Editorial Medicalfacts / Janine Budding October 24, 2023 – 11:20 PM
In two publications in the renowned scientific journal Nature Reviews Drug Discovery Anneliene Jonker (TechMed Center) emphasizes the importance of care for rare diseases and the innovation that is needed. The personalized medicine program manager shows how drug repurposing (reuse of medicines) can offer a solution for rare diseases, and also how developments in innovative therapies for rare diseases are influenced by personalized medicine.
Around the world, there are many different definitions for when a disease is rare. As a result, no one knows exactly how many rare diseases there are. This makes it difficult to efficiently arrive at good treatments. By thinking about that definition and, for example, combining certain subgroups, you can maximize the impact of treatments.
Different definitions
“There are about ninety different definitions all over the world, the World Health Organization is also working on such a definition,” says Jonker, “But how this definition is influenced by increasingly looking at the individual, as happens with personalized medicine We actually didn’t know that. We looked at how we increasingly break down common diseases into small subgroups. That way sometimes you go towards a rare entity. For example, a more common type of cancer can become a rare disease because we divide it so far with specific biomarkers. Finally, we looked at shared molecular characteristics, which means that some different rare diseases can still be treated in the same way. “
Reuse of medicines
In her second publication, Jonker looked at ‘drug repurposing’. That is reusing medicine for another disease. “The best-known example is Viagra; which was developed as a medicine against cardiovascular disease, but is now better known as a medicine against erectile dysfunction.”
Developing medication for a rare disease is difficult. There are few people to conduct clinical trials with and also little expertise about the course of the disease. As a result, it costs a lot of money and time to develop a medicine for a rare disease. By reusing a medicine, you need much less money and time for development. This can be a solution for rare diseases.
Overview of initiatives
To this end, Jonker mapped out which initiatives have already taken place worldwide to make reuse possible. “If you know what is possible, you as a developer can prepare a better route for the development of reuse of a medicine.” For her publication, together with an international group of experts, she made an overview of initiatives, including checklists to see when the best time is to make use of those initiatives. In this way, reuse of medicines can shorten the development of a medicine for a rare disease from an average of thirty years to about ten years.
More information
Dr. Anneliene Jonker is Personalized Medicine program manager at the Technical Medical Center. She is also vice-chair of the Therapies Scientific Committee of the International Rare Diseases Research Consortium (IRDiRC). She published twice in the scientific journal in a short period of time Nature Reviews Drug Discovery titled ‘Defining rare conditions in the era of personalized medicine’ in ‘IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases’.
Source: UT
Editorial Medicalfacts / Janine Budding
I studied physiotherapy and health care business administration. I am also a registered independent client supporter and informal care broker. I have a lot of experience in various positions in healthcare, the social domain and the medical and pharmaceutical industries, nationally and internationally. And I have broad medical knowledge of most specialties in healthcare. And the healthcare laws from which healthcare is regulated and financed. I attend most of the leading medical conferences in Europe and America every year to keep my knowledge up to date and to keep up with the latest developments and innovations. I am currently doing a Masters in applied psychology.
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2023-10-24 21:56:09
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