Php293 - Orphan Drugs in Germany - Lessons Learned From Amnog, Best and Worst Practices and Strategic Implications

Kirchmann, T.; Ewald, Ariane Patrícia; Schönermark, Matthias P.

doi:10.1016/j.jval.2018.09.1187

Cited by 5 publications

(4 citation statements)

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“…If the drug has additional clinical benefits compared with standard treatments, the drug’s manufacturer has 6 months to negotiate a price with the Federal Association of Sickness Funds—a nongovernmental body similar to America's Health Insurance Plans. The price premium is supposed to reflect the added therapeutic value of the drug as determined by the Federal Joint Committee, which generally assesses orphan drugs as having a nonquantifiable additional benefit . If no agreement is reached after 6 months of negotiations, an independent arbitration body has 3 months to determine a price.…”

Section: Germanymentioning

confidence: 99%

Drug Reimbursement Regulation in 6 Peer Countries

et al. 2020

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Section: Germanymentioning

confidence: 99%

Drug Reimbursement Regulation in 6 Peer Countries

et al. 2020

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“…This is seen in Germany where a simplified submission without comparative data is permissible for OMPs as added benefit is automatically assumed. However, those drugs for which no comparative data is presented are likely to be categorised as "non-quantifiable" added benefit, which may prove to be more challenging when it comes to negotiations around price [23,24].…”

Section: The (Add-on or Adjusted) Features Adopted Allow For Differenmentioning

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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“…For example, no comparative data are required for OMPs in Germany as their added benefit is assumed to be proven. However, some manufacturers may choose not to use this route as those medicines for which there are no comparative data are likely to be categorised with "non-quantifiable" added benefit, which may render price negotiations more challenging [25,26].…”

Section: Different Standardsmentioning

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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Php293 - Orphan Drugs in Germany - Lessons Learned From Amnog, Best and Worst Practices and Strategic Implications

Cited by 5 publications

References 0 publications

Drug Reimbursement Regulation in 6 Peer Countries

Drug Reimbursement Regulation in 6 Peer Countries

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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