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:

https://www.ema.europa.eu/documents/scientific-guideline/concept-paper-revision-guideline-evaluation-anticancer-medicinal-products-man_en.pdf

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.

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.

Academia:
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.

CROs:
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.

Do you have further idea how can this novel approach change pharma insutry? Please let me know in comment.

Marketing Authorization Transfer (MAT) can cause seriuos extra workload on every related departments. As a consequence sometimes there is not enough time to ensure tha cross-departmental communication flows efficiently and important details can be missed out. Most of the administrative work is done by the Legal and Regulatory Departments but clear communication among Pharmacovigilance and IT is also essential.

If you have only a few affected products, it is usually not a big deal to handle the process. However, when a bigger portfolio moves even experienced Regulatory professionals can forget about the importance of pre-defined rules between companies.

Just consider the following few points and you will see what is the issue:
• The approval process of bigger transfered portfolio can last longer than expected. Are you prepared for handling some products even long after the official deadline determined in the MAT contract?
• You need to be really lucky to transfer only products where no other assessment is ongoing. You need to agree with your MAT partner how to handle the transition in these cases.
• Companies track regulatory submissions really differently. Consult with you IT department and check the partner’s system. Maybe they don’t track the workflows or have different steps/deadlines which needs to be syncronized.
• Your compliance measurement points might track different actions compared to the partner. Who will be on charge when an issue pops up?
• You have 30 days reporting deadline for XEVMPD (EU countries) after MAT. Will you receive every necessary documents in time?

And these are just a few points on my list what I check while preparing for a MAT.

Sometimes it is worthy to consider the involvment a third party consultant in order to mediate the MAT. Providing access to the consultant for the Transferor’s and Transferee’s system can be really helpful as she/he will have a comprehensive overview on the overall tasks.

Now you should check your MAT process again and determine what kind of tasks are really essential to the successfull transfer.