Strengthening regulatory science in academia: STARS, an EU initiative to bridge the translational gap

Starokozhko, Viktoriia; Kallio, Marko J.; Howell, Åsa Kumlin; Salmi, Anna Mäkinen; Andrew-Nielsen, Gunilla; Goldammer, Mark; Burggraf, Manja; Löbker, Wiebke; Böhmer, Anne C.; Agricola, Eleonora; Vries, Corinne S de; Pasmooij, Anna M. G.; Mol, Peter G. M.

doi:10.1016/j.drudis.2020.10.017

Cited by 27 publications

(35 citation statements)

References 19 publications

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“…The main hurdles from the side of healthcare professionals involved in drug repurposing were that they have little knowledge about regulatory and reimbursement processes and instead are focused on scientific progress and patient care. In the case of etidronate for pseudoxanthoma elasticum for example, clinicians seem to focus mainly on providing the scientific evidence in drug repurposing and due to lack of knowledge about the regulatory framework as well as restricted time in academia, ideas for drug repurposing might fail unnecessarily or are prematurely halted ( Verbaanderd et al, 2020 ; Starokozhko et al, 2021 ). In addition, when a drug is available for patients and reimbursed without registration, e.g., as off-label use, they may not be motivated and incentivized to assist in steps towards commercialization.…”

Section: Discussionmentioning

confidence: 99%

“…First, healthcare professionals involved in drug repurposing should become better educated in the regulatory field and understand the advantages of a marketing authorization over off-label use. Increasing knowledge in academia about the regulatory system, perhaps centralized on a national level as well as international efforts such as the STARS initiative can increase alignment ( Starokozhko et al, 2021 ). Second, healthcare professionals involved in drug repurposing together with private and regulatory actors will have to learn and understand each other’s drive and language.…”

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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“…It's important that the hub of the network provides a dedicated infrastructure to harmonize the roles and responsibilities, facilitate the communication between the trial promoter and each center/ethical committee/national and local competent authority, supervise the timing of each step and provide help in those centers requiring expertise and support for specific duties related to the trial. Furthermore, it should produce and diffuse specific guidelines to enhance the comparability of data acquired by molecular imaging and to boost molecular imaging so that it becomes a standard diagnostic modality in future clinical medicine and research ( 7 , 8 ).…”

Section: Discussionmentioning

confidence: 99%

ERANET JTC 2011: Submission and Activation of an International Academic Translational Project in Advanced Breast Cancer. Experience From the ET-FES Study

et al. 2022

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Background: Academic research is important to face unmet medical needs. The Oncological community encounters many hurdles in setting up multicenter investigator-driven trials mainly due to administrative complexity. The purpose of a network organization at a multinational level is to facilitate clinical trials through standardization, coordination, and education for drug development and regulatory approval.Methods: The application of an European grant foresees the creation of a consortium which aims at facilitating multi-center academic clinical trials.Results: The ERA-NET TRANSCAN Call 2011 on “Validation of biomarkers for personalized cancer medicine” was released on December 2011. This project included Italian, Spanish, French and German centers. The approval process included Consortium constitution, project submission, Clinical Trial Submission, and activation on a national level. The different timescales for submitting study documents in each Country and the misalignment of objections by each Competent Authority CA, generated several requests for changes to the study documents which meant amendments had to be made; as requested by the 2001/20/EC Directive, the alignment of core documents is mandatory. This procedure impacted significantly on study activation timelines. Time to first patient in was 14, 10, 28, and 31 months from the date of submission in Italy, France, Spain, and Germany, respectively. Accrual was stopped on 22nd January 2021 due to an 18F FES shortage as the primary reason but also for having exceeded the project deadlines with consequent exhaustion of the funds allocated for the project.Conclusions: Pharmaceutical companies might be reluctant to fund research projects aimed at treatment individualization if the approval for a wider indication has already been achieved. Academic trials therefore become fundamental for promoting trials which are not attractive to big pharma. It was very difficult and time consuming to activate an academic clinical trial, for this reason, a study may become “old” as new drugs entered into the market. National institutions should promote the development of clinical research infrastructures and network with competence in regulatory, ethical, and legal skills to speed up academic research.

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“…Better and earlier engagement between academic researchers, regulatory agencies, and biotech/pharma could help to fill this gap. 16 In addition, mechanisms to enhance returns of revenues into academic research could reduce this unused potential and could further advance the successful development of future innovative drugs.…”

mentioning

confidence: 99%

“…However, many other potential breakthroughs have been unable to progress to the development stage and we suspect that a substantial part of academic research remains unexploited. Better and earlier engagement between academic researchers, regulatory agencies, and biotech/pharma could help to fill this gap 16 . In addition, mechanisms to enhance returns of revenues into academic research could reduce this unused potential and could further advance the successful development of future innovative drugs.…”

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confidence: 99%

Advanced therapies are ready to take centre stage: Academia's involvement with regulation needs to raise its game

Hidalgo‐Simon

Fibbe

2021

Brit J Clinical Pharma

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Several CAR-T cells-based therapies have been approved for the treatment of blood cancers. 1 Gene therapies are constantly maturing and are now targeting not only rare diseases but also more common ailments such as beta thalassemia. Although toxicity issues are by no means minor, 2 it marks the beginning of an era where gene therapies will become widely applied.Clinical trials for advanced therapies (ATMPs, which comprise genetic and somatic cells therapies and tissue engineered products) have grown at a rapid pace, with a dramatic global increase in the number of clinical trials, but much less so in Europe than in other

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Strengthening regulatory science in academia: STARS, an EU initiative to bridge the translational gap

Cited by 27 publications

References 19 publications

Drug Repurposing for Rare Diseases: A Role for Academia

Drug Repurposing for Rare Diseases: A Role for Academia

ERANET JTC 2011: Submission and Activation of an International Academic Translational Project in Advanced Breast Cancer. Experience From the ET-FES Study

Advanced therapies are ready to take centre stage: Academia's involvement with regulation needs to raise its game

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