European Medicines Agency’s Priority Medicines Scheme at 2 Years: An Evaluation of Clinical Studies Supporting Eligible Drugs

Neez, Emilie; Hwang, Thomas J.; Sahoo, Samali Anova; Naci, Huseyin

doi:10.1002/cpt.1669

Cited by 11 publications

(11 citation statements)

References 25 publications

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“…66 Complicating matters further is the regulatory agencies' increasing use of terms like "breakthrough therapies" in the US and "priority medicines" in Europe to refer to products in expedited programs. 67 In a randomised survey study among US adults, labeling a drug as a "breakthrough" altered people's planned behaviour and increased their positive perceptions in the drug's effectiveness. 68…”

Section: A Fragmented Evidence Base For Decision Making In Health Sysmentioning

confidence: 99%

Generating comparative evidence on new drugs and devices before approval

et al. 2020

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Fewer than half of new drugs have data on their comparative benefits and harms against existing treatment options at the time of regulatory approval in Europe and the US. Even when activecomparator trials exist, they may not produce meaningful data to inform decisions in clinical practice and health policy. Recently, the uncertainty associated with the paucity of well-designed active-comparator trials has been compounded by legal and regulatory changes in Europe and the US that have created a complex mix of expedited programs aimed at facilitating faster access to new drugs. Comparative evidence generation is even sparser for medical devices. Some have argued that the current process for regulatory approval needs to generate more evidence that is useful for patients, clinicians, and payers in health care systems. We propose a set of 5 key principles relevant to the European Medicines Agency (EMA), European medical device regulatory agencies, and the US Food and Drug Administration (FDA), as well as payers, that we believe will provide the necessary incentives for pharmaceutical and device companies to generate comparative data on drugs and devices and assure timely availability of evidence that is useful for decision making. First, labeling should routinely inform patients and clinicians whether comparative data exist on new products. Second, regulators should be more selective in their use of programs that facilitate drug and device approvals on the basis of incomplete benefit and harm data. Third, regulators should encourage the conduct of randomised trials with active comparators. Fourth, regulators should use prospectively-designed network meta-analyses based on existing and future randomised trials. Fifth, payers should use their policy levers and negotiating power to incentivise the generation of comparative evidence on new and existing drugs and devices, for example, by explicitly considering proven added benefit in pricing and payment decisions.

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Section: A Fragmented Evidence Base For Decision Making In Health Sysmentioning

confidence: 99%

Generating comparative evidence on new drugs and devices before approval

et al. 2020

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“…Following closely with a positive perception was Sakigake, although the perception was more varied, which may be because this pathway has only been introduced recently [16]. Respondents exhibited more ambivalence about the perceived value of Fast Track and PRIME, where for PRIME this may also be due to the novel nature of this pathway [17], while for Fast Track the opposite is true where the value of this older FRP seems lower compared with the newer BTD. Across all the FRPs, the companies believed the impact of the designation on stakeholder perception of that product was generally positive for most stakeholders (their own company, patients, physicians, regulators, and investors) except for HTA/payers, where it was mixed.…”

Section: Discussionmentioning

confidence: 98%

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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“…Nevertheless, recent approvals are likely associated with even more uncertainty (e.g., because they are more often based on single‐arm studies that include small numbers of patients). 46 Therefore, if anything, a more negative impact on HTA decision making outcomes can be expected. Third, differences in the type of medicines assessed by each HTA agency as well as differences in assessment methods, responsibilities, and mandates may have caused variation in assessment outcomes between HTA agencies that affected our results.…”

Section: Discussionmentioning

confidence: 99%

Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies

Bloem

Vreman

Peeters

et al. 2021

Clinical Translational Sci

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We aimed to determine whether uncertainties identified by the European Medicines Agency (EMA) were associated with negative relative effectiveness assessments (REA) and negative overall reimbursement recommendations by national health technology assessment (HTA) agencies. Therefore, we identified all HTA reports from HAS (France), NICE (England), SMC (Scotland) and ZIN (the Netherlands) for a cohort of innovative medicines that EMA had approved in 2009-2010 (excluding vaccines). Uncertainty regarding pivotal trial methodology, clinical outcomes and their clinical relevance were combined to reflect a low, medium or high level of uncertainty. We assessed associations by calculating risk ratios (RR) and 95% confidence intervals (CI), and agreement between REA and overall reimbursement recommendation outcomes. We identified 36 medicines for which 121 reimbursement recommendations had been issued by the HTA agencies between September 2009 and July 2018. High vs. low uncertainty was associated with an increased risk for negative REAs and negative overall reimbursement recommendations: RRs 1.9 (95% CI 0.9-3.9) and 1.6 (95% CI 0.7-3.5), respectively, which was supported by further sensitivity analyses. We identified a lack of agreement between 33 (27%) REA and overall reimbursement recommendation outcomes, which were mostly restricted recommendations that followed on negative REAs in case of low or medium uncertainty. In conclusion, high uncertainty identified by EMA was negatively associated with REAs and overall reimbursement recommendations. To reduce uncertainty and ultimately facilitate efficient patient access, regulators, HTA agencies and other stakeholders should discuss how uncertainties should be weighed and addressed early in the drug life cycle of innovative treatments.

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European Medicines Agency’s Priority Medicines Scheme at 2 Years: An Evaluation of Clinical Studies Supporting Eligible Drugs

Cited by 11 publications

References 25 publications

Generating comparative evidence on new drugs and devices before approval

Generating comparative evidence on new drugs and devices before approval

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

Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies

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