Repurposing Medicines Toolkit - Guidance for navigating the process

The Best Pharmaceuticals for Children Act (BPCA), initially enacted in 2002, is legislation aimed to improve the knowledge of the dosing, safety, and efficacy of medications prescribed for children. The BPCA, in addition to the Pediatric Research Equity Act (PREA) and overseen by the Food and Drug Administration (FDA), has led to additional data in more than 1000 drug labels.  Section 409I of the BPCA legislation, also authorizes the National Institutes of Health (NIH) to sponsor an off-patent pediatric drug development program to improve drug labels.  The NIH BPCA activities include the development of a clinical trial infrastructure to conduct off-patent pediatric clinical trials (the Pediatric Trials Network) and have led to more than 15-drugs/therapeutics prioritized for further study, as well as more than 46 clinical trials funded to improve the knowledge of medicines used in children.  Clinical trials conducted under the NIH BPCA program are submitted to the FDA and thus far have led to 19 label changes to improve pediatric labeling.

Contact details:
Perdita Taylor-Zapata M.D.
Program Director, Best Pharmaceuticals for Children Act Clinical Program
Program Officer, Obstetric and Pediatric Pharmacology and Therapeutics Branch
Eunice Kennedy Shriver National Institute of Child Health and Human Development, NIH
Email: taylorpe@mail.nih.gov

The CURE Drug Repurposing Collaboratory (CDRC) is a public-private partnership founded to explore whether already marketed drugs can be repurposed for diseases that are not commercially attractive. CDRC is interested in systematically capturing data from diverse populations, including pediatric patients, pregnant women, and under-served populations to evaluate approved drugs used for a new indication, identify candidates for repurposing, and inform the design of clinical trials of existing marketed drugs for new indications.  This real-world data is made publicly available through the CURE ID platform which enables the global health community to openly share patient treatment outcomes from prescribers that use existing drugs in new ways.

Contact details: cdrc@c-path.org

CURE ID is a collaboration between the US government health agencies, FDA and NCATS/NIH. It is a web and mobile tool for sharing novel uses of existing drugs for difficult to treat diseases. The goal of the platform is to identify potentially effective treatments for diseases with high unmet medical needs. Case reports are collected from healthcare providers, patients, the published literature, and electronic health record extraction to form an openly available database of real-world data on treatments with repurposed drugs. Initially focused on repurposing for infectious diseases, the platform has now expanded to encompass rare cancers and is progressing into capturing cases of rare genetic diseases and diseases affecting special populations (such as pregnant patients and neonates).

Contact details: heather.stone@fda.hhs.gov or curesupport@nih.gov

Hosted by the NHS, we bring together the national agencies responsible for researching, licensing, and assessing medicines in England. We provide tailored support to medicines that already have a licence for treating people in the UK and have the potential to be used in a new way that is not included in the current licence. For example, the drug might be used for a different illness, at a different dose, or in a new form. Potentially we can support phase 3 research, licensing, and patient access. For more information please visit Repurposing medicines in the NHS in England or email england.repurposing@nhs.net.

REMEDi4ALL is an EU-funded research initiative to drive forward new uses for investigational and approved medicines in Europe. The REMEDi4ALL consortium brings together a unique combination of expertise to address the complexities of drug repurposing. Under the leadership of EATRIS, the European infrastructure for translational medicine, 24 organisations in the fields of clinical and translational research, clinical operations, patient engagement and education, regulatory framework, funding, governance, Health Technology Assessment (HTA) and pricing and reimbursement will closely collaborate to make drug repurposing mainstream.

Contact details:
Our website does not show an email address via which REMEDi4ALL can be reached. Instead, it comes to us via a form, that ends up in the inbox of the following address: info@remedi4all.org
Visit this link to access the form directly.

RePo4EU is a EU funded initiative to build a Euro-Global platform for validated mechanism based drug repurposing, with the ultimate goal of spearheading the transition from imprecise drug therapy to AI powered precision medicine. Mechanism based drug repurposing will revolutionize the way we approach drugs, it will help us find new uses for already registered drugs, increase precision and radically cut down on costs and time in the drug development process. RePo4EU’s infrastructure will provide insights, training and matchmaking for the drug repurposing community at every stage in the value chain, from in silico to market access and will cover everything from freedom-to-operate to repurposing specific IP protection and business development, regulatory affairs, health technology assessment, clinical validation, ethics and data protection.

28 partners have joined forces to make RePo4EU a reality, pooling stakeholders and expertise at a global stage to create a “made in Europe”, fully fledged infrastructure for drug repurposing.

Contact details: info@repo4.eu