Find out more about the hot topic of drug repurposing in regulatory affairs

Following on from an in-depth article in TOPRA’s Regulatory Rapporteur, this news item outlines our company’s additional views on the rationale behind drug repurposing.

“Drug repurposing allows us to dramatically reduce the cost of the early stages of drug development and accelerate progress to clinical testing.”

Drug repurposing, or repositioning, is the process of identifying a new use for an existing medicine in an indication which is not included in the original marketing authorisation. It is an area of great interest in regulatory science as it has the potential for major public health benefits. Current European regulatory frameworks incentivise the repurposing of approved medicines, however additional systems are required to tackle some of the regulatory challenges experiences by drug developers.

The current framework offers provisions such as new therapeutic indications for approved medicinal products or well-established substances, paediatric use marketing authorisations (PUMA), and orphan drug designation. In recent years, various initiatives have been started to support these provisions:

  • The Safe and Timely Access to Medicines for Patients (STAMP) expert group was set up in 2015 to improve the safe and timely access, and availability of medicines to patients.
  • The European Medicines Agency (EMA) has drafted a plan for enhancing the development and implementation of a repurposing framework. (section 3.4)
  • Increasing the number of scientific advice meetings with regulators such as the EMA and UK Medicines Health and Regulatory Agency (MHRA), which provide access to expert regulatory advice and guidance for organisations developing innovative medicines.
  • The UK Early Access to Medicines Scheme (EAMS), launched in April 2014, aims to give patients with serious conditions access to medicines without a marketing authorisation, when there is a defined unmet clinical need.
  • The Accelerated Access Collaborative (AAC) was formed to allow NHS patients to be among the first to get life-changing treatments, by “removing barriers to the uptake of innovation”, such as high costs. Life-changing treatments could include medical technologies, diagnostics or repurposed medicines.

Medical research charities are also working to deliver life-changing medicines for rare diseases, however, with limited funding, they are unable to reach their targets alone. They require access to resources, knowledge and skills to advance their projects, which can be provided by the appropriate regulatory authorities such as the MHRA.

Developers involved in a drug repurposing programme should allow time to conjure the best strategic approach for their condition, and fully comprehend the routes open to them. It is critical to identify regulatory “bottlenecks” in the system to ensure the work of concerned organisations can be used to facilitate enhanced drug repurposing, potentially delivering life-saving treatments at lower prices.