notable differences in the delimitation of age groups. 14 countries also followed WHO in their recommendations for adults, and 11 countries have additional advice on avoiding inactivity and sitting among adults. 18 Member States have recommendations for older adults, 12 of which follow WHO. Thirteen countries also address at least one special population (eg, pregnant women, people with disabilities and people with chronic diseases), but the level of detail varies substantially between countries.ConclusionsThe large majority of EU Member States either has physical activity recommendations in place or is in the process of developing them. There is a general tendency to use the WHO Global Recommendations as a basis, with the greatest variation observable for children and adolescents. Comparing results with a previous round of data collection shows that the number of EU countries with physical activity recommendations is increasing and that both special groups and sedentary behaviour have become more important in recent years.

Project description:BackgroundThe adoption of digital health care within health systems is determined by various factors, including pricing and reimbursement. The reimbursement landscape for digital health in Europe remains underresearched. Although various emergency reimbursement decisions were made during the COVID-19 pandemic to enable health care delivery through videoconferencing and asynchronous care (eg, digital apps), research so far has primarily focused on the policy innovations that facilitated this outside of Europe.ObjectiveThis study examines the digital health reimbursement strategies in 8 European countries (Belgium, France, Germany, Italy, the Netherlands, Poland, Sweden, and the United Kingdom) and Israel.MethodsWe mapped available digital health reimbursement strategies using a scoping review and policy mapping framework. We reviewed the literature on the MEDLINE, Embase, Global Health, and Web of Science databases. Supplementary records were identified through Google Scholar and country experts.ResultsOur search strategy yielded a total of 1559 records, of which 40 (2.57%) were ultimately included in this study. As of August 2023, digital health solutions are reimbursable to some extent in all studied countries except Poland, although the mechanism of reimbursement differs significantly across countries. At the time of writing, the pricing of digital health solutions was mostly determined through discussions between national or regional committees and the manufacturers of digital health solutions in the absence of value-based assessment mechanisms. Financing digital health solutions outside traditional reimbursement schemes was possible in all studied countries except Poland and typically occurs via health innovation or digital health-specific funding schemes. European countries have value-based pricing frameworks that range from nonexistent to embryonic.ConclusionsStudied countries show divergent approaches to the reimbursement of digital health solutions. These differences may complicate the ability of patients to seek cross-country health care in another country, even if a digital health app is available in both countries. Furthermore, the fragmented environment will present challenges for developers of such solutions, as they look to expand their impact across countries and health systems. An increased emphasis on developing a clear conceptualization of digital health, as well as value-based pricing and reimbursement mechanisms, is needed for the sustainable integration of digital health. This study can therein serve as a basis for further, more detailed research as the field of digital health reimbursement evolves.

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Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries.

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Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries.

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