Pimping up Drugs Recovered, Superannuated and Under Exploited Drugs - An Introduction to the Basics of Drug Reprofiling

Dilly, Suzanne J.; Morris, Gareth

doi:10.2174/1570163814666170117120005

Cited by 8 publications

(3 citation statements)

References 30 publications

(35 reference statements)

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“…Some repurposed medicines require product changes (e.g., change in dose, pharmaceutical form, route of administration) or are combined with other medicines or medical devices in the new indication. This can be a commercially interesting repurposing strategy as product changes may generate new intellectual property and enable a pharmaceutical developer to rebrand a product for its new use (Novac, 2013;Dilly and Morris, 2017). A final scenario covers the repurposing of approved medicines that are out of basic patent or regulatory protection and are used "as-is," thus do not require any substantial product changes (Bloom, 2016).…”

Section: Box 1 | Repurposing a Medicinementioning

confidence: 99%

How to balance valuable innovation with affordable access to medicines in Belgium?

et al. 2022

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Background: Countries are struggling to provide affordable access to medicines while supporting the market entry of innovative, expensive products. This Perspective aims to discuss challenges and avenues for balancing health care system objectives of access, affordability and innovation related to medicines in Belgium (and in other countries).Methods: This Perspective focuses on the R&D, regulatory approval and market access phases, with particular attention to oncology medicines, precision medicines, orphan medicines, advanced therapies, repurposed medicines, generics and biosimilars. The authors conducted a narrative review of the peer-reviewed literature, of the grey literature (such as policy documents and reports of consultancy agencies), and of their own research.Results: Health care stakeholders need to consider various initiatives for balancing innovation with access to medicines, which relate to clinical and non-clinical outcomes (e.g. supporting the conduct of pragmatic clinical trials, treatment optimisation and patient preference studies, optimising the use of real-world evidence in market access decision making), value assessment (e.g. increasing the transparency of the reimbursement system and criteria, tailoring the design of managed entry agreements to specific types of uncertainty), affordability (e.g. harnessing the role of generics and biosimilars in encouraging price competition, maximising opportunities for personalising and repurposing medicines) and access mechanisms (e.g. promoting collaboration and early dialogue between stakeholders including patients). Conclusion:Although there is no silver bullet that can balance valuable innovation with affordable access to medicines, (Belgian) policy and decision makers should continue to explore initiatives that exploit the potential of both the on-patent and off-patent pharmaceutical markets.

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Section: Box 1 | Repurposing a Medicinementioning

confidence: 99%

How to balance valuable innovation with affordable access to medicines in Belgium?

et al. 2022

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“…However, a report from June 2017 on pharmaceutical incentives and rewards in Europe showed that this last incentive had never been granted for any centrally approved substance (Copenhagen Economics, 2018). In certain cases, medicine developers may choose to reformulate, protect (e.g., through second medical use patents) and rebrand an established medicinal product to create sufficient legal and strategic protection from generic competitors (Smith, 2011;Novac, 2013;Dilly and Morris, 2017). For developers other than the original marketing authorization holder, a new full marketing authorization application may even offer a 10-year period of data and marketing protection for the repurposed product (Murteira et al, 2014a).…”

Section: Regulatory Incentives For Marketing Authorizationmentioning

confidence: 99%

On-Label or Off-Label? Overcoming Regulatory and Financial Barriers to Bring Repurposed Medicines to Cancer Patients

et al. 2020

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Repurposing of medicines has gained a lot of interest from the research community in recent years as it could offer safe, timely, and affordable new treatment options for cancer patients with high unmet needs. Increasingly, questions arise on how new uses will be translated into clinical practice, especially in case of marketed medicinal products that are out of basic patent or regulatory protection. The aim of this study was to portray the regulatory framework relevant for making repurposed medicines available to cancer patients in Europe and propose specific policy recommendations to address the current regulatory and financial barriers. We outlined two routes relevant to the clinical adoption of a repurposed medicine. First, a new indication can be approved, and thus brought on-label, via the marketing authorization procedures established in European and national legislation. Such procedures initiate a detailed and independent assessment of the quality and the benefit-risk balance of a medicinal product in a specific indication, benefiting both prescribers and patients as it reassures them that the scientific evidence is robust. However, the process of marketing authorization for new therapeutic indications entails a high administrative burden and significant costs while the return-on-investment for the pharmaceutical industry is expected to be low or absent for medicines that are out of basic patent and regulatory protection. Moreover, most of the repurposing research is conducted by independent or academic researchers who do not have the expertise or resources to get involved in regulatory procedures. A second option is to prescribe a medicine off-label for the new indication, which is managed at the national level in Europe. While off-label use could provide timely access to treatments for patients with urgent medical needs, it also entails important safety, liability and financial risks for patients, physicians, and society at large. In view of that, we recommend finding solutions to facilitate bringing new uses on-label, for example by developing a collaborative framework between not-for-profit and academic organizations, pharmaceutical industry, health technology assessment bodies, payers, and regulators.

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“…Third, sometimes a new dosage form, route of administration, strength, xed combination or formulation of the existing medicine is needed for use in a new therapeutic indication. Such changes may generate new IP and can enable a pharmaceutical developer to rebrand a product for its new use, making it a commercially interesting repurposing opportunity (7)(8)(9). A fourth and nal scenario covers the repurposing of approved medicines that are out of basic patent or regulatory protection and that do not require any signi cant product changes, which is sometimes referred to as generic or off-patent drug repurposing (10).…”

Section: Introductionmentioning

confidence: 99%

New Mechanisms to Fund Independent Clinical Trials for Repurposing Off-Patent or Generic Medicines

Verbaanderd

Rooman

Huys

2020

Preprint

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Finding new therapeutic uses for approved, off-patent or generic medicines could lead to safe, affordable and timely new treatment options for patients with high medical needs. However, due to a lack of economic incentives, pharmaceutical developers are rarely interested to invest in this type of research. Consequently, potential new uses for off-patent or generic medicines are mainly studied in independent proof-of-concept clinical trials initiated and led by researchers from academia, research institutes or collaborative groups. Yet, these researchers need additional financial support to conduct expensive phase III clinical trials to confirm the results from exploratory research. In this study, scientific and grey literature was searched to identify and evaluate new mechanisms for funding clinical trials with repurposed medicines. Semi-structured interviews were conducted with 16 European stakeholders with expertise in clinical research, funding mechanisms and/or drug repurposing between November 2018 and February 2019 to consider the future perspectives of applying new funding mechanisms. Traditional grant funding awarded by government and philanthropic organisations or companies is well known and widely implemented in all research fields. In contrast, the application potential of newer mechanisms to fund independent clinical research, such as social impact bonds or crowdfunding, is not yet known. Interviewees stated that there is a substantial need for additional financial support in health research, especially in disease domains where there is limited commercial interest. However, the implementation of new funding mechanisms is facing several practical and financial challenges, such as a lack of expertise and guidelines, high transaction costs and difficulties to measure health outcomes. Furthermore, increased collaboration and centralisation at a European and international level is recommended to make clinical research more efficient and reduce the need for additional funding. New funding mechanisms to support clinical research may become more important in the future, but the unresolved issues warrant further exploration in pilot projects.

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Pimping up Drugs Recovered, Superannuated and Under Exploited Drugs - An Introduction to the Basics of Drug Reprofiling

Cited by 8 publications

References 30 publications

How to balance valuable innovation with affordable access to medicines in Belgium?

How to balance valuable innovation with affordable access to medicines in Belgium?

On-Label or Off-Label? Overcoming Regulatory and Financial Barriers to Bring Repurposed Medicines to Cancer Patients

New Mechanisms to Fund Independent Clinical Trials for Repurposing Off-Patent or Generic Medicines

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