Regulatory Science to 2025: An Analysis of Stakeholder Responses to the European Medicines Agency's Strategy

Hines, Philip A.; González‐Quevedo, Rosa; Lambert, Apolline I O M; Janssens, Rik; Freischem, Barbara; Edo, Jordi Torren; Claassen, Ivo J T M; Humphreys, A. J. B.

doi:10.3389/fmed.2020.00508

Cited by 15 publications

(11 citation statements)

References 7 publications

(9 reference statements)

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“…Patient preference studies use qualitative and quantitative methods to understand which treatment and disease-related characteristics (efficacy outcomes, side-effects, and symptoms) are important to patients, how important they are to patients, the trade-offs patients are willing to make between these characteristics, and how preferences may vary according to individual patient characteristics (1)(2)(3). Stakeholders involved in drug development and evaluation-such as drug developers, regulators, Health Technology Assessment (HTA) bodies and payers-have acknowledged the potential value of using patient preference studies to inform their respective decisions (4)(5)(6). More specifically, patient preference studies could: (i) reveal the patient perspective on unmet treatment needs in early drug development, (ii) inform the development of patient reported outcome measures and the selection of clinical trial endpoints, (iii) help understand which are the key favorable and unfavorable effects and uncertainties in regulatory benefit-risk assessment, (iv) quantify the relative importance of treatment characteristics in HTA and payer decisions, and (v) inform the development of decision aids used in shared individual treatment decisionmaking between patients and clinicians (1,(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)(16)(17)(18).…”

Section: Introductionmentioning

confidence: 99%

“…On the regulatory level, the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) intend to systematically include preference studies in regulatory benefit-risk assessment (4)(5)(6)(19)(20)(21). Mirroring the FDA's efforts toward guidance surrounding patient preference studies, the EMA aims to develop guidance on appropriate methods for patient preference study design, conduct, analysis, and presentation for regulatory purposes, to ensure that high quality methodologies are applied (4).…”

Section: Introductionmentioning

confidence: 99%

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Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

et al. 2021

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Background: Investigational and marketed drugs for the treatment of multiple myeloma (MM) are associated with a range of characteristics and uncertainties regarding long term side-effects and efficacy. This raises questions about what matters most to patients living with this disease. This study aimed to understand which characteristics MM patients find most important, and hence should be included as attributes and levels in a subsequent quantitative preference survey among MM patients.Methods: This qualitative study involved: (i) a scoping literature review, (ii) discussions with MM patients (n = 24) in Belgium, Finland, Romania, and Spain using Nominal Group Technique, (iii) a qualitative thematic analysis including multi-stakeholder discussions.Results: MM patients voiced significant expectations and hopes that treatments would extend their lives and reduce their cancer signs and symptoms. Participants however raised concerns about life-threatening side-effects that could cause permanent organ damage. Bone fractures and debilitating neuropathic effects (such as chronic tingling sensations) were highlighted as major issues reducing patients' independence and mobility. Patients discussed the negative impact of the following symptoms and side-effects on their daily activities: thinking problems, increased susceptibility to infections, reduced energy, pain, emotional problems, and vision problems. MM patients were concerned with uncertainties regarding the durability of positive treatment outcomes, and the cause, severity, and duration of their symptoms and side-effects. Patients feared short-term positive treatment responses complicated by permanent, severe side-effects and symptoms.Conclusions: This study gained an in-depth understanding of the treatment and disease-related characteristics and types of attribute levels (severity, duration) that are most important to MM patients. Results from this study argue in favor of MM drug development and individual treatment decision-making that focuses not only on extending patients' lives but also on addressing those symptoms and side-effects that significantly impact MM patients' quality of life. This study underscores a need for transparent communication toward MM patients about MM treatment outcomes and uncertainties regarding their long-term efficacy and safety. Finally, this study may help drug developers and decision-makers understand which treatment outcomes and uncertainties are most important to MM patients and therefore should be incorporated in MM drug development, evaluation, and clinical practice.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

et al. 2021

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“…Each methodology has value and addresses different needs. Together these activities will help to further develop and strengthen the patient voice in regulatory procedures, which is further reinforced by the recommendations in the EMA Regulatory Science to 2025 ( 15 ) and comments received during the public consultation ( 16 ).…”

Section: Discussionmentioning

confidence: 95%

The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study

et al. 2022

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The European Medicines Agency provides Scientific Advice to medicines developers and patient input has been an integral part of this process for many years. As end users of medicines, patients bring their perspectives to many different processes along EMA's regulatory pathway, complementing the scientific expertise. While the value of including patients has been well-demonstrated over the years, requests for evidence of their impact continue. Using Scientific Advice as a case study, data was collected over a four-year period to assess the number of patients involved, where they contributed, as well as the impact and added value of their input. In this paper, we show that patients' contributions have a tangible impact on the recommendations provided to developers and in over half of the cases, this led to further discussion on relevant patient perspectives. These data provide quantitative evidence of the value of patient input in medicines development and supports EMA's continued inclusion of their voice throughout the medicine's lifecycle.

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“…The most important feature of future UK regulation identified by survey participants was flexibility (Table 2 )—the capability of regulations to adapt to novel products and target patient outcomes facilitated through cooperation between relevant agencies, which is essential to enable some element of regulatory science future-proofing [ 9 ]. This mirrors international trends supporting increased globalised development of health products through collaborative working across international regulatory agencies.…”

Section: Discussionmentioning

confidence: 99%

“…These come with vast opportunities but also some significant risks to users, innovators, and payers [ 8 ]. However, through the development of regulatory and scientific strategy, planning and governance regulatory science can be future-proofed [ 9 ]. Secondly, post-Brexit, the UK intends to separate from EU regulations at the end of the transition period, 31 December 2020.…”

Section: Introductionmentioning

confidence: 99%

Advancing UK Regulatory Science Strategy in the Context of Global Regulation: a Stakeholder Survey

Rivera

Torlińska

Marston

et al. 2021

Ther Innov Regul Sci

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Background The UK’s transition from the European Union creates both an urgent need and key opportunity for the UK and its global collaborators to consider new approaches to the regulation of emerging technologies, underpinned by regulatory science. This survey aimed to identify the most accurate definition of regulatory science, to define strategic areas of the regulation of healthcare innovation which can be informed through regulatory science and to explore the training and infrastructure needed to advance UK and international regulatory science. Methods A survey was distributed to UK healthcare professionals, academics, patients, health technology assessment agencies, ethicists and trade associations, as well as international regulators, pharmaceutical companies and small or medium enterprises which have expertise in regulatory science and in developing or applying regulation in healthcare. Subsequently, a descriptive quantitative analyses of survey results and directed thematic analysis of free-text comments were applied. Results Priority areas for UK regulatory science identified by 145 participants included the following: flexibility: the capability of regulations to adapt to novel products and target patient outcomes; co-development: collaboration across sectors, e.g. patients, manufacturers, regulators, and educators working together to develop appropriate training for novel product deployment; responsiveness: the preparation of frameworks which enable timely innovation required by emerging events; speed: the rate at which new products can reach the market; reimbursement: developing effective tools to track and evaluate outcomes for “pay for performance” products; and education and professional development. Conclusions The UK has a time-critical opportunity to establish its national and international strategy for regulatory science leadership by harnessing broader academic input, developing strategic cross-sector collaborations, incorporating patients’ experiences and perspectives, and investing in a skilled workforce.

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Regulatory Science to 2025: An Analysis of Stakeholder Responses to the European Medicines Agency's Strategy

Cited by 15 publications

References 7 publications

Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study

Advancing UK Regulatory Science Strategy in the Context of Global Regulation: a Stakeholder Survey

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