Envisioning an actionable research agenda to facilitate repurposing of off-patent drugs

Austin, Christopher P.; Mount, Bobbie Ann; Colvis, Christine M.

doi:10.1038/d41573-021-00090-y

Cited by 8 publications

(5 citation statements)

References 2 publications

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“…An integrated solution for the described hurdles may require both changes in the interaction between key actors and changes in legislation. Suggestions for change have been made by Austin et al (2021) including, amongst others, improved education, financial and regulatory incentives that create viable business cases, and reimbursement strategies for off-label use. We propose the following recommendations for changes in legislation that build on these suggestions:…”

Section: Discussionmentioning

confidence: 99%

“…First, when reality shows that some repurposed drugs are not being registered and widespread off-label use is the result, supported by scientific evidence, other options to reach long-term availability and appropriate use driven by academia could be explored (“label change last” ( Austin et al, 2021 )). For example, this could entail close monitoring and structured assessment of off-label use by regulators and reimbursement authorities, and providing regulators with the right to change a label or add an indication to a label as proposed by Gyawali et al (2021) .…”

Section: Discussionmentioning

confidence: 99%

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Drug Repurposing for Rare Diseases: A Role for Academia

et al. 2021

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Background: The European Commission highlights in its Pharmaceutical Strategy the role of academic researchers in drug repurposing, especially in the development of orphan medicinal products (OMPs). This study summarizes the contribution of academia over the last 5 years to registered repurposed OMPs and describes barriers to success, based upon three real world cases.Methods: OMPs granted marketing authorization between January 2016 and December 2020 were reviewed for repurposing and whether the idea originated from academia or industry. Three cases of drug repurposing were selected from different therapeutic areas and stages of development to identify obstacles to success.Results: Thirteen of the 68 OMPs were the result of drug repurposing. In three OMPs, there were two developments such as both a new indication and a modified application. In total, twelve developments originated from academia and four from industry. The three cases showed as barriers to success: lack of outlook for sufficient return of investments (abatacept), lack of regulatory alignment and timing of interaction between healthcare professionals and regulators (etidronate), failure to register an old drug for a fair price, resulting in commercialization as a high priced orphan drug (mexiletine).Conclusion: While the majority of repurposed OMPs originates in academia, a gap exists between healthcare professionals, regulators and industry. Future strategies should aim to overcome these hurdles leading to more patient benefit through sustainable access of repurposed drugs. Potential solutions include improved regulatory and reimbursement knowledge by academia and the right for regulators to integrate new effectiveness data into product labels.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Drug Repurposing for Rare Diseases: A Role for Academia

et al. 2021

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“…Personalized trials can also be useful for Per-DS investigators to study the potential benefits of off-label use of a medication ( Austin et al, 2021 ). Given the exploratory nature of such investigations, close guidance and supervision from the clinician is essential.…”

Section: Typology Of Per-ds Investigationsmentioning

confidence: 99%

Personalized Data Science and Personalized (N-of-1) Trials: Promising Paradigms for Individualized Health Care

Duan

Norman

Schmid

et al. 2022

Harvard Data Science Review

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The term 'data science' usually refers to the process of extracting value from big data obtained from a large group of individuals. An alternative rendition, which we call personalized data science (Per-DS), aims to collect, analyze, and interpret personal data to inform personal decisions. This article describes the main features of Per-DS, and reviews its current state and future outlook. A Per-DS investigation is of, by, and for an individual, the Per-DS investigator, acting simultaneously as her own investigator, study participant, and beneficiary, and making personalized decisions for study design and implementation. The scope of Per-DS studies may include systematic monitoring of physiological or behavioral patterns, case-crossover studies for symptom triggers, pre-post trials for exposure-outcome relationships, and personalized (N-of-1) trials for effectiveness. Per-DS studies produce personal knowledge generalizable to the individual's future self (thus benefiting herself) rather than knowledge generalizable to an external population (thus benefiting others). This endeavor requires a pivot from data mining or extraction to data gardening, analogous to home gardeners producing food for home consumption-the Per-DS investigator needs to 'cultivate the field' by setting goals, specifying study design, identifying necessary data elements, and assembling instruments and tools for data collection. Then, she can implement the study protocol, harvest her personal data, and mine the data to extract personal knowledge. To facilitate Per-DS studies, Per-DS investigators need support from community-based, scientific, philanthropic, business, and government entities, to develop and deploy resources such as peer forums, mobile apps, 'virtual field guides,' and scientific and regulatory guidance.

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“…Repurposing's advantages seem tantalizingly straightforward. However, its practical implementation has proven to be less widespread than initially hoped for (13). Major hurdles to repurposing were highlighted by Austin et al in 2021, as discussed at the National Center for Advancing Translational Sciences (NCATS), Reagan-Udall Foundation (RUF) and FDA workshop on repurposing in 2019 (14).…”

Section: Introductionmentioning

confidence: 99%

International regulatory and publicly-funded initiatives to advance drug repurposing

Spin,

Mantel-Teeuwisse,

Pasmooij

2024

Front. Med.

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IntroductionAlthough drug repurposing holds great potential in addressing unmet needs, successful practical implementation is challenging and has been less widespread than anticipated. Regulators may play a critical role in addressing this, and recent years have seen the conception of regulator-initiated and publicly-funded repurposing initiatives, with significant regulator involvement.MethodsInternational regulators and public funders (n = 8) were interviewed to obtain insight in how repurposing can be advanced from a regulatory perspective. Transcripts were thematically analyzed.ResultsMost initiatives employed a broad concept of repurposing. While patient access was the main focus, label extension remained the gold standard. Commonly perceived barriers were a lack of regulatory expertise, limited downstream drug development, insufficient financial incentives, inadequate awareness of challenges, and poor collaboration. Ways for regulators to facilitate repurposing include early and accessible involvement fostering education, collaboration, and awareness. Increased stakeholder engagement, including internationally, was recommended. Legislative changes may enable the current repurposing ecosystem to evolve.DiscussionRegulators may play a central role in advancing repurposing by reconsidering their responsibilities within the current regulatory framework, both in mitigating repurposing pitfalls and actively encouraging repurposing initiatives by industry and non-traditional drug developers.

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Envisioning an actionable research agenda to facilitate repurposing of off-patent drugs

Cited by 8 publications

References 2 publications

Drug Repurposing for Rare Diseases: A Role for Academia

Drug Repurposing for Rare Diseases: A Role for Academia

Personalized Data Science and Personalized (N-of-1) Trials: Promising Paradigms for Individualized Health Care

International regulatory and publicly-funded initiatives to advance drug repurposing

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