Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?

Eichler, Hans‐Georg; Bedlington, Nicola; Boudes, Mathieu; Bouvy, Jacoline C.; Cerreta, Francesca; Faulkner, Stuart; Forda, Susan R.; Joos, Angelika; Cam, Yann Le; Mayer, Mark H.; Pirard, Vinciane; Corriol‐Rohou, Solange

doi:10.1002/cpt.1121

Cited by 7 publications

(4 citation statements)

References 9 publications

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“…The ability for PE to improve research is only as good as the way it is defined and operationalised [22]. While there is broad agreement that PE across all stages of research should be impactful, meaningful and measurable [5,6,11,[23][24][25][26], a number of terms such as patient engagement, patient involvement, or patient centredness have been used interchangeably by some, whilst others see subtle differences in terminology and context to be important. Some have argued that this led to the inappropriate and incomplete application of the underlying principles of meaningful PE [22,27] and slowed progress.…”

Section: A Matter Of Terminologymentioning

confidence: 99%

Using Patient Perspectives to Inform Better Clinical Trial Design and Conduct: Current Trends and Future Directions

Faulkner¹,

Somers²,

Boudes³

et al. 2023

Pharm Med

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The approach to patient engagement (PE) in drug development has changed rapidly due to many factors, including the complexity of innovative drugs and the need to demonstrate outcomes of relevance to patients, the desire to show ‘value add’ of PE, and the pandemic-related changes to how clinical trials are run, e.g., decentralised studies. In parallel, there have been changes in technology-assisted ways of running clinical trials, capturing patient health outcomes and preferences, an increasing societal demand for diversity and inclusion, and efforts to improve clinical trial efficiency, transparency, and accountability. Organisations are beginning to monitor PE activities and outcomes more effectively to learn and inform future PE strategies. As a result, these factors are facilitating the incorporation of patients’ lived experience, preferences and needs into the design and running of clinical trials more than ever before. In this paper, the authors reflect upon these last few years, the emerging trends and their drivers, and where we may expect PE in clinical research to progress in the near future.

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Section: A Matter Of Terminologymentioning

confidence: 99%

Using Patient Perspectives to Inform Better Clinical Trial Design and Conduct: Current Trends and Future Directions

Faulkner¹,

Somers²,

Boudes³

et al. 2023

Pharm Med

Self Cite

View full text Add to dashboard Cite

show abstract

“…However, an EMA official cited the case of alemtuzumab (Lemtrada), a high‐cost multiple sclerosis drug, as a case study in how even under conventional regulatory procedures, the argument for cost‐effectiveness of a drug can evolve over time . As the EMA seems committed to the Adaptive Pathways program, these issues will need to be addressed by HTAs and payers . It also noted that the Adaptive Pathways program and PRIME involve discussions with payers in which the payer evidentiary needs can be highlighted.…”

Section: Novel Pathways For Faster Drug Accessmentioning

confidence: 99%

Innovation in Regulatory Science Is Meeting Evolution of Clinical Evidence Generation

Lee

Möller³

et al. 2019

Clin Pharma and Therapeutics

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At the turn of the century, the pharmaceutical industry began a transition toward a focus on oncology, rare diseases, and other areas of high unmet need that required a new, more complex approach to drug development. For many of these disease states and novel approaches to therapy, traditional approaches to clinical trial design fall short, and a number of innovative trial designs have emerged. In light of these changes, regulators across the globe are implementing new programs to provide regular development program support, facilitate accelerated access, use real‐world data, and use digital tools to improve patients’ lives. Emerging market regulators are also focusing on simplifying their regulatory pathways via regional harmonization schemes with varying levels of ambition. These changes in the external environment imply that biopharma regulatory teams need to adapt and evolve, leveraging digital tools, data, and analytics, and positioning themselves as strategic advisors during development.

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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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Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?

Cited by 7 publications

References 9 publications

Using Patient Perspectives to Inform Better Clinical Trial Design and Conduct: Current Trends and Future Directions

Using Patient Perspectives to Inform Better Clinical Trial Design and Conduct: Current Trends and Future Directions

Innovation in Regulatory Science Is Meeting Evolution of Clinical Evidence Generation

The Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways

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