Demands for access to new therapies: are there alternatives to accelerated access?

Pace, Jessica; Ghinea, Narcyz; Kerridge, Ian; Lipworth, Wendy

doi:10.1136/bmj.j4494

Cited by 16 publications

(12 citation statements)

References 18 publications

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“…28 One rationale for introducing such programs is to meet patient demand for potentially effective therapies for life-threatening diseases for which there is no existing treatment. 29,30 Studies have confirmed that drugs that qualify for expedited programs have shorter development times and receive regulatory approval faster. Between 2012 and 2016, the duration of clinical development was almost one year shorter for drugs in the FDA's expedited programs than for drugs that were not.…”

Section: Expedited Programsmentioning

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: Expedited Programsmentioning

confidence: 99%

Generating comparative evidence on new drugs and devices before approval

et al. 2020

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“…FRPs can expedite the product development and regulatory review of medicines by providing alternatives to the timing and data comprehensiveness in standard product development and regulatory review routes prior to authorization, with the understanding that further data will be developed to either confirm or refute the plausible benefit upon which the authorization was based. While the ultimate goal is to expedite patient access to safe, effective, quality medicines for high unmet needs [ 13 ], including in crisis situations, such as with the current coronavirus disease 2019 (COVID-19) pandemic [ 14 ], this remains a challenge as the outcomes of FRPs are not always widely embraced by HTAs and payers because of the uncertainty around the effectiveness of such treatments.…”

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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“…In addition, the decision modifiers mentioned earlier imply that a larger degree of uncertainty in the evidence base may be accepted in the reviewed cases compared with cases where these modifiers do not apply. A recently suggested approach may be to use price as a lever for reimbursement explicitly linking acceptable price to the extent of uncertainty in evidence . However, as many new drugs, at least in this review, have a real potential to harm, a direct link to pricing may not be readily made.…”

Section: Discussionmentioning

confidence: 99%

Balancing early access with uncertainties in evidence for drugs authorized by prospective case series – systematic review of reimbursement decisions

Wallerstedt

Henriksson

2018

Brit J Clinical Pharma

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AimsTo review clinical and cost‐effectiveness evidence underlying reimbursement decisions relating to drugs whose authorization mainly is based on evidence from prospective case series.MethodsA systematic review of all new drugs evaluated in 2011–2016 within a health care profession‐driven resource prioritization process, with a market approval based on prospective case series, and a reimbursement decision by the Swedish Dental and Pharmaceutical Benefits Agency (TLV). Public assessment reports from the European Medicines Agency, published pivotal studies, and TLV, Scottish Medicines Consortium and National Institute of Health and Care Excellence decisions and guidance documents were reviewed.ResultsSix drug cases were assessed (brentuximab vedotin, bosutinib, ponatinib, idelalisib, vismodegib, ceritinib). The validity of the pivotal studies was hampered by the use of surrogate primary outcomes and the absence of recruitment information. To quantify drug treatment effect sizes, the reimbursement agencies primarily used data from another source in indirect comparisons. TLV granted reimbursement in five cases, compared with five in five cases for Scottish Medicines Consortium and four in five cases for National Institute of Health and Care Excellence. Decision modifiers, contributing to granted reimbursement despite hugely uncertain cost‐effectiveness ratios, were, for example, small population size, occasionally linked to budget impact, severity of disease, end of life and improved life expectancy.ConclusionFor drugs whose authorization is based on prospective case series, most applications for reimbursement within public health care are granted. The underlying evidence has limitations over and above the design per se, and decision modifiers are frequently referred to in the value‐based pricing decision making.

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Demands for access to new therapies: are there alternatives to accelerated access?

Cited by 16 publications

References 18 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

Balancing early access with uncertainties in evidence for drugs authorized by prospective case series – systematic review of reimbursement decisions

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