BRUSSELS - Rare cancers are difficult to cure. The need for more research on treatments is pressing. Precision medicine seems to be the way ahead. But there are more options. The repurposing of non-cancer drugs can extend the search space of drug targets.

The Anticancer Fund is actively participating in the 29th EURORDIS workshop Round Table of Companies on Wednesday, 19 February 2020, entitled: How to teach an old medicine new tricks. The importance of repurposing medicines for patients. Lydie Meheus, managing director of the Anticancer Fund, and Ciska Verbaanderd, PhD Student, both participating to the workshop, will stress the importance of repurposing medicines as additional solutions to treat rare cancers.

200 rare cancers

22 percent of the cancer patients are diagnosed with a rare cancer. There are approximately 200 different indications, including pediatric types of rare cancers. Just as for other rare diseases commercial development of new and specific treatments is not always rewarding for the pharma industry. Curing a rare type of cancer requires precision medicine. Tailormade treatments, that are of no interest for commercial parties.

Financial incentives such as the orphan drug regulation tried to resolve this issue, but more and more they result in unaffordable price settings. An example of this problem is Zolgensma, the first gene therapy for spinal muscular atrophy in babies. Life saving but too expensive. Therefore we think it is important to join forces with all partners concerned about the patients suffering from less common illnesses.

Creative solutions

Therapies that have come to market so far cover less than 5 percent of all rare diseases, demonstrating a need to speed up the development of new treatments. The pace at which science is revealing the underlying biology of many of these diseases inspires scientists to come up with creative solutions based on existing drugs targeting the pathways involved. Innovation can lie in products that are already authorised and that can target a different condition. Counterintuitively clinical development of these drugs for a new rare indication is still expensive. A ‘third party’, being a foundation or academia, could perform and fund the clinical research with these off-label drugs. The problem? The regulatory framework. In the current legal European framework only the owner of the drug (the Market Authorization Holder) is empowered to make the drug legally available for the patients and bring it to on-label use.

Doctors worldwide use marketed drugs off-label to treat rare diseases. The outcome of these treatments should be registered and shared, so that the scientific community can learn from it. This data, gathered in a non-competitive matter, could be a source of inspiration to start clinical trials with existing drugs for new indications. This would lead to the discovery of new treatments that could be further developed with public and philanthropic support.

The workshop has clear objectives: the ambition is to have 3 to 5 times more new therapies for rare diseases approved per year by 2025. The Anticancer Fund adds some more for rare cancer types: the new treatments should be safely available, rapidly accessible and affordable for all.

You can have a look at the program of the EURORDIS Round Table here.

Note:

EURORDIS is a non-governmental patient-driven alliance of patient organisations representing 894 rare disease patient organisations in 72 countries. It is the voice of 30 million people affected by rare diseases throughout Europe.

Read the concept paper of EURORDIS: