Background:
Last month EMA published its overview of key recommendations for 2020 on the authorisation and safety monitoring of medicines for human use. In total, the European Medicines Agency (EMA) recommended 97 medicines for marketing authorisation in 2020, which represents a 47% increase compared with 2019, when the Agency recommended 66 medicines. Of the 97 approved medicines, 39 have a new active substance.
How does that compare to 2019?
It is interesting that the number of orphan medicines, which received a positive recommendation by EMA increased more than 3-fold between 2019 with 7 orphan drugs and 2020 with 22 orphan drugs (Figure 1). Similarly, there were five medicines approved under exceptional circumstances in 2020 compared to just one in 2019.
Of note, the Agency recommended one vaccine (tozinameran, Pfizer) and one treatment for treatment of COVID-19 (remdesivir), which were key events for 2020 that many people anticipated given the times we are currently living in. Also, by the end of the year, one additional COVID-19 vaccine by Moderna was under evaluation by the Committee and there were two ongoing rolling reviews – one for the vaccine by AstraZeneca and one for the vaccine by Janssen Cilag.
Another trend that we can see is that there is an increasing number of PRIME therapies with three drugs being launched under this scheme in 2019 vs eight drugs in 2020. Six medicines received a recommendation for marketing authorisation following accelerated assessment, which gives them the benefit to be assessed by the EMA Committee within 150 days instead of 210 days.
Of the recommended medicines in 2020, 21 were indicated for oncology, followed by 14 for haematology and 11 pneumology/allergology as well as 11 treatments targeting infections.
What happens next?
It will be now interesting to see what the market access implications for these drugs will be, taking into account the significant financial and healthcare burden caused by the COVID-19 pandemic in all Member States.
In particular, it will be interesting to observe how HTA bodies will evaluate the products approved under exceptional circumstances, where the immature clinical data sets will likely result in a more limited evidence base to support a product’s value.
Additionally, the increase in orphan drug approvals, where manufacturers typically anticipate a high price, are likely to be closely scrutinized by HTA bodies to ensure that they clearly address an unmet need and have sufficient evidence to demonstrate patient benefits to justify the price.
The full Human Medicines Highlights 2020 report by EMA can be found here.