The Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways

McAuslane, Neil; Liberti, Lawrence; Connelly, Patricia

doi:10.1002/cpt.1315

Cited by 10 publications

(14 citation statements)

References 14 publications

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“…Such trust is key both from patients to understand the decisionmaking process, as well as from other stakeholders particularly in the case of agencies aligning their decision-making processes. 5,6 In addition, the routine assessment of the QDMPs has the ability to measure change over time in order to determine the impact of any improvement initiatives. The ongoing use of quality frameworks for making decisions will also reduce uncertainty around decision-making and might result in more predictable and favourable outcomes.…”

Section: Resultsmentioning

confidence: 99%

“…This applies to both how individuals decisions on a daily basis, as well as how key strategic decisions are made across a range of organisations including the pharmaceutical industry. 6 A recent review of the literature identified general paucity of research in this area, particularly regarding the development and systematic application of techniques for evaluating quality decision-making, such as through the studies undertaken as part of this research. 9 Indeed this is the first assessment to evaluate quality of decision-making within the different organisations involved in the development, review and reimbursement of medicines.…”

Section: Discussionmentioning

confidence: 99%

“…This is particularly important with the growing trend and efforts to align regulatory and HTA standards and processes both within companies as well as across agencies, in order to further increase effectiveness and efficiency during the development, review and reimbursement of medicines. 5,6 This gap in research into decision-making during medicines development was initially addressed through the research of Donelan and colleagues, which resulted in defining the best practices in decision-making during the lifecycle of medicines, namely the 10 Quality Decision-Making Practices (QDMPs) ( Figure 1). Donelan et al also developed an instrument, the Quality of Decision-Making Orientation Scheme (QoDoS), which can be used to measure the incorporation of these 10 QDMPs into the operational processes of companies and agencies.…”

Section: Introductionmentioning

confidence: 99%

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A Process for Evaluating Quality Decision-Making Practices During the Development, Review and Reimbursement of Medicines

Bujar

McAuslane

Walker

et al. 2020

Int J Health Policy Manag

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Background: The development of a medicine is not only underpinned by good science but also by Quality DecisionMaking Practices (QDMPs). Indeed, it is important to ensure that all organisations involved in the lifecycle of medicines are aligning their practices in decision-making to the QDMPs to ensure quality, transparent and consistent decisionmaking processes. Methods: The aim of this study was to evaluate the practicality of QoDoS (Quality of Decision-Making Orientation Scheme) in assessing the incorporation of ten QDMPs during the development, review and reimbursement of medicines, illustrated by case studies with a pharmaceutical company, a regulatory authority and a health technology assessment (HTA) agency. Individuals from each organisation completed the 47-item QoDoS questionnaire. Results: The results demonstrate the applicability of QoDoS in identifying favourable and unfavourable practices and in assessing the consistency and transparency of the QDMPs within each organisation, as well as across the different stakeholders. Furthermore, the study established the value of the methodology in raising awareness of the biases and best practices in decision-making, as well as having a basis for discussion for differences within and across stakeholders to promote consistency and alignment in decision-making. Finally, the QoDoS demonstrated the need for improvement across a number of decision-making practices for the 3 organisations such as the evaluation of alternatives and of the decision impact. Conclusion: The QoDoS can be used to benchmark organisations’ decision-making practices to provide a basis for discussion to ultimately encourage a level of trust across and within organisations and helping to identify areas for improvement.

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Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

A Process for Evaluating Quality Decision-Making Practices During the Development, Review and Reimbursement of Medicines

Bujar

McAuslane

Walker

et al. 2020

Int J Health Policy Manag

Self Cite

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“…The company perception may be as a result of high uncertainty around the effectiveness of such treatments that HTA agencies and payers may be concerned to accept [18,19]. This also highlights the need to better align accelerated regulatory pathways with flexible access and reimbursement pathways to expedite the equitable availability of high-quality, safe, and effective medicines that provide a value-based approach to meeting society's most important healthcare needs [20].…”

Section: Discussionmentioning

confidence: 99%

The Qualitative Value of Facilitated Regulatory Pathways in Europe, USA, and Japan: Benefits, Barriers to Utilization, and Suggested Solutions

2021

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Background Despite the growing application of facilitated regulatory pathways (FRPs), little attention has focused on assessing the perception of pharmaceutical companies regarding their usefulness beyond increasing timeliness. Objectives The aim of this study was to characterize the perceived value of four key FRPs, based on industry experiences in using these pathways. In addition, we sought to characterize the perceived impact based on benefits and barriers as well as suggested solutions for their use and recommendations as identified by companies, to outline how these FRPs may be further evolved as tools for expediting the development and regulatory review of important medicines. Methods A study was undertaken to characterize the perceived value and impact of US FDA (i.e., Breakthrough Therapy Designation, Fast Track), European Medicines Agency (i.e., PRIME), and Japanese Pharmaceutical and Medical Devices Agency (i.e., Sakigake) FRPs through a comprehensive analysis of strengths, weaknesses, opportunities, and threats (SWOT) as well as suggested solutions based on industry experiences with their use. The finalized survey comprised six questions and was sent to senior management in regulatory affairs departments at 22 multinational pharmaceutical companies in March 2019, with a deadline for completion by April 2019. The responses were analyzed using descriptive statistics. SWOT and free-text responses were reviewed and manually grouped into key themes according to high concordance. Results Survey results were returned by 11 pharmaceutical companies. Based on their perceived value and positive impact, the evaluated FRPs seem to be generally recognized as helpful tools for ensuring timely development and review of important medicines while ensuring multistakeholder involvement. Respondents overwhelmingly felt that the Breakthrough Therapy Designation carried a positive influence, both within and outside their organizations. Following closely with a positive although varied perception was Sakigake, but respondents exhibited more ambivalence about Fast Track and PRIME. Companies felt the impact of the FRPs was generally positive for most stakeholders except for health technology assessors/payers, highlighting the need to better align FRPs with flexible access and reimbursement pathways to expedite the equitable availability of high‐quality, safe, effective medicines. Conclusions This study highlighted common recommendations across all four FRPs (relating to resource optimization, education, alignment, and communication to improve effective use), as well as agency-specific recommendations, some of which are already being addressed by the regulators. Supplementary Information The online version contains supplementary material available at 10.1007/s40290-020-00372-7.

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“…Furthermore, there are concerns that the role of HTA agencies as final gatekeepers may be compromised through early involvement with developers. However, according to McAuslane et al, if the current challenges with early dialogue are properly addressed, it "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" (67).…”

Section: Tripartite Early Dialoguesmentioning

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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The Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways

Cited by 10 publications

References 14 publications

A Process for Evaluating Quality Decision-Making Practices During the Development, Review and Reimbursement of Medicines

A Process for Evaluating Quality Decision-Making Practices During the Development, Review and Reimbursement of Medicines

The Qualitative Value of Facilitated Regulatory Pathways in Europe, USA, and Japan: Benefits, Barriers to Utilization, and Suggested Solutions

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

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