Repurposing old drugs in oncology: Opportunities with clinical and regulatory challenges ahead

Shah, Rajiv; Stonier, Peter D.

doi:10.1111/jcpt.12759

Cited by 46 publications

(36 citation statements)

References 218 publications

(391 reference statements)

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“…In summary, the legal basis will vary depending on the type of applicant (e.g., only the marketing authorization holder can apply for a variation or extension procedure) and on the amount of data that can be extracted from published literature. Scientific and regulatory advice can be requested at national or European level to better understand the various regulatory pathways and the level of evidence required for marketing authorization of the new indication (Pantziarka, 2017;Shah and Stonier, 2019). Another important aspect that needs to be taken into consideration when selecting which regulatory pathway to follow are the available incentives described below.…”

Section: European Regulatory Framework 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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Section: European Regulatory Framework 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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“…В последние годы также рекомендуется проведение исследований, направленных на расширение показаний к применению старых, более дешевых, онкологических ЛС и сравнение их экономической эффективности с инновационными препаратами [56].…”

Section: возможные пути решения экономических проблем противоопухолевunclassified

Economic challenges of oncological diseases’ pharmacotherapy

2020

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Oncological diseases rank high in the structure of population morbidity and mortality. They entail considerable direct and indirect economic costs. In the past decades, the cost of oncotherapy has increased significantly, which is largely conditioned by high prices of antitumor drugs, which on average increased by ten times in the past ten years. At the same time, many innovative medications have only minor advantages over cheaper old medications because they are registered based on the data on the achievement of the surrogate endpoint – extension of progression-free survival. The high cost of oncotherapy is associated with financial toxicity that affects negatively the patients’ quality of life, their adherence to treatment and consequently survival. To reduce the cost of oncotherapy, it is necessary to conduct pharma-economic analysis, the results of which can serve as the basis to negotiate price-cutting with the manufacturers, as well as to use high-quality generics and biosimilars as effective and safe as their originals, and to monitor effectiveness and safety of all antitumor drugs within the pharmacovigilance framework.

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“…DR has generated increasing interest in the past decade owing to its use as an explicit development strategy and substantial benefits over the traditional development of new drugs (de novo drugs) by reducing development risks and facilitating the development process . Indeed, the number of publications related to DR in PubMed has increased rapidly since 2004 . Moreover, the number of registrations of clinical trials for DR has increased in Japan (Figure ).…”

Section: Introductionmentioning

confidence: 99%

Drug repositioning in cancer: The current situation in Japan

et al. 2020

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Cancer is a leading cause of death worldwide, and the incidence continues to increase. Despite major research aimed at discovering and developing novel and effective anticancer drugs, oncology drug development is a lengthy and costly process, with high attrition rates. Drug repositioning (DR, also referred to as drug repurposing), the process of finding new uses for approved noncancer drugs, has been gaining popularity in the past decade. DR has become a powerful alternative strategy for discovering and developing novel anticancer drug candidates from the existing approved drug space. Indeed, the availability of several large established libraries of clinical drugs and rapid advances in disease biology, genomics/transcriptomics/ proteomics and bioinformatics has accelerated the pace of activity-based, literaturebased and in silico DR, thereby improving safety and reducing costs. However, DR still faces financial obstacles in clinical trials, which could limit its practical use in the clinic. Here, we provide a brief review of

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Repurposing old drugs in oncology: Opportunities with clinical and regulatory challenges ahead

Cited by 46 publications

References 218 publications

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

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

Economic challenges of oncological diseases’ pharmacotherapy

Drug repositioning in cancer: The current situation in Japan

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