How long does it take to get a drug approved?

The COVID-19 pandemic opened the world’s eyes to the clinical trial process—and the importance of investing in research and development to get new prescription drugs to patients. And while the world is grateful for the speed with which the biopharmaceutical industry has developed COVID-19 vaccines and therapeutics, this kind of speed is uncommon, as a new report explains.

BIO’s forthcoming report, Clinical Development Success Rates and Contributing Factors 2011 – 2020, takes a look at how long does it normally takes a new drug to be developed and approved and how likely? The report was published in conjunction with Informa Pharma Intelligence and QLS Advisors LLC.

Here are some key takeaways from the new report:

  • On average, it takes 10.5 years for a Phase I program to progress to regulatory approval.
  • From 2011–2020, a drug in a Phase 1 clinical trial had a 7.9% likelihood of approval (LOA).
  • Of the 14 major disease areas, hematology therapies had the highest LOA from Phase I (23.9%). This is seven times greater than the least-successful group, urology (3.6%).
  • Rare disease therapies were also notably successful with an overall LOA of 17.0%.
  • Immuno-oncology therapies provide a rare pocket of success in oncology R&D with an overall LOA of 12.4%, compared to 5.3% for all oncology approaches.
  • Development programs with trials employing patient preselection biomarkers are twice as likely to be approved (15.9%). Nearly one in two that reach Phase II are successful.

Clinical development success rates, five years on

In BIO and Informa’s 2016 report, the average likelihood of approval (LOA) was 9.6% for an asset from Phase I to approval based on data from 2006 to 2015. Now, the LOA has decreased to 7.9% based on data from 2011 to 2020.

This decrease can be attributed to two main factors: A greater participation from smaller firms participating in drug candidate development and their willingness to disclose early failures.

“That all ties into what we see—which is a lower success rate overall from our last paper in 2016,” he continued. These rates do not change much over time. “The reason we waited five years to publish an update is because this data doesn’t really change year to year,” David Thomas, BIO Vice President, Industry Research, explained regarding the gap between reports.

The impact of COVID-19 on clinical development in 2020

There have been “close to 900 programs launched for COVID-19 since January 2020,” Thomas explained. In 2020, “biotech had a successful year, we did get two very efficacious and safe vaccines [Pfizer-BioNTech and Moderna], and potentially a couple more in the next two months out of that progress.” Noteworthy successes besides the vaccines include the novel Regeneron and Eli Lilly antibody therapies as well as the Remdesivir antiviral.

But while there were noteworthy successes in 2020, there were also many failures. Some programs were suspended due to COVID-19 and some COVID-19 programs failed. To formally record a failure, the company must fully suspend a program and disclose that they dropped it. For some programs, clinical trials fail but companies go back and try again or pause the program operations indefinitely. Less of the latter was seen in recent years.

Clinical development beyond COVID-19

Thomas also noted the growing importance of biologics as a part of research and development as well as the rise in rare disease research, which has contributed to a heightened success rate.

Another notable data point among the high LOAs is the inclusion this year of CAR T-cell therapies in oncology treatment, which while these treatments existed at the time this report was first published in 2016, they were only just starting to enter human trials.

Yet another key cancer development not included in the 2016 report was the development of immune-oncology drugs, such as PD-1 immune checkpoint inhibitors.

Development programs whose trials employ patient preselection biomarkers, ultimately resulting in two-fold higher LOAs from Phase I. The incorporation of patient preselection biomarkers in clinical trial design has undoubtedly been a revolutionary development for drug candidates.

Looking ahead

Ultimately, drug development remains difficult with far less than 1 in 10 clinical drug programs ever reaching patients. It usually takes 10 to 10.5 years to develop a vaccine, which makes the existing COVID-19 vaccines on the market all the more incredible. This is why success requires “many shots on goal,” says Thomas, noting there have been more than 200 COVID-19 vaccines in development. All the public really sees right now are the two existing vaccines—but of course, many more are working their way through the clinical trial process.

Beyond COVID-19, especially in rare disease drug development and breakthrough oncology treatments, there are many reasons to be hopeful regarding the outcomes of this report and what this means for future patient care.

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