The pandemic has placed drug repurposing high on the agenda, with medications such as the steroid dexamethasone and rheumatoid arthritis treatment tocilizumab being used to treat patients severely ill with Covid-19.
Charities and patient groups seeking treatments for rare diseases are often particularly interested in the potential of new uses for existing drugs because they offer the possibility of lower overall costs and shorter timeframes to get treatments to patients and licensed medications already have known safety profiles.
The guide, Repurposing medicines: the opportunity and the challenges was launched today at the Findacure's virtual Drug Repurposing for Rare Diseases Conference 2021 and has a range of practical tips and advice for overcoming potential barriers including:
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Seeking scientific advice from the regulator
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Developing an intellectual property strategy
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Collaborating with industry on drug repurposing opportunities
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Taking steps to ensure medicines are made available to patients
Commenting on the report LifeArc CEO Dr Melanie Lee, CBE said:
The pandemic has shown what is possible in the field of drug repurposing. However, we know that not–for–profits and researchers can be confused by the complex commercial and regulatory challenges that repurposing presents. We want to improve awareness of those challenges so that applications for repurposing proceed smoothly – making it more likely that life-changing medicines will reach patients in the clinic. That translational science is LifeArc’s area of expertise – ensuring that successful lab research results in new treatments or therapies for patients as quickly as possible.”
Nicole Jadeja, life sciences partner at Pinsent Masons, said:
“We are delighted to have partnered with LifeArc on this important project and hope that this will provide practical advice to help convert more repurposing projects into new treatments for patients. One thing that really stands out is how important collaboration is in delivering on the promise of repurposing and drawing upon the experiences of the wide range of stakeholders in the guide."
The guide provides clear examples of the benefits of finding new uses for existing medicines including the repurposing of the breast cancer drug tamoxifen for treating X-linked myotubular myopathy (a rare neuromuscular disorder that mainly affects boys), using a vertigo drug as a treatment for certain rare and devastating neurodegenerative diseases and the successful repurposing of a drug called rapamycin which has transformed the lives of women with lymphangioleiomyomastosis (LAM).
Findacure CEO Dr Rick Thompson said:
"In the world of rare diseases, millions of patients live with no treatment. Drug repurposing offers a quick, cheap, accessible and patient-friendly route to develop new treatments for rare diseases, and in recent years the field has grown steadily. However, we must address roadblocks – including a lack of financial incentive for industry and a lack of regulatory knowledge in academia. We can't wait to get working with LifeArc to deliver change. COVID-19 has shown what can be achieved in repurposing with the right funding and government incentives. Now, those benefits must reach the world's 350 million rare disease patients."