Comparing access to orphan medicinal products in Europe

Zamora, Bernarda; Maignen, F.; O’Neill, P.; Mestre-Ferrandiz, J.; Garau, Martina

doi:10.1186/s13023-019-1078-5

Cited by 64 publications

(60 citation statements)

References 10 publications

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“…Since the 2011 introduction of legislation aiming at controlling prices of patented pharmaceuticals and to curb spending (Act to Reorganize the Pharmaceuticals' Market in the Statutory Health Insurance System, AMNOG) until March 1st 2017, 51 orphan drug reimbursement procedures have been finalized by Germany's Federal Joint Committee (Gemeinsame Bundesausschuss, G-BA) 31 . OMPs are most widely accessible in Germany and France (69).…”

Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

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A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Czech¹,

Baran-Kooiker

Atikeler

et al. 2020

Front. Public Health

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Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

“…France reimburses 116 orphan drugs, England 68, Scotland 55, and Wales 47 (65). England, <50% of centrally authorized OMPs are routinely funded by the NHS, with one-third of these recommended by NICE (69).…”

Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Czech¹,

Baran-Kooiker

Atikeler

et al. 2020

Front. Public Health

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“…The features included in supplemental processes aim to accelerate the HTA process to guarantee more timely access to patients to RDTs. Delays and variability in access to OMPs across Member States is one of the key issues in Europe [28,29]. Different mechanisms to grant earlier access to patients are being adopted.…”

Section: Some Countries Allow Exemptions From Submitting An Economicmentioning

confidence: 99%

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

Nicod

Whittal

Drummond

et al. 2020

Preprint

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BACKGROUND There is increasing recognition that conventional appraisal approaches may be unsuitable for assessing the value for money of rare disease treatments (RDTs). This research examines whether supplemental appraisal processes for RDTs are needed, and if so, what form should these take. A qualitative research design was used that included (1) documentation of country appraisal/reimbursement processes for RDTs via questionnaires, desk research and iterative interactions with country experts to produce country vignettes, and (2) a cross-country analysis of these processes to identify and characterise countries with supplemental processes for RDTs, and compare them to countries without supplemental processes. RESULTS Thirty-two of the 37 invited countries participated in this research. Forty-one percent (13/32) use supplemental processes for RDTs. Their level of integration within standard processes ranged from low to high, characterised by whether they are separate or partially separate from the standard process, adapted or accelerated standard processes, or standard processes that account for rarity. They are characterised by features implemented throughout the appraisal process. These features are mechanisms that allow application of different standards to assess the value of the medicine, support to the appraisal/decision-making process, overcome the issues of lack of cost-effectiveness, or exempt from part of/the full HTA process. They apply to ultra-RDTs and/or RDTs, and are often combined. They increase the likelihood of reimbursement by adjusting and/or foregoing part of the assessment process, or accepting to pay more for the same added benefit as for common conditions, which is likely to lead to accepting high prices. A large proportion of countries with standard processes include one or more of these features (formally or informally) or are discussing potential changes in their systems. CONCLUSIONS Results suggest revealed preferences to treat RDTs differently than conventional medicines. Some of the challenges around uncertainty and high price remain, but supplemental process features allow for more adapted and consistent decision-making. Many of these processes are new and countries continue to adjust as they gain experience. These results do not outline what an ideal process for RDTs looks like, but identify some key features that can be implemented to facilitate this process.

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“…A survey of access to OMPs in the United Kingdom, France, Germany, Italy, and Spain found that since the implementation of the OMP Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorization in the European Union. 4 These OMPs are most widely accessible in Germany and France, while in the other countries between 30 and 60% of OMPs are reimbursed. In addition, in some countries, such as Italy, differences in treatment availability and access to medicines, diagnosis, and care can occur regionally, depending on each regional health management system.…”

Section: Disparities In the Availability Of Omps In The Eumentioning

confidence: 99%

Access to Affordable Orphan Medicines in Europe: An EHA Position Paper

et al. 2020

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O rphan medicinal products (OMPs) are pharmaceuticals designed for the treatment of rare diseases, a family of more than 6000 medical conditions, each of which affects a small to ultra-small population of patientstypically less than 1 in 2000 people. 1 The June 2009 European Council Recommendation on action in the field of rare diseases emphasizes that "the principles and overarching values of universality, access to good quality care, equity and solidarity [...] are of paramount importance for patients with rare diseases." Although important advances have been made in the OMP field during the last decade, several deadlocks still impede optimal accessibility. 2 Disparities in the availability of OMPs in the EUA survey conducted in 12 Eurasian countries on rare disease policies and orphan drug reimbursement systems showed that inequality in patient access to new OMPs still exists due to variations in national healthcare budgets, health insurance, and reimbursement systems. 3 Fragmentation of reimbursement policies translates into unequal access to treatment. The number of reimbursed OMPs ranged from >100 in Germany, France, Italy, and the Netherlands to zero in Armenia. For instance, France reimburses 116 orphan drugs, England 68, Scotland 55, and Wales 47. A survey of access to OMPs in the United Kingdom, France, Germany, Italy, and Spain found that since the implementation of the OMP Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorization in the European Union. 4 These OMPs are most widely accessible in Germany and France, while in the other countries between 30 and 60% of OMPs are reimbursed. In addition, in some countries, such as Italy, differences in treatment availability and access to medicines, diagnosis, and care can occur regionally, depending on each regional health management system. The cost of OMPs is clearly a major factor explaining the disparities among different European countries. 5 The complex issue of drug pricing is dealt with in an accompanying European Hematology Association (EHA) position paper, 6 but here we will briefly consider the issues related to OMP prices. In addition, differences in normative approaches to rare diseases and OMPs between countries also play an important role.

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Comparing access to orphan medicinal products in Europe

Cited by 64 publications

References 10 publications

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

Access to Affordable Orphan Medicines in Europe: An EHA Position Paper

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