A review of international coverage and pricing strategies for personalized medicine and orphan drugs

Degtiar, Irina

doi:10.1016/j.healthpol.2017.09.005

Cited by 28 publications

(32 citation statements)

References 81 publications

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“…Although recently developed drugs, including cell and gene therapy drugs, have optimal beneficial effects and minimal side effects, their timely delivery can be challenging; there may also be insufficient evidence regarding their efficacy because of the difficulties in recruiting sufficient participants for clinical trials. Moreover, because of the high prices of such drugs, decisions about reimbursement are made while considering a variety of factors, including incentives to improve treatment methods, financial resources available for health insurance, and access to treatment methods [9,10,11,12].…”

Section: Introductionmentioning

confidence: 99%

Improving Patient Access to New Drugs in South Korea: Evaluation of the National Drug Formulary System

Yoo

Kim

Lee

et al. 2019

IJERPH

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This study reviews and evaluates the national drug formulary system used to improve patient access to new drugs by making reimbursement decisions for new drugs as part of the South Korean national health insurance system. The national health insurance utilizes three methods for improving patient access to costly drugs: risk-sharing agreements, designation of essential drugs, and a waiver of cost-effectiveness analysis. Patients want reimbursement for new drugs to be processed quickly to improve their access to these drugs, whereas payers are careful about listing them given the associated financial burden and the uncertainty in cost-effectiveness. However, pharmaceutical companies are advocating for drug prices above certain thresholds to maintain global pricing strategies, cover the costs of drug development, and fund future investments into research and development. The South Korean government is expected to develop policies that will improve patient access to drugs with unmet needs for broadening health insurance coverage. Simultaneously, the designing of post-listing management methods is warranted for effectively managing the financial resources of the national health insurance system.

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Section: Introductionmentioning

confidence: 99%

Improving Patient Access to New Drugs in South Korea: Evaluation of the National Drug Formulary System

Yoo

Kim

Lee

et al. 2019

IJERPH

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“…The distinctive contribution is in the primary nature of the data collected from experts closely involved within these processes, which allow the first comprehensive and detailed review of the supplemental processes targeting RDTs in Europe and beyond. This issue has been previously explored through systematic reviews or case studies, however, these earlier findings, target a narrow sample of countries (with scarce information on Eastern European countries [6]), and focus on their evaluation criteria [6,[20][21][22], and/or on governance structures for pricing and reimbursement [22]. This study additionally identified and characterised the process features that facilitate appraisal of RDTs.…”

Section: Discussionmentioning

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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“…The distinctive contribution is in the primary nature of the data collected from experts closely involved within these processes, which allow the first comprehensive and detailed review of the supplemental processes targeting RDTs in Europe and beyond. This issue has been previously explored through systematic reviews or case studies, however, these earlier findings target a narrow sample of countries (with scarce information on Eastern European countries [ 6 ]), and focus on their evaluation criteria [ 6 , 22 – 24 ], and/or on governance structures for pricing and reimbursement [ 24 ]. This study additionally identified and characterised the process features that facilitate appraisal of RDTs.…”

Section: Discussionmentioning

confidence: 99%

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

Nicod

Whittal

Drummond

et al. 2020

Orphanet J Rare Dis

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Background: There is increasing recognition that conventional appraisal approaches may be unsuitable for assessing the value rare disease treatments (RDTs). This research examines what supplemental appraisal/reimbursement processes for RDTs are used internationally and how they can be characterised. 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 crosscountry analysis of these processes to identify and characterise features in 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 may be applied to RDTs. 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 appraisal/reimbursement process. 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. 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 can support decision-making that is more flexible and consistent. Many of these processes are new and countries continue to adjust as they gain experience.

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A review of international coverage and pricing strategies for personalized medicine and orphan drugs

Cited by 28 publications

References 81 publications

Improving Patient Access to New Drugs in South Korea: Evaluation of the National Drug Formulary System

Improving Patient Access to New Drugs in South Korea: Evaluation of the National Drug Formulary System

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

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

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