2021 Predictions for Regenerative Medicine as the Pandemic Continues
Industry Insight Jan 29, 2021 | By Mark W. Sawicki, PhD, Cryoport Systems
Last year, we saw unexpected and unprecedented changes worldwide. Despite the clinical trial disruptions early in the year due to COVID-19, companies in the regenerative medicine space still raised a record $15.9 billion through the third quarter of 2020.1 In 2021, we can expect the pandemic’s lingering impacts to continue to reverberate throughout regenerative medicine. With that in mind, here is a snapshot of initial expectations for the year.
Clinical trial impact for cell and gene therapies
Due to COVID-19, many clinical trials will continue to face challenges with patient enrollment. While the overall number of programs will likely increase throughout 2021, the number of patients who are dosed may be impacted to a certain degree. This will likely cause cycle times for clinical progression to lengthen from historical norms.
In addition, due to the significant demand on the contract manufacturing space in support of COVID-19 vaccine production, we should anticipate delays in new clinical starts, particularly for allogeneic programs that utilize batch manufacturing, which may compete for capacity with vaccine production. The good news is that, despite the above, we should expect to see up to 21 biologics license application and marketing authorization application filings in the cell and gene therapy space in 2021, albeit some of them may push out to 2022 due to the US Food and Drug Administration’s (FDA's) focus on getting COVID-19 vaccines approved for the global population.
Bristol Myers Squibb recently warned about this in its Q3 earnings release, where the company described, “the increased possibility of the COVID-19 pandemic delaying the timing of the FDA’s approval decisions” for the company’s CAR T-cell therapy candidate Liso-cel/JCAR017/Breyanzi.2
COVID-19’s impact on vaccine development and cell and gene therapy distribution
COVID-19 vaccine and treatment distribution has taken up significant capacity from major cold chain logistics companies, cold storage providers, and packaging and freezer manufacturers. There has also been a strong increase in manufacturing dedicated to the vaccines at companies such as Lonza, WuXi and Fuji/Diosynth. The pivot to vaccines impacts personnel and available capacity for cell and gene therapy production. Additionally, it will likely have an impact on new clinical starts.
The lack of air cargo capacity due to the reduction in commercial flights has also been well documented, causing increased costs, evaluation and implementation of alternative transport methods related to clinical distribution globally. Rates have increased by as much as 107 percent for some routes over 2019 and are likely to remain elevated through early 2022,3,4 as shown in the Baltic Exchange Airfreight Index below.
Commercial activity within regenerative medicine
Fortunately for the industry, a robust pipeline of late-stage programs continues to push through to the finish line with five or more new commercial products potentially receiving approval in 2021, including multiple products for bluebird bio, Orchard Therapeutics, Bristol Myers Squibb and Mesoblast, as well as approvals for a host of others including, but not limited to, Atara Biotherapeutics, Iovance, SanBio, Poseida and Audentes Therapeutics.5
Moreover, due to the industry’s ample financial resources as a result of prolific financing activities, the space has the reserves to fund most of these programs’ commercial activity, even if delayed slightly due to the FDA’s COVID-19 preoccupation. We anticipate 2021 being a record year for commercial launches, which will further cement regenerative medicine as the third pillar in the pharmaceutical market in conjunction with small molecules and biologics for the foreseeable future.
Both the FDA and European Medicines Agency have been adapting to the COVID-19 reality and transitioned to supporting commercial filing activities virtually. In a statement released in May 2020, the FDA said that “CDER and CBER are leveraging technology to host virtual meetings, rather than in-person meetings, with the industry during the COVID-19 public health emergency. At this time, all meetings will be held virtually.”6
Regulatory considerations within regenerative medicine
In June 2020, the ISO/TC 276 Biotechnology Technical Committee defined a new standard for transporting cells for therapeutic use. The standard, ISO 21973, focuses on the complete traceability of all equipment, processes and logistics handling used to manage the therapy’s environmental control while in transit, which can be referred to as the “Chain of Compliance®”.
This traceability, control and oversight of the entire transportation process is key to viable scalability.7 While some existing standards (e.g., USP 1044) reference shipping advanced therapy products,8 ISO 21973 is the first standard to specifically address, in detail, the requirements for the cell and gene therapy supply chain, with a focus on transportation, recognizing that these products are significantly more fragile and valuable than most small molecule and biological medicines.
Damage to products during shipping is not always obvious, and damaged cell and gene products, in particular, do not change in smell, color or other physical ways that are immediately apparent. Slight temperature deviations can render the product ineffective, and minor physical damage can also make the product unusable.
Today, the growing prevalence of chronic medical ailments and genetic disorders across the world are major factors driving the regenerative medicine industry’s growth.9 As the world continues to battle the COVID-19 pandemic at the start of 2021, never has investing in regenerative medicine been so important.
1. Record Financing Drives Sector Growth. Alliance for Regenerative Medicine. https://alliancerm.org/sector-report/q3-2020-trend-talk/. Accessed January 29, 2021.
2. US FDA approval tracker: November. Evaluate.com. https://www.evaluate.com/vantage/articles/news/snippets/us-fda-approval-tracker-november-0. Published December 2, 2020. Accessed January 29, 2021.
3. Airfreight rates - Baltic exchange airfreight index. Air Cargo News. https://www.aircargonews.net/data-hub/airfreight-rates-tac-index/. Accessed January 29, 2021.
4. Airfreight rates expected to remain elevated in 2021. Air Cargo News. https://www.aircargonews.net/business/airfreight-rates-expected-to-remain-elevated-in-2021/. Published January 8, 2021. Accessed January 29, 2021.
5. Anticipated clinical data & events. Alliance for Regenerative Medicine. https://alliancerm.org/anticipated-clinical-data-events/. Accessed January 29, 2021.
6. Effects of the COVID-19 public health emergency on formal meetings and user fee applications - questions and answers. US Food and Drug Administration. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/effects-covid-19-public-health-emergency-formal-meetings-and-user-fee-applications-questions-and. Published May 2020. Accessed January 29, 2021.
7. Biotechnology - General requirements for transportation of cells for therapeutic use (ISO Standard No. 21973:2020). International Organization for Standardization. https://www.iso.org/standard/72326.html. Accessed January 29, 2021.
8. 〈1044〉 Cryopreservation of cells. USP. https://www.usp.org/sites/default/files/usp/document/our-work/biologics/resources/gc-1044-cryopreservation-of-cells.pdf Accessed January 29, 2021.
9. Regenerative medicine: Market trends and legal developments on the horizon for 2021. MedTech Intelligence. https://www.medtechintelligence.com/column/regenerative-medicine-market-trends-and-legal-developments-on-the-horizon-for-2021/Published December 11, 2020. Accessed January 29, 2021.
Written by: Mark W. Sawicki, Ph.D., President & CEO, Cryoport Systems
Mark Sawicki brings 15 years of business development and sales management experience, having consistently delivered on corporate revenue and market share goals in the pharmaceutical and biotechnology industries. Sawicki was most recently the chief business officer at AAIPharma Services Corporation/Cambridge Major Laboratories Inc. Additionally, he has served in senior business development roles at CMC Biologics and Albany Molecular Research Inc. (AMRI), where he increased revenue at rates far outpacing industry standards. Sawicki holds a bachelor’s in biochemistry from the State University of New York at Buffalo and a Ph.D. in biochemistry from the State University of New York at Buffalo, School of Medicine and Biomedical Sciences. He also received graduate training at the Hauptman Woodard Medical Research Institute. Sawicki has authored a dozen scientific publications in drug discovery with a focus on oncology and immunology.