Encouraging New Uses for Old Drugs

Sachs, Rachel; Ginsburg, Paul Β.; Goldman, Dana P.

doi:10.1001/jama.2017.17535

Cited by 31 publications

(29 citation statements)

References 3 publications

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“…Pharmaceutical companies often choose not to invest in new therapeutic indications after expiry of basic patent and regulatory protection periods of their approved products (Langedijk et al, 2016;Nayroles et al, 2017). Patent claims for secondary uses often offer weaker protection compared to the primary basic product patent claims so the risk of free-riding by competitors is high (Sachs et al, 2017). Developing a new indication outside of a company's therapeutic focus is also high-risk and costly (Novac, 2013;Pushpakom et al, 2019).…”

Section: Challenges In Bringing New Uses On-labelmentioning

confidence: 99%

“…Developing a new indication outside of a company's therapeutic focus is also high-risk and costly (Novac, 2013;Pushpakom et al, 2019). Instead, companies may actually benefit from off-label prescribing of their products because it expands the patient population without them having to apply for a variation or extension of the marketing authorization (Sachs et al, 2017; STAMP European Commission expert group, 2017a). In addition, research has shown that the evidence base supporting new uses for marketed, off-patent medicines in anticancer treatment is largely built through academic or independent research .…”

Section: Challenges In Bringing New Uses On-labelmentioning

confidence: 99%

“…The opportunities offered by drug repurposing have not gone unnoticed: stories about the success of old "miracle drugs" in new disease areas have been published by the media on numerous occasions (Stone, 2019;. Moreover, several not-for-profit, patient, and governmental organizations have expressed their interest (Hernandez et al, 2017), and the concept has gained recognition among researchers, as illustrated by several influential publications in scientific journals of high impact (Nosengo, 2016;Sachs et al, 2017).…”

Section: Introductionmentioning

confidence: 99%

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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: Challenges In Bringing New Uses On-labelmentioning

confidence: 99%

Section: Challenges In Bringing New Uses On-labelmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

et al. 2020

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“…Finding new uses outside the scope of the original medical indication for existing drugs, referred to as drug repurposing or repositioning, is one solution to achieve efficiency. Existing drugs have already been tested in humans, have been demonstrated an acceptable level of safety and tolerability, and are often approved by regulatory agencies for human use (Sachs, et al, 2017). This could potentially increase the success rate of drug development and reduce the cost in terms of time.…”

Section: Introductionmentioning

confidence: 99%

EK-DRD: a comprehensive database for drug repositioning inspired by experimental knowledge

Zhao

Dai

et al. 2019

Preprint

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Drug repositioning, or the identification of new indications for approved therapeutic drugs, has gained substantial traction with both academics and pharmaceutical companies because it reduces the cost and duration of the drug development pipeline and it reduces the likelihood of unforeseen adverse events. So far, there has not been a systematic effort to identify such opportunities, in part because of the lack of a comprehensive resource for an enormous amount of unsystematic drug repositioning information to support scientists who could benefit from this endeavor. To address this challenge, we developed a new database, Experimental Knowledge-Based Drug Repositioning Database (EK-DRD) by using text and data mining, as well as manual curation. EK-DRD contains experimentally validated drug repositioning annotation for 1861 FDA-approved and 102 withdrawn small molecule drugs. Annotation was done at four levels, using 70,212 target assay records, 3999 cell assay records, 585 organism assay records, and 8910 clinical trial records. Additionally, approximately 1799 repositioning protein or target sequences coupled with 856 related diseases and 1332 pathways are linked to the drug entries. Our webbased software displays a network for integrative relationships between drugs, their repositioning targets, and related diseases. The database is fully searchable and supports extensive text, sequence, chemical structure, and relational query searches. Availability: EK-DRD is freely available at

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“…The availability of long-term, real-world data with established safety records provides us with an opportunity to use generic drugs to improve health outcomes in new indications at a lower cost. 1 However, there exist inherent limitations, such as unmeasured confounders, in the use of real-world data. New strategies to validate the potential use of old drugs in addition to existing big data are warranted.…”

Section: Introductionmentioning

confidence: 99%

New use for old drugs: The protective effect of atypical antipsychotics on hepatocellular carcinoma

Chen

Chan

Hsu

et al. 2018

Intl Journal of Cancer

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It has been encouraged to use large existing data like insurance claims data to investigate the new indications of old drugs. New strategies of research are warranted to identify feasible drugs. We conducted a dual research model with a population‐based case–control study using Taiwan's National Health Insurance Research Database and an in vitro study to investigate the association between atypical antipsychotic and Hepatocellular carcinoma (HCC) risk. The study herein consists of two components. The first is a population‐based case–control study using existing data from the Taiwan National Health Insurance Research Database. The second component was an in vitro study in which HCC cell lines (Huh7 and Hep G2) were treated with risperidone, quetiapine and clozapine. after treatment of the foregoing antipsychotics, the HCC cell lines were assessed for cell proliferation, invasion and apoptosis. Multivariate conditional logistic regression analysis revealed that antipsychotic use was independently and inversely associated with HCC risk (adjusted odds‐ratio [aOR]:0.85, 95% CI: 0.81–0.89). The protective effect was dose‐dependent: compared to the low cumulative defined daily dose (cDDD) group (0–29 cDDD), the 30–89 cDDD and ≥90 cDDD groups were associated with significantly reduced risk for HCC (aOR: 0.56, 95% CI: 0.41–0.76; aOR: 0.37, 95% CI: 0.27–0.50, respectively). In vitro study results indicated that risperidone, quetiapine and clozapine significantly inhibited cell proliferation, invasion and induced apoptosis in human HCC cell lines. Our results herein suggested that antipsychotic use might reduce the risk of HCC and may provide evidence for new uses of old drugs.

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Encouraging New Uses for Old Drugs

Cited by 31 publications

References 3 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

EK-DRD: a comprehensive database for drug repositioning inspired by experimental knowledge

New use for old drugs: The protective effect of atypical antipsychotics on hepatocellular carcinoma

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