About this Research Topic
Drug repurposing or repositioning is defined as the process of finding new uses outside the scope of the original medical indication for existing drugs. It might relate to new patient populations, dosage forms or routes of administration. The drug candidate for a repurposed use could be a medicine already marketed, including old medicines with an expired intellectual property protection, or a drug under development for its first intended medical use, when it is discovered to be effective for the treatment of another condition. Drug repositioning is expected to reduce time, costs, and development risks. Utilizing approved drugs has been used in case of urgent needs, like COVID-19 pandemic when drugs for other viruses, anti-bacterials, immunosuppressants, anti-rheumatoid agents, anti-hypertensives have been studied or used in COVID-19 patients.
Repurposing represents an emerging and dynamic drug development strategy. In settings with special needs, like pediatric and rare diseases characterized by well-known challenges, repurposing is considered as an opportunity to cover unmet medical needs. As an example, off-label prescriptions from clinicians are considered as a unique therapeutic opportunity to treat patients when no alternative treatment is available. One of the most recent examples is within the pediatric oncologic field, where a drug may be useful for more tumors, due to its mechanism of action and/or its target. This has been recently discussed in the EU and US regulatory contexts dealing with medicines for pediatric oncologic diseases. Collaboration between academia and industry is usually established to undertake R&D processes of medicines with a poor commercial interest. However, only a limited part of off-label uses goes into a repurposing path and turns into an authorized use.
This Research Topic is dedicated to repurposing medicines to cover unmet therapeutic needs for small populations, like pediatric and rare disease patients. We aim to collect manuscripts as original research, review, mini review, policy and practice review, hypothesis and theory, perspective, clinical trial, case report, policy brief, brief research report, general commentary, opinion, on the developments, applications, and policies on repurposing for small populations. Key themes within this Research Topic on repurposing for small populations: - Industry and academic approaches as a R&D opportunity; - Scientific evaluations and regulatory decision-making; - Successes and failures of off-label uses gone into a repurposing path; - Commercial perspectives and strategies, including public-private partnerships; - Formulations to comply with the specific patient needs; - Covering needs for ultra-rare diseases; - Tools like Real World Data and pharmacometrics providing evidence to endorse drug use; - international and national initiatives to exploit repurposing pathways.
Repurposing represents an emerging and dynamic drug development strategy. In settings with special needs, like pediatric and rare diseases characterized by well-known challenges, repurposing is considered as an opportunity to cover unmet medical needs. As an example, off-label prescriptions from clinicians are considered as a unique therapeutic opportunity to treat patients when no alternative treatment is available. One of the most recent examples is within the pediatric oncologic field, where a drug may be useful for more tumors, due to its mechanism of action and/or its target. This has been recently discussed in the EU and US regulatory contexts dealing with medicines for pediatric oncologic diseases. Collaboration between academia and industry is usually established to undertake R&D processes of medicines with a poor commercial interest. However, only a limited part of off-label uses goes into a repurposing path and turns into an authorized use.
This Research Topic is dedicated to repurposing medicines to cover unmet therapeutic needs for small populations, like pediatric and rare disease patients. We aim to collect manuscripts as original research, review, mini review, policy and practice review, hypothesis and theory, perspective, clinical trial, case report, policy brief, brief research report, general commentary, opinion, on the developments, applications, and policies on repurposing for small populations. Key themes within this Research Topic on repurposing for small populations: - Industry and academic approaches as a R&D opportunity; - Scientific evaluations and regulatory decision-making; - Successes and failures of off-label uses gone into a repurposing path; - Commercial perspectives and strategies, including public-private partnerships; - Formulations to comply with the specific patient needs; - Covering needs for ultra-rare diseases; - Tools like Real World Data and pharmacometrics providing evidence to endorse drug use; - international and national initiatives to exploit repurposing pathways.
Keywords: Repurposing, small populations, orphan medicines, pediatric medicines, therapeutic needs
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