The evolution of adaptiveness: balancing speed and evidence

Eichler, Hans‐Georg; Barker, Richard; Bedlington, Nicola; Bouvy, Jacoline C.; Bucsics, Anna; Cerreta, Francesca; Corriol‐Rohou, Solange; Granados, Alı́cia; Cam, Yann Le; Schuurman, Ad

doi:10.1038/nrd.2018.90

Cited by 6 publications

(6 citation statements)

References 3 publications

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“…Coupling pricing with the level of available evidence for a positive healthcare effect following the example of the NICE framework could help health insurers to manage their risk of financial misinvestment. This could be achieved by using adaptive licensing models, 31 or making performance-based managed entry agreements in the form of a patient-level payment-by-result or a population-level coverage basis. 32 Combining the risk-based approach of the EU MDR 2017/745 to clinical investigations with patient-reported outcome measures and health economic impact could pave the way for a fair pricing of DiGA in value-based healthcare settings.…”

Section: Learnings and Future Prospectsmentioning

confidence: 99%

The German Fast Track Toward Reimbursement of Digital Health Applications: Opportunities and Challenges for Manufacturers, Healthcare Providers, and People With Diabetes

Schliess

Dicenzo

Gaus³

et al. 2022

J Diabetes Sci Technol

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Background: Digital health applications (DiGA) supporting the management of diabetes are among the most commonly available digital health technologies. However, transparent quality assurance of DiGA and clinical proof of a positive healthcare effect is often missing, which creates skepticism of some stakeholders regarding the usage and reimbursement of these applications. Methods: This article reviews the recently established fast-track integration of DiGA in the German reimbursement market, with emphasis on the current impact for manufacturers, healthcare providers, and people with diabetes. The German DiGA fast track is contextualised with corresponding initiatives in Europe. Results: The option of a provisional prescription and reimbursement of DiGA while proving a positive healthcare effect in parallel may expedite the adoption of DiGA in Germany and beyond. However, hurdles for a permanent prescription and reimbursement of DiGA are high and only one of 12 that have achieved this status specifically addresses people with diabetes. Conclusion: The DiGA fast track needs to be further enhanced to cope with remaining skepticism and contribute even more to a value-based diabetes care.

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Section: Learnings and Future Prospectsmentioning

confidence: 99%

The German Fast Track Toward Reimbursement of Digital Health Applications: Opportunities and Challenges for Manufacturers, Healthcare Providers, and People With Diabetes

Schliess

Dicenzo

Gaus³

et al. 2022

J Diabetes Sci Technol

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“…As discussed above, the ADAPT SMART Consortium has established a platform to facilitate and accelerate the availability of MAPPs to all healthcare stakeholders. MAPPs present stakeholders with an “evidence vs. access” conundrum; there are trade‐offs between ensuring rapid access to promising treatments for unmet urgent need, balanced with ensuring that patients, healthcare professionals, and other decision makers possess adequate information on the benefits and risks at the time of launch . Importantly, there is momentum behind the MAPPs concept with the ADAPT‐SMART Consortium identifying four key stages for stakeholder alignment: preclinical, preliminary evidence generation, prelaunch, and postlaunch …”

Section: Opportunities For Alignmentmentioning

confidence: 99%

The Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways

McAuslane

Liberti

Connelly

2019

Clin Pharma and Therapeutics

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There is a growing interest in aligning accelerated regulatory pathways with flexible access and reimbursement pathways to expedite the equitable availability of high-quality, safe, and effective medicines that provide a valuebased approach to meeting society's most important healthcare needs. The Centre for Innovation in Regulatory Science (CIRS) identified key issues regarding the confluence of regulatory and health technology assessment processes from discussions and presentations given by international regulators, health technology assessment bodies, payers, patient representatives, and multinational pharmaceutical company representatives on this topic at CIRS workshops held in 2014 and 2017 that focused on the commonalties and differences across these pathways. Recent publications have also been highlighted. The barriers to and opportunities for aligning stakeholder expectations and needs were investigated and recommendations provided. Early dialogue among the stakeholders is the process that will likely provide the greatest return on investment of time and effort to identify, develop, review, and recommend important new medicines, especially those that address an unmet medical need.

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“…The generation of epidemiological evidence is sometimes relevant to support the early approval of medicines. This highlights the need for reinforcement of those concepts, to enhance trust in postapproval epidemiological research …”

Section: Stakeholders' Opinionmentioning

confidence: 99%

“…This highlights the need for reinforcement of those concepts, to enhance trust in postapproval epidemiological research. 17,24 Trust building is further supported by increased transparency and all stakeholders is necessary to further strengthen transparency in postauthorisation research, a concept also pursued by the EU pharmacovigilance legislation. 19 New emphasis has been put on performing joint PASS encompassing studies performed by multiple marketing authorisation holders, which is also covered by the Code.…”

Section: Regulatory Perspectivementioning

confidence: 99%

The ENCePP Code of Conduct: A best practise for scientific independence and transparency in noninterventional postauthorisation studies

Gini

Fournie²,

Dolk

et al. 2019

Pharmacoepidemiology and Drug

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Purpose The ENCePP Code of Conduct provides a framework for scientifically independent and transparent pharmacoepidemiological research. Despite becoming a landmark reference, practical implementation of key provisions was still limited. The fourth revision defines scientific independence and clarifies uncertainties on the applicability to postauthorisation safety studies requested by regulators. To separate the influence of the funder from the investigator's scientific responsibility, the Code now requires that the lead investigator is not employed by the funding institution. Method To assess how the revised Code fits the ecosystem of noninterventional pharmacoepidemiology research in Europe, we first mapped key recommendations of the revised Code against ISPE Good Pharmacoepidemiology Practices and the ADVANCE Code of Conduct. We surveyed stakeholders to understand perceptions on its value and practical applicability. Representatives from the different stakeholders' groups described their experience and expectations. Results Unmet needs in pharmacoepidemiological research are fulfilled by providing unique guidance on roles and responsibilities to support scientific independence. The principles of scientific independence and transparency are well understood and reinforce trust in study results; however, around 70% of survey respondents still found some provisions difficult to apply. Representatives from stakeholders' groups found the new version promising, although limitations still exist. Conclusion By clarifying definitions and roles, the latest revision of the Code sets a new standard in the relationship between investigators and funders to support scientific independence of pharmacoepidemiological research. Disseminating and training on the provisions of the Code would help stakeholders to better understand its advantages and promote its adoption in noninterventional research.

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The evolution of adaptiveness: balancing speed and evidence

Cited by 6 publications

References 3 publications

The German Fast Track Toward Reimbursement of Digital Health Applications: Opportunities and Challenges for Manufacturers, Healthcare Providers, and People With Diabetes

The German Fast Track Toward Reimbursement of Digital Health Applications: Opportunities and Challenges for Manufacturers, Healthcare Providers, and People With Diabetes

The Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways

The ENCePP Code of Conduct: A best practise for scientific independence and transparency in noninterventional postauthorisation studies

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