Citeline’s annual Clinical Trials Roundup demonstrates the robust growth of clinical trial initiations in 2023, following a decline of 12% in 2022.
The biopharma industry weathered post pandemic adjustments, endured economic hardships, slowed growth in major markets, met regulatory pressures to incorporate diversity, equity, and inclusion (DE&I), and suffered the impacts of geopolitical conflicts.
While the “COVID effect” peters out over the last two years, clinical research in COVID-19 is here to stay as a mainstay within the infectious diseases (ID) therapy area. Going forward, we may have reached the point where we can scrap the “COVID effect” in future analyses.
Industry-sponsored trials, which accounted for over 70% of all trials initiated in 2023, have been a driving force in clinical research.
The growth of industry sponsored trials during pre-pandemic years (2017–19) somewhat mirrored that of the total trial growth in Table 1.
In 2020, that trend broke due to the epic rise of non-industry-sponsored COVID trials (26% total trials vs. 5% industry trials). However, excluding COVID trial counts significantly narrowed that gap (-4% total trials vs. -8% industry trials). The huge gain of 22% from excluding COVID trials in 2021 was largely non-industry-sponsored, because only 6% were accounted for in industry-sponsored trials.
Exhibiting 13% growth excluding COVID-19 trials, 2023 also a cut above the average growth rate of 7% for non-COVID trials during 2017–23.
Will the oscillating cycles between growth and decline continue, or is the biopharma industry on track to full recovery?
Clinical Trial By Therapeutic Area
The lineup of therapy areas (TAs) by trial initiations has remained relatively unchanged since 2020, when the COVID-19 pandemic boosted the ID TA to second place, and it remains at this ranking to this day. Oncology continues to have a distant lead over all other TAs, as cancer remains the most prolific area of research.
ID trials continue to recede as they have since 2021, narrowing the ID TA’s lead over CNS. With the renewed boom in neurology and recent success in obesity drugs, it may soon be a toss-up between CNS and metabolic/endocrinology to snatch away the second position from infectious diseases. It is also worth noting that the number of genitourinary trials went up by 100 in 2023, raking in 61% growth, the largest expansion rate of all TAs.
Top Disease Areas For 2023 Trial Initiations
Each year, Citeline surveys the top contenders of the most active diseases in clinical trials. The Number one spot in Exhibit 3 was finally reclaimed by unspecified solid tumors in 2022, and it retained its rank in 2023. The biggest upset is non-small cell lung cancer (NSCLC) ascending to second place, helping push COVID-19 down the rank from second to eighth place (Exhibit 3). Oncology diseases comprised six of the top 10 list in 2023. Type 2 diabetes (T2D) had an impressive ascent from sixth to third place this year.
The established success of Ozempic (semaglutide), Trulicity (dulaglutide), and Farxiga (dapagliflozin) has brought fresh focus to this disease area, with additional promising contenders Mounjaro (tirzepatide) and Rybelsus (semaglutide) emerging with impressive performances in 2023. Both respiratory infections and colorectal cancer moved up two positions, but non-Hodgkin’s lymphoma (NHL) fell by two.
This year, we welcomed new joiners' hypertension and pancreatic cancer to round out the top 10, displacing respiratory vaccines and nociceptive pain from last year. Given the large oncology presence in the top 10 diseases, most of its clinical development is early-phase heavy (Phase I–II).
Unspecified solid tumors, NSCLC, breast cancer, and colorectal cancer are the hotbeds of trial initiations. Type 2 diabetes also had significant trial initiations in Phase I. As we turn our eye to Phase III trials, therapy area diversity becomes more prominent, given the top diseases here consisted of Type 2 diabetes, breast cancer, and respiratory infections.
Rare Disease Trials
Clinical advances in rare diseases must not be overlooked. Citeline defines a rare disease as one with a prevalence of below 1 in 2,000 people in the EU or affecting fewer than 200,000 people in the US (equivalent to around 1 in 1,600 people). The steady climb over the last decade in Exhibit 4 peaked in 2021 with 3,581 trials but dipped back down to pre-pandemic levels in 2022 and 2023. As clinical trials become more complex and expensive over the years, this is sharpened for rare diseases, as these inherently niche areas face harder circumstances with patient enrolment, retention and follow-up.
The steep setback of rare disease trials in 2022 (-14%) wiped away the rebound gain in 2021; however, the decline in 2023 was less severe at -5% with 2,914 trials. Rare disease trials represented 29.3% of the total 2023 trial initiations, which is consistent with its proportion in prior years, ranging between 27% and 30%.
The growing importance of rare diseases has pulled together community lobbyists, patient advocates and drug developers, driving the influence behind the US Food and Drug Administration’s (FDA) creation of the rare disease “hub,” which will function as a Center of Excellence, much like the Oncology Center of Excellence it instituted in 2016.
The hub will aim to provide needed regulatory flexibility for rare disease drug development. The decrease in trial initiations during 2023 may seem to hinder post-pandemic recovery, but the hub’s emergence could reignite momentum.
Rare disease R&D remains a strong focus in the healthcare industry because of the debilitating nature of these conditions. The number of targeted rare diseases continues to rise, reaching 761 in 2023, as reported by Citeline’s Pharma R&D Annual Review 2024. This translates to a 7.6% rise in the number of drugs under development from the prior year, representing 30% of all drugs in the pipeline.
Geographical Survey Of Trial Activity
The last 10 years have witnessed a remarkable increase in clinical trials across Asia, with China as the primary driver, followed by Japan and South Korea.
For many years, the US had consistently reigned as the leading clinical trial market with China trailing closely behind. A significant shift occurred in 2021, when China overtook the US to become the leading market, a position it has held to date.
War also has a devastating impact on health care infrastructure, as hospitals, clinics, and medical supply chains are often targeted and destroyed in conflict zones. The consequences are far reaching and long lasting, exacerbated by limited access to lifesaving medicines and a shortage of healthcare workers due to displacement. Recognizing the massive loss of clinical trial momentum, Citeline will reassess the outlook for these countries next year.
