Pcp56 - Early Access Programs: Recommendations for Real-World Data Collection

Stein, David; Soni, Mayuri Ganguly

doi:10.1016/j.jval.2018.09.531

Cited by 1 publication

(1 citation statement)

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“…The concept has, however, been renamed to "adaptive pathways (APs)" to better reflect a focus on the development and managed introduction of medicines rather than a new way of regulating and authorizing medicines (83). Moreover, over the past years, regulators in different jurisdictions have had several mechanisms in place to facilitate an earlier access of new promising medicines especially in areas of high unmet medical needs and for orphan diseases (84). In Europe, the EMA early access and registration tools include Priority Medicine (PRIME) (85), conditional marketing, and authorization and approval under exceptional circumstances (86), as well as compassionate use exist (87).…”

Section: Adaptive Licensing Pathwaysmentioning

confidence: 99%

Improving Interactions Between Health Technology Assessment Bodies and Regulatory Agencies: A Systematic Review and Cross-Sectional Survey on Processes, Progress, Outcomes, and Challenges

2020

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The need to optimize drug development and facilitate faster access for patients has ignited discussions around the importance of improving interactions between health technology assessment (HTA) bodies and regulatory agencies. In this study, we conducted a systematic review to examine processes, progress, outcomes, and challenges of harmonization/interaction initiatives between HTA bodies and regulatory agencies. MEDLINE, EMBASE, and the International Pharmaceutical Abstracts database were searched up to 21 October 2019. Searches for gray literature (working papers, commissioned reports, policy documents, etc.) were performed via Google scholar and several institutional websites. An online cross-sectional survey was also conducted among HTA (n = 22) and regulatory agencies (n = 6) across Europe to supplement the systematic review. Overall, we found that while there are areas of divergence, there has been progress over time in narrowing the gap in evidentiary requirements for HTA bodies and regulatory agencies. Most regulatory agencies (4/6; 67%) and half (11/22, 50%) of the HTA bodies reported having a formal link for "collaborating" with the other. Several mechanisms such as early tripartite dialogues, parallel submissions (reviews), adaptive licensing pathways, and postauthorization data generation have been explored as avenues for improving collaboration. A number of pilot initiatives have shown positive effects of these models to reduce the time between regulatory and HTA decisions, which may translate into faster access for patients to life-saving therapies. Thus, future approaches aimed at improving harmonization/interaction between HTA bodies and regulatory agencies should build on these existing models/mechanisms while Ofori-Asenso et al. Improving HTA-Regulatory Interactions examining their long-term impacts. Several barriers including legal, organizational, and resource-related factors were also identified, and these need to be addressed to achieve greater alignment in the current regulatory and reimbursement landscape.

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Section: Adaptive Licensing Pathwaysmentioning

confidence: 99%