In 2023, the US pharma sector recorded the second ever highest level of new drug approvals since 2018. A total of 55 novel drugs were approved by the Food & Drug Administration’s Center for Drug Evaluation and Research (CDER), nearly 50% more than in 2022.
The number of approvals varies markedly by year with just over 20 novel drugs being approved in 2016, compared with about 60 products in 2018.
From 2013 through 2022, the CDER has averaged about 43 novel drug approvals each year. The CDER identified 20 of the 37 novel drugs approved in 2022 (54%) as first-in-class; that is, drugs having mechanisms of action different from those of existing therapies.
This compares with Europe, where the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended a total of 47 new medicines for approval by the second half of November 2023. This compares with 53 CHMP positive opinions in 2022, down from an exceptional 66 in 2021.
Fluctuations in US drug approvals tend to attract more attention than those in Europe. This is believed to be due to companies still treating the US as a key testing ground and as a market to establish favourable pricing and market-access strategies.
Many observers believe that this higher rate of US new drug approvals for 2023 allays fears that a tougher FDA stance on expedited submissions might slow innovation.
Nonetheless, there are indications that the less permissive stance is already having an impact in the US, and will continue to do so in years to come.
There was a stipulation that a drug approved under the accelerated approval pathway using a surrogate endpoint should later provide more advanced clinical data to confirm its benefit. But until recently, the FDA hadn’t been actively enforcing the mandate, allowing some cancer drug indications to stay for years without any confirmatory evidence.
Evidence generation for innovative medicines requires a global approach to meet regulatory requirements around the world. Evidence generation also continues long after the first registration of a new medicine. Some medicines benefit from increased support, such as enhanced regulator interactions, due to the level of complexity in development or unmet medical need and/or the ethical urgency. Expedited regulatory pathways (ERPs) were developed by multiple international authorities to respond to these needs. Enhanced early and frequent interactions facilitate development of innovative treatments and rapid processes to increase efficiency and reduce time, both in development and subsequent regulator assessment.
ERPs can be considered a ‘regulatory toolbox’ that can be leveraged to best support the development and efficient assessment of important innovative treatments, so patients can receive them in a timely manner.
No similar initiatives have emerged in the EU to date, however, although expedited regulatory pathways (ERPs) are used less frequently than in the US. In fact, the European Commission’s wide-ranging revision of pharmaceutical legislation does include proposals to reduce the time taken to evaluate new medicines centrally in the EU.
Back in April 2023, the European Commission published the three times-delayed draft of legislation that could reshape the regulation of the pharma sector for years to come.
The draft was the product of a lengthy debate that has seen politicians disparagingly call the delays ‘a huge victory for the pharmaceutical lobby’ while the trade group EFPIA warned that a leaked, earlier draft would ‘irretrievably sabotage’ the industry and ‘send Europe to the back of the queue for healthcare treatments, clinical research, jobs and global investment’.
Stella Kyriakides, the European Commissioner for Health and Food Safety, hailed the proposal as tackling the ‘silent pandemic’ of antimicrobial resistance by offering incentives for novel drugs; providing concrete action on shortages with clearer obligations for companies and more transparent supply chains; and creating a single market for medicines with leaner and faster rules that keep pace with science and reduce costs for business.
‘It’s not a secret that big member states up to now had better chances to obtain [certain drugs] faster. But this should not create a two-tiered approach of first- and second-class citizens. We want to change it to a smart system of incentives that replaces the current blanket protection system for protecting medicines in the single market, so the more you are compliant with enhancing market access, the better protected your products are in the single market on affordability,’ said Margaritis Schinas, Vice President of the Commission.
The EMA would have to assess products within 180 days (150 days for medicines of ‘major public health interest’), down from 210 days at present. The European Commission would then have 46 rather than 67 days to give its seal of approval.
So, what about 2024? A recent report from the IQVIA Institute for human data science suggests that global use and spending on medicines is now exceeding pre-pandemic growth rates and is expected to continue significantly above those trends up to 2028.
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