As the global Covid-19 pandemic enters its third year, the life sciences innovation system is setting new records in the level of investment, activity, and scientific progress, according to the IQVIA Institute. In its report, Global Trends in R&D: Overview through 2021, the IQVIA assesses the trends in new drug approvals and launches, overall pipeline activity in terms of actively researched medicines, and the number of initiated clinical trials. It also profiles the state of R&D funding and the activity of companies of different types, and the results of research are compared to the input effort in a Clinical Development Productivity Index.
1. New drug approvals and launches
A record 84 novel active substances (NASs) were launched globally in 2021, double the number of five years ago. Among the 72 NASs launched in the US in 2021, a record 44 (over 60%) were deemed by the FDA as “first-in-class” — drugs that use a new and unique mechanism of action for treating a medical condition — and more than half (40) carried an orphan drug designation for patients with rare diseases.
A range of clinical trial designs was used to support regulatory submissions in 2021, reflecting the diversity of therapeutic innovation approaches to clinical development. This included increased use of real-world evidence as part of FDA approval decisions. Achieving appropriate race and ethnicity representation in the clinical trials used as the basis for US FDA approval remains a challenge, though some progress is seen among recently approved NASs.
2. R&D pipeline
The total number of products in active development globally exceeds 6000, up 68% over the 2016 level. This shows how the life sciences sector continues to invest in and advance innovative therapeutics and vaccines across a wide range of disease areas despite the Covid-19 pandemic disruption. Pipeline expansion has been most significant in oncology, gastrointestinal disorders, and neurology. While infectious diseases and vaccines had declining numbers of pipeline products until recently, they saw significant growth, largely driven by Covid-19-related treatments and vaccines. Almost all of the oncology pipeline is some type of targeted therapy. More than 40% of the pipeline is for rare cancers for which next-generation biotherapeutics — including cell and gene therapies — are increasingly being deployed.
3. Clinical trial activity
Clinical trial activity has been sustained through the pandemic, as the industry has adapted and developed new approaches to enable research to continue. Last year saw 5,500 new clinical trial starts, a 14% increase over the number started in 2020 and 19% more than started in 2019. While the number of intended clinical trial starts dipped significantly in early 2020, the recovery began in June 2020 and has increased to record levels since then. This excludes the surge of clinical trials related to Covid-19 vaccines and treatments, which has resulted in over 1,200 industry-sponsored interventional trial starts since the beginning of 2020.
4. Clinical development productivity
One measure of clinical development productivity is to relate molecule success rates to the complexity and duration of clinical trials. Efforts to improve productivity are largely focused on these areas. By these measures, clinical development productivity fell to its lowest point in 2021. The decline was driven by a lower success rate that was offset by reduced trial complexity.
Across disease areas, 2021’s composite success rate fell below the 10-year trend except for vaccines and cardiovascular, although the probability of success varied considerably across diseases. The low success rate reflects efforts by drug developers to take on tougher clinical challenges in the hopes of achieving clinical breakthroughs, even though these remain rare. These lower success rates have been offset by higher volumes of investigational drugs in recent years. These factors have resulted in a higher number of new drug approvals and launches.
5. R&D funding
Venture capital deal activity and investment flows accelerated in the past two years as interest in life sciences intensified. Specifically, in 2021, there were more than 2,000 deals and over $45 billion of deal value. The 15 largest pharmaceutical companies invested a record $133 billion of R&D expenditure in 2021, representing an increase of 45% since 2016 and a level of investment close to 20% of their recorded sales.
Over the past five years, deal activity has shifted geographically to include more companies headquartered in China, Korea and other APAC countries, and fewer Europe-based companies. North American companies represent the largest number and total increase of deal activity over the five-year period.
6. Emerging Biopharmaceutical (EBP) Companies’ contribution to innovation
Emerging biopharma companies — those with an estimated R&D expenditure of less than $200 million and annual revenue of less than $500 million — are responsible for a record 65% of the molecules in the R&D pipeline. That’s a significant increase compared to less than 50% in 2016 and 33% in 2001. EBPs headquartered in China now account for 17% of the EBP innovation pipeline, up from 6% just five years ago. While 20% come from Europe-based companies and 46% from U.S.-based companies, both have lost share over the same period.
7. Accelerators of innovation cycles
Novel trial designs have been embraced as a means to accelerate innovation and are now incorporated into 8% of new trial starts. That equated to roughly 400 trials in 2021, with oncology trials responsible for almost 60% of this activity. The median time from first patent filing to launch for US NASs fell to its lowest level in 2020 and 2021. This includes 21 drugs that were launched less than five years into their patent terms, compared to 16 over the prior nine years. Reducing the “white space” — the difference between the time a molecule takes to progress through clinical development and its clinical trial duration — is a major area of focus.
In the case of Covid-19 vaccines, pivotal trial white space and execution timelines were reduced by 70% — an estimated 26-month time savings over benchmark. The main time-intensive elements of clinical trials include partner selection, trial startup, patient enrollment, database lock, and analysis. Key factors that enable faster trial cycles are regulatory allowances, patient interest, leadership focus, and government funding that de-risk critical decision-making, process innovation, and resource utilisation agencies.