Consultant Mira about the treatment of rare diseases
Looking at the media and the current sentiment in the world nowadays, being dominated by news regarding COVID-19, people often tend to forget that the pharmaceutical industry handles more than just “popular” diseases. Worldwide more than 7000 rare diseases have an impact on over 300 million people. These incredible numbers show the urgent need to invest time and resources in this field of research, because everyone deserves to live a healthy and happy life regardless the rarity of their disease treatment.
In the light of Rare Disease Day, I wanted to emphasize the fact that behind the scenes a lot is being done for the treatment of rare diseases. My job, as a Local Trial Manager, contributes to this wonderful cause which I hold very dear. On my behalf: welcome to the world of clinical studies!
It might surprise you, but Rome and a newly developed medicine have something in common: they weren’t (and aren’t) built in one day. In fact, most of the time it takes at least 10 to 12 years. The story usually begins in a laboratory where a new potential molecule is tested on animals. Once researchers have approved its potential of becoming a future medicine, the drug is tested on humans in 4 different phases before it eventually becomes available on the market. Without going too much into detail, all of this is executed to ensure the safety and the efficacy of the medicine with the principal question: will it really improve the patients’ health and make the world a better place for everyone.
At Boehringer Ingelheim, A Local Trial Manager (LTM) is responsible for the start-up phase of a clinical study in his or her own country (in my case Belgium). Once the potential new medicine has been tested and approved in the lab, it will have to be studied on humans. It is my job to search for potential hospitals and doctors willing to test the new drug on their patients. Once the hospitals are selected, the Belgian Regulatory Authority (the FAMHP) and an Ethics Committee (EC) must give their approval to conduct the Clinical Trial in the hospital.
As an LTM it is of great importance to prepare all necessary documents in order to receive approval of the FAMHP and EC. Once approved, the testing on patients can start when I handover the knowledge of the Clinical Trial to a Clinical Research Associate (CRA). The CRA will further monitor the study on-site to ensure that all procedures are executed correctly following the study protocol. When adaptations or questions arise on the protocol, my role is to make the necessary changes to the documents or to answers the posed questions.
Currently I’m setting up a CT to study a medication for an illness that is estimated to affect about 7 people per million people (so quite a rare disease you could say). The future of science and pharma is looking brighter and promising every day, allowing research in rare diseases to improve. We may not hear about it often, but because you don’t see it doesn’t mean it is not happening. The fact that I’m able to contribute to these developments at the pharmaceutical company Boehringer Ingelheim brings me a lot of joy and satisfaction. There’s nothing more fulfilling than receiving data about a patient which you helped participating in a CT, and seeing that their health is improving. Everyone deserves the chance to battle their diseases, however rare they may be.
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