The biopharma industry was certainly not immune to the turbulence brought about by the COVID-19 pandemic.
The pandemic disrupted — and in some cases, stopped completely — clinical trials around the globe. In fact, as of January 2021, more than 2,000 clinical trials had been halted explicitly due to COVID-19 restrictions.
In addition to trial delays, many biopharma companies jumped into the fight against COVID, causing other development projects to slow or come to a stop.
But in the midst of all the disruption, the industry accomplished the incredible: developing, testing, and manufacturing some of the most impactful innovations in modern medicine — in under a year. While the industry definitely had some big wins in vaccines and therapeutics, other advancements made during the pandemic may have a lasting impact on the biopharma industry.
Here are just a few ways the biopharma industry’s pandemic response has opened the door to more therapeutic opportunities.
Modernizing the Clinical Trial Paradigm
Clinical trials are the key to the drug development process, representing a significant portion of the overall R&D budget in the biopharma industry. As such, if companies can improve their trial models, they can improve drug development as a whole.
Discussion surrounding the push to modernize clinical trial models has for years included talk of “trial decentralization.” Decentralization involves bringing a larger proportion of a trial’s activities to the patients, rather than sticking with the more traditional paradigm of bringing patients to the trial site.
The concept of decentralized clinical trials is not new to the biopharma industry. In fact, the digital technology needed to enable more remote, virtual clinical trials has been available for years. And yet, adoption has been slow. Limited experience with the approach and the perception of regulatory barriers using data gathered from decentralized trials has held back progress.
But COVID-19 helped set aflame this long-simmering trend. During the pandemic, trial sponsors were forced to build capabilities and processes for handling clinical trials more remotely — for example, by turning to virtual recruitment or using telehealth rather than site visits to monitor trial participants.
The U.S. FDA did its part by releasing new guidance for clinical trial conduct, with allowances such as:
- Using telehealth to limit in-person visits and follow-up appointments
- Shipping oral medications to participants’ homes, rather than requiring the medications to be picked up at a clinic
- Using outside labs to conduct tests closer to where participants live
Many in the industry are optimistic that the decentralized practices implemented during the pandemic will continue once the pandemic subsides, benefiting future clinical trials and making them more accessible to a wider range of participants.
In particular, clinical trials for new cell and gene therapies, especially those addressing rare diseases, present a unique set of challenges in terms of eligibility screening, recruitment, and geographic logistics. A decentralized trial model and the better utilization of digital tools could reduce participant burden while improving critical trial data, which could ultimately speed much-needed therapies to market.
Encouraging a More Efficient Regulatory Environment
Many factors enabled the biopharma industry to develop, manufacture and distribute vaccines quickly during the pandemic. In addition to massive government funding and unprecedented collaboration between biopharma companies, the timeline was also sped up by the FDA’s efforts to streamline the COVID regulatory process.
In normal times, it takes an average of 10-12 years to develop a new vaccine. Remarkably, two COVID-19 vaccines were developed and granted emergency use authorization (EUA) by the FDA just 11 months after the SARS-CoV-2 sequence was published. The agency’s new efficiencies played a big role in accelerating the regulatory process.
One of these advancements facilitated by the FDA was the use of master protocols. A master protocol is a trial protocol designed with multiple sub-studies. It involves coordinated efforts to evaluate one or more investigational drugs, in one or more disease subtypes, with one or more objectives — all within the same overarching trial structure.
Master protocols that are well designed and executed can accelerate drug development by maximizing the amount of information obtained from the research effort and reducing administrative costs and time.
One of the best examples of master protocols in action during the pandemic is the U.K.’s RECOVERY trial. With over 43,000 participants and counting, and 177 active sites throughout the U.K., the study has produced data on four therapies that have informed global authorization and approval decisions.
A second successful example of master protocols being used across multiple trials was the National Institutes of Health’s public-private initiative, Accelerating COVID-19 Therapeutics Interventions and Vaccines (ACTIV). An ACTIV working group coordinated and contributed to the development of harmonized protocols for vaccine evaluation in Phase 3 clinical trials, including trials for Johnson & Johnson’s FDA-authorized single-shot COVID-19 vaccine as well as Moderna’s FDA-authorized mRNA vaccine.
Another FDA effort propelled by the pandemic’s urgency was the use of Real-World Evidence. During the pandemic, the FDA collaborated with public and private partners to collect and analyze various real-world data sources. The information was used to evaluate potential therapies, vaccines, and diagnostics for COVID-19. In recent years, the agency has taken steps to leverage modern, rigorous analyses of real-world data — such as data from electronic health records, insurance claims, and lab results.
The FDA plans to capitalize on these lessons learned during the pandemic and create a better, more efficient agency.
“To the extent that the innovations and adaptations we implemented during the pandemic crisis worked and would be appropriate to implement outside of a pandemic situation, we will incorporate them into standard FDA procedures,” said then-FDA Commissioner Stephen Hahn in a June 2020 speech. “And to the extent that we identified unnecessary barriers, we will remove them….Permanent change where needed will take place, and will make FDA an even stronger agency.”
Opening the mRNA Floodgates
On Sunday, Dec. 13, 2020, many watched in awe as trucks carrying the country’s first authorized COVID-19 vaccines slowly pulled out of Pfizer’s Portage, Mich. manufacturing plant. Although the therapeutic potential of mRNA has been explored for decades, the pandemic marked the first time in history that any mRNA vaccine was approved for human use.
Now, with two of the most protective and widely used vaccines in the world relying on mRNA technology — and one of them securing the first full FDA approval — the mRNA sector is exploding with potential.
Billions of dollars in capital have flowed into mRNA-focused companies, and the thriving field is attracting top scientific talent. Industry titans like Gilead Sciences and Sanofi are inking multimillion-dollar deals with smaller biotechs, betting on next-generation mRNA vaccine technology beyond the COVID-19 pandemic.
MRNA vaccines might immunize against Zika, HIV, or shingles in the future, and the technology has potential use in treatments for cystic fibrosis, cancer, and other hard-to-treat diseases.
Even more broadly, the FDA authorization and approval of mRNA-based vaccines has demonstrated that the agency is open to previously unproven technology, which could encourage drug developers to press forward with the development of more complex, advanced therapies that could prove to be life-changing for patients.
The COVID-19 pandemic continues to disrupt all aspects of life. But turbulence often kickstarts lasting change — and in this case, this is good news for both the biopharma industry and patients worldwide.
The BioTechLogic team is particularly excited about applying our cell and gene therapy CMC expertise to help bring future mRNA-based therapies, and treatments based on other gene therapy platforms, to patients in need. Don’t hesitate to contact us if we can help.