Background: Despite increasing informal and formal use of unmet medical need (UMN) in drug development, regulation, and assessment, there is no insight into its definitions in use. This study aims to provide insight into the current definitions in use and to provide a starting point for a multi-stakeholder discussion on alignment. Methods: A scoping and a gray literature review were performed to locate definitions of UMN in literature and on stakeholder websites. These definitions were categorized and then discussed among the multi-stakeholder author group via semistructured group discussions and open session workshops with a broader stakeholder audience. Issues with the formation of a common definition and mechanisms for use were discussed. Results: The reviews yielded 16 definitions. Differences were evident, but all included 1 or more of the following elements: (adequacy of) available treatments (16 of 16: 100%), disease severity or burden (6 of 16: 38%), and patient population size (1 of 16: 6%). The stakeholder discussions led to a suggestion for a definition including the first 2 items and, depending on context, population size. The discussions also showed that quantification of UMN is highly dependent on the scope and the value framework in which it is used based on different stakeholder preferences and responsibilities. Conclusion: We encourage stakeholders that want to promote alignment on the concept of UMN to prospectively discuss the scope in which they want to apply the concept, what elements they find important for consideration in each case, and how they would measure UMN within the broader regulatory or value framework applicable.
Aims
Guidelines establish a framework for how therapeutics and vaccines are developed, assessed and approved. They influence which innovations are likely to be approved in the European Union (EU), and thus, they have an impact on the pipeline decisions taken by research‐based industry. This study analyses the level of acceptance for changes suggested by stakeholders within the authoring groups at the European Medicines Agency (EMA).
Methods
We looked at 87 guidelines from EMA Working Parties (WPs) launched for consultation between 2013 and 2017. Acceptance of stakeholder proposals and the time between the end of consultation and guideline adoption were studied as well as the openness of different WPs to accept changes.
Results
Adoption of a guideline after the close of public consultation took at least 4 months, with an average of 12–16 months. The number of accepted and rejected comments were nearly equal across the stakeholders, with government having slightly higher chance for acceptance. Academia and nongovernmental organizations (NGOs) had generally higher chances to have their comments accepted for general and indication‐level guidelines. Government and individual companies had highest acceptance for molecule‐level guidelines and trade associations for indication‐level guidelines. Only a third of the comments related to scientific content of the guideline.
Conclusion
This pattern of progress in regulatory science at EMA demonstrates the essential and interrelated role of academia, industry, government and civil society—described as the quadruple helix model—to promote establishment of a strong innovation ecosystem in Europe.
Aim: Guidelines establish a framework for how therapeutics and vaccines
are developed, assessed and approved. They influence which innovations
are likely to be approved in the EU, and by that, they have an impact on
the pipeline decisions taken by the research-based industry. This study
analyses the level of acceptance for changes suggested by stakeholders
within the authoring groups at the EMA. Methods: We looked at 87
guidelines from EMA Working Parties (WPs) launched for consultation
between 2013-2017. Acceptance of stakeholder proposals and the time
between the end of consultation and guideline adoption were studied as
well as the openness of different Working Parties to accept changes.
Results: Adoption of a guideline after the close of public consultation
took at least 4 months, with average 12-16 months. The number of
accepted and rejected comments were nearly equal across the
stakeholders, with government having slightly higher chance for
acceptance. Academia and NGOs had generally higher chances to have their
comments accepted for general and indication-level guidelines.
Government and individual companies had highest acceptance for
molecule-level guidelines and trade associations for indication-level
guidelines. The EMA WPs working with emerging technologies were more
open to accept proposed changes. Conclusion: This pattern of progress in
regulatory science at EMA demonstrates the essential and interrelated
role of academia, industry, government and civil society – described as
the quadruple helix model - to promote establishment of a strong
innovation ecosystem in Europe. Further integration and utilisation of
competences of each stakeholder is necessary for guideline development.
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