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Repurposed Medicines

Drug repurposing is the strategy of developing existing licensed drugs for new medical uses. Traditional drug discovery and development is a time-consuming, laborious, expensive, and high-risk process.

Drug repurposing has the potential to decrease the risk of failure in the development phase, shorten the time to market and reduce the development costs accordingly. Listen how Prof. Dr. Harald Schmidt, coordinator of the Repo4EU platform program for drug repurposing, explains the concept and value of drug repurposing:

It has enormous potential to quickly and cost-effectively help find therapeutics that could help treat chronic, high unmet need diseases and expand the repertoire of drugs available to improve human health. For most repurposed drugs a phase 2 trial followed by a phase 3 trial will be sufficient to obtain marketing approval in US and Europe. By reducing the average development time from compound identification to registration to 6 years and development costs to $300 million, there are significant economic advantages as traditional de novo drug discovery takes 12-16 year of development and $1-2 billion development costs.

In line with the Pharmaceutical Strategy for Europe, multiple initiatives are being launched to support
repurposing of drugs*. One of them being the creation of a dedicated regulatory pathway by EMA and the possibility of appropriately rewarding the investment and resources dedicated to developing a medicine based on a well-known substance, including by introducing proportionate regulatory incentives.

*Pharmaceutical Strategy for Europe (adopted in November 2020) https://ec.europa.eu/health/medicinal-products/pharmaceutical-strategy-europe_en

Current medicine is basically focused on symptom treatment. We lack insights on underlying disease mechanisms and therefore are far away from curing diseases.

The precision medicine at the moment is the answer to imprecision medicine that we currently have. We basically do not understand most of the diseases, therefore we have to wait until symptoms arise. We name the disease after a symptom and then we treat the symptoms. But because that does not cure the problem, the diseases become chronic, so we have a lot of chronic diseases.

Prof. Harald Schmidt, interview at the 2021 precision medicine conference in Rouen

Network medicine is a holistic approach trying to look at the whole system at once rather than trying to find a single “magic treatment bullet”. Causal disease mechanisms are identified, and low-dose drug combinations target the causal disease mechanism and reach a synergy effect. This is a big step forward versus traditional symptom-based therapy (upper panel) which treats symptoms independently of underlying disease mechanisms. This concept, mechanism-based drug repurposing, is a revolution in medicine, enabling to cure disease rather than to treat symptoms¹.

This concept, mechanism-based drug repurposing, is a revolution in medicine, enabling to cure disease rather than to treat symptoms¹.

symptom-based combination therapy
Differences between a mechanistic network pharmacology and a symptomatic combination therapy drug intervention*

* Nogales. C. et al. Network pharmacology: curing causal mechanisms instead of treating symptoms. Trends in Pharmacological Sciences feb 2022; vol 43, issue 2 : 136-150

Precision repurposed medicines combining repurposed drugs with a companion diagnostic which preselects eligible patients with specific underlying disease mechanisms, offers better predictability of clinical response and lower treatment failures, overall increasing cost effectiveness.