Is paradigm shift is close to us? – EMA’s public consultation on evaluation of anticancer medicinal products
European Medicines Agency (EMA) recently published an open consulation about the evaluation of anticancer medicinal products.
The concept paper anticipates that EMA considering to change the current assessment procedure from indication (location) based approval to the pathological mechanism approach.
For details please check the original document:
The partial implementation of this new approach had been already made in the US. In 2017, FDA approved pembrolizumab for the treatment of adult and pediatric patients with unresectable or metastatic solid tumors that have been identified as having a biomarker referred to as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).
For the first sight maybe it is not a big change but it has really deep impact on our current system.
By the time of approval of pembrolizumab, every other anticancer treatments were approved based on the origin of the cancer cells. That is why we call them skin, brain or lung cancer. Due to this approach clinical trials had to be conducted for brain or lung or skin cancer separately, even if the common biochemical and pathological routes were proven. As a consequence the Indication section has became enormous.
If EMA truly consider acceptable to implement new this assessment approach this will change and we all need to reevaulate our current practices within the full spectre of pharmaceutical industry.
Repurposing of currently owned drugs will work fine and the companies can win dozens of patents but this won’t work too long. Find new signaling pathways will be essential and in silico start-ups will be really valuable in the race.
Point-of-care testing (POCT) devices will produce huge amount of money. For every medicine a test will be needed to prove that the medicine fits into the indication. If the company can develop a good POTC and sell along with the medicine (as some company has already started) the share-holder will be really satisfied.
Medical experts and regulatory officers needs to be prepared as all new regulations as conseqence will keep them busy for a while.
Biochemistry studies will be strongly supported in order ot detect and prove all type of cancer signalling pathways. To make this possible the instrumental background should be improved fast. Currently only 20% of protein-protein interactions are revealed and the most popular high-throughput methods creates many false positive findings. Well designed and characterised in vitro cell line will be alse greatly appreaciated.
This is a great opportunity to the research groups to create some outstanding result and develop cooperation with industry.
Clinical trial settings planning and the trial inclusion and exlsusion criterias will be based on biomarker tests. Maybe less trials will be started but the laboratory background will be more important. The biostatistical model also needs to be tailored accordingly.