“Adaptive pathways” to drug authorisation: adapting to industry?

Davis, Courtney; Lexchin, Joel; Jefferson, Tom; Gøtzsche, Peter C; McKee, Martin

doi:10.1136/bmj.i4437

Cited by 43 publications

(45 citation statements)

References 48 publications

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“…Patients receiving allogeneic cells may need long-term immune suppression to avoid rejection. More broadly, concerns have been raised that the evidence for benefits to patients of adaptive pathway approaches is lacking or contradictory [28]. There is also concern about the follow-up evidence for some treatments granted conditional approval by the EMA, with inconsistencies and delays in the fulfilment of specific obligations [29, 30].…”

Section: Discussionmentioning

confidence: 99%

Innovative regenerative medicines in the EU: a better future in evidence?

Corbett

Webster

Hawkins

et al. 2017

BMC Med

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BackgroundDespite a steady stream of headlines suggesting they will transform the future of healthcare, high-tech regenerative medicines have, to date, been quite inaccessible to patients, with only eight having been granted an EU marketing licence in the last 7 years. Here, we outline some of the historical reasons for this paucity of licensed innovative regenerative medicines. We discuss the challenges to be overcome to expedite the development of this complex and rapidly changing area of medicine, together with possible reasons to be more optimistic for the future.DiscussionSeveral factors have contributed to the scarcity of cutting-edge regenerative medicines in clinical practice. These include the great expense and difficulties involved in planning how individual therapies will be developed, manufactured to commercial levels and ultimately successfully delivered to patients. Specific challenges also exist when evaluating the safety, efficacy and cost-effectiveness of these therapies. Furthermore, many treatments are used without a licence from the European Medicines Agency, under “Hospital Exemption” from the EC legislation. For products which are licensed, alternative financing approaches by healthcare providers may be needed, since many therapies will have significant up-front costs but uncertain benefits and harms in the long-term. However, increasing political interest and more flexible mechanisms for licensing and financing of therapies are now evident; these could be key to the future growth and development of regenerative medicine in clinical practice.ConclusionsRecent developments in regulatory processes, coupled with increasing political interest, may offer some hope for improvements to the long and often difficult routes from laboratory to marketplace for leading-edge cell or tissue therapies. Collaboration between publicly-funded researchers and the pharmaceutical industry could be key to the future development of regenerative medicine in clinical practice; such collaborations might also offer a possible antidote to the innovation crisis in the pharmaceutical industry.

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Section: Discussionmentioning

confidence: 99%

Innovative regenerative medicines in the EU: a better future in evidence?

Corbett

Webster

Hawkins

et al. 2017

BMC Med

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“…While some stakeholders embraced it or were at least willing to explore further, others voiced a range of concerns. Concerns raised included the lowering of regulatory evidence standards, putting patients at risk of unsafe treatments, and forcing healthcare systems to pay for unproven drugs …”

Section: The Mapps Conceptmentioning

confidence: 99%

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

Eichler¹,

Bedlington

Boudes

et al. 2018

Clin Pharma and Therapeutics

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Medicines Adaptive Pathways to Patients (MAPPs) seeks to foster access to novel beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span, in a sustainable fashion. We summarize the MAPPs engagement process and critical questions to be asked at each milestone of the product life-span. These considerations are of relevance for regulatory and access pathways that strive to address the "evidence vs. access" conundrum.

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“…The standards for approval continue to fall, most recently illustrated by the EMA's introduction of adaptive pathways, which will allow drugs to be approved based on observational data only . Some patient groups support the industry's demands for faster approvals that will give them rapid access to the latest drugs.…”

Section: Countering Myths About Drug Regulationmentioning

confidence: 99%

Patients not patents: Drug research and development as a public enterprise

Gøtzsche

2018

Eur J Clin Investigation

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“Adaptive pathways” to drug authorisation: adapting to industry?

Cited by 43 publications

References 48 publications

Innovative regenerative medicines in the EU: a better future in evidence?

Innovative regenerative medicines in the EU: a better future in evidence?

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

Patients not patents: Drug research and development as a public enterprise

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