Four scenarios for the future of medicines and social policy in 2030

Leufkens, Hubert G. M.; Kusynová, Zuzana; Aitken, Murray; Hoekman, Jarno; Stolk, Pieter; Klein, Kevin; Mantel‐Teeuwisse, Aukje K.

doi:10.1016/j.drudis.2022.03.018

Cited by 8 publications

(12 citation statements)

References 22 publications

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“…However, also in the EU, one of the four pillars of the ‘Pharmaceutical strategy for Europe’ aims at ensuring access to affordable medicines for patients, and at addressing unmet medical needs, such as CF [ 55 ].…”

Section: Discussionmentioning

confidence: 99%

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

Costa

Girotti

Pauro

et al. 2022

Orphanet J Rare Dis

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Background Over the past decade, a new class of drugs called CFTR (cystic fibrosis transmembrane conductance regulator) modulators have shown to be able to improve clinical outcomes in patient with Cystic Fibrosis. In this analysis, we have extensively reviewed the regulatory pathways and decisions adopted by FDA and EMA to speed up the development, the review and the approval of these drugs, with the aim of identifying possible clinical and public health implications associated with differences. Results CFTR modulators have been developed towards addressing three main genetic domains: (1) F508del homozygous (F508del/F508del), (2) F508del heterozygous, and (3) genotypes not carrying F508del mutation; and expanded from adult to paediatric population. Programs to expedite the reviewing and licensing of CFTR modulators were extensively adopted by FDA and EMA. All CFTR modulators have been licensed in the US as orphan drugs, but in the EU the orphan status for LUM/IVA was not confirmed at the time of marketing authorization as results from the pivotal trial were not considered clinically significant. While FDA and EMA approved CFTR modulators on the basis of results from phase III double-blind RCTs, main differences were found on the extension of indications: FDA accepted non-clinical evidence considering a recovery of the CFTR function ≥ 10% based on chloride transport, a reliable indicator to correlate with improvement in clinical outcomes. By contrast, EMA did not deem preclinical data sufficient to expand the label of CFTR modulators without confirmatory clinical data. Conclusions Regulators played an important role in fostering the development and approval of CFTR modulators. However, differences were found between FDA and EMA in the way of reviewing and licensing CFTR modulators, which extended beyond semantics affecting patients’ eligibility and access: FDA’s approach was more mechanistic/biology-driven while the EMA’s one was more oriented by clinical evidence. This might refer to the connection between the EMA and the Member States, which tends to base decisions on pricing and reimbursement on clinical data rather than pre-clinical ones. Here we have proposed a two-step personalized-based model to merge the ethical commitment of ensuring larger access to all potential eligible patients (including those harboring very rare mutations) with the one of ensuring access to clinically assessed and effective medicines through Real World Data.

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Section: Discussionmentioning

confidence: 99%

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

Costa

Girotti

Pauro

et al. 2022

Orphanet J Rare Dis

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“…However, the question is already being debated, either explicitly or-more often-implicitly, and will come to the fore with the advent of more high-price orphan drugs. Leufkens et al (2022) have recently sketched future scenarios where one might expect that "some patient advocates still March for enabling everything that is possible. .…”

Section: The Risks Of Allocative and Technical Inefficienciesmentioning

confidence: 99%

“…. critical exposure" (Leufkens et al, 2022). There is now a real risk of loss of solidarity among the community (Leufkens et al, 2022) of insured persons in the face of ultra-high costs for a few (Thomas and Abelson, 2019).…”

Section: The Risks Of Allocative and Technical Inefficienciesmentioning

confidence: 99%

“…. .." (Leufkens et al, 2022). Duchenne muscular dystrophy (DMD) affords another example: several therapeutic strategies have been investigated and a small number of products have received marketing authorisation; while the effect sizes are non-null, they are far away from being a cure (Yao S et al, 2021).…”

Section: Introductionmentioning

confidence: 99%

“…More often we are faced, e.g. in rare cancers, with a “[p]rogression-free survival gain of 4 months between treated and non-treated, a response rate of 65%, but no survival benefit, single-arm studies with challenges to interpret the results….” ( Leufkens et al, 2022 ). Duchenne muscular dystrophy (DMD) affords another example: several therapeutic strategies have been investigated and a small number of products have received marketing authorisation; while the effect sizes are non-null, they are far away from being a cure ( Yao S et al, 2021 ).…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Orphan drugs’ clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement

Eichler¹,

Kossmeier²,

Zeitlinger

et al. 2023

Front. Pharmacol.

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Legislations incentivising orphan drug development and scientific advances have made orphan drugs pharma’s high-end favourite for the past two decades. Currently, around 50% of new marketing authorizations are for orphan drugs. For third-party healthcare payers (“payers”) the rise of orphan drugs presents new challenges, including a high degree of uncertainty around clinical benefits and harms, a moderate effect size (for many orphan drugs), and a high price tag. The association of high clinical uncertainty and moderate effect sizes is not surprising in small target populations but in combination with high prices creates the risk of allocative and technical inefficiencies for payers. We here discuss and illustrate these risks. A combination of policies is needed for mitigation of allocative inefficiency: while there may be a rationale for higher prices for orphan than non-orphan drugs, a focus of pricing and reimbursement negotiations should include considerations of product profitability and of the consequences of orphan drug costs on the distribution inequality of medication costs for individual insured persons, coupled to knowledge generation from reimbursement contracts covering high-price orphan drugs that would benefit the wider patient community. Performance-based managed entry agreements could help to de-risk the economic consequences of clinical uncertainty and to mitigate technical inefficiency.

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Exploring the association between combined formal and informal educational approaches and cultural intelligence among undergraduate nursing students: A cross-sectional study

Cieślak,

Jaworski,

Panczyk

et al. 2025

Nurse Education Today

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Four scenarios for the future of medicines and social policy in 2030

Cited by 8 publications

References 22 publications

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

Orphan drugs’ clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement

Exploring the association between combined formal and informal educational approaches and cultural intelligence among undergraduate nursing students: A cross-sectional study

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