The promise of gene therapy treatments is upon us. In more than 20 years of experience in the clinical research space, I heard a lot of excitement over the years about genetically engineered treatments. In the last few years, that excitement has begun translating into reality. We see an accelerating gene therapy market, expected to grow globally by 16.6 percent between 2020-2027. Early phase research may be conducted in the halls of large academic medical centers who are equipped and experienced in working with genetically engineered treatments and biosafety requirements. However, all those new therapies must eventually go through larger scale clinical trials on their way to market approval. As gene therapies enter the pipeline at an exponentially-growing pace, how can private and hospital-based clinical trial sites prepare themselves to take advantage of the coming rush?
In this blog, we will explore how proactive clinical research sites, from small private clinics to large integrated health systems, are preparing for the accelerated trend of gene therapy research.
Understanding the Regulations
When treatments involve recombinant DNA, synthetic DNA, or messenger RNA, additional oversight is necessary. These treatments make permanent changes to the humans they are introduced into. Unlike typical drug treatments which are metabolized by the body; gene therapy treatments, in many cases, are making permanent changes to the human’s genetic profile and cannot be undone. Additional precautions and oversight by an institutional biosafety committee (IBC), are necessary in the handling and administration of these novel research compounds. The US National Institutes of Health, Office of Science Policy sets the guidelines for research involving recombinant or synthetic nucleic acid molecules. If you want to learn more about the additional rules and when IBC review is required, see our extensive IBC resources, blogs and webinars at Advarra.com.
Most clinical trial sites likely already have the basic facilities needed to conduct gene therapy research. Human gene therapy treatments typically fall into biosafety level 2 requirements, which are consistent with good laboratory and clinical practice precautions (e.g. sharps protocol, blood born pathogen protection, PPE, etc). For gene therapy treatments, some additional precautions need to be taken to protect the site staff and the surrounding community from inadvertent exposure to a compound which could permanently change their cells and genes. Some common items sites need to address:
- Eye wash stations near where the gene therapy product will be administered
- Non-porous surfaces (e.g. chairs, floors, etc) in all areas where the gene therapy product will be administered
- No carpet or cloth chairs that cannot be sanitized
- Designated rooms where the product will be administered
- A fume hood if the product is prepared or mixed on-site
- Hazardous waste disposal facilities and service
Preparing your site in advance, before a gene therapy clinical trial is at your doorstep, can save days or weeks off your study activation. The IBC will inspect your facility to determine if it is adequate. If you need help in making necessary preparations, the biosafety consulting team at Advarra can support your facility planning.
Standard Operating Procedures
In accordance with the NIH OSP Guidelines, the IBC is required to review the site’s standard operating procedures relating to safety precautions. The IBC is looking to ensure adequate plans are in place to keep the site staff and the surrounding community safe from inadvertent exposure to an investigational product which could make permanent changes to human cells and genes.
At a minimum, sites need to prepare the following procedures in advance of IBC review:
- OSHA blood borne pathogen exposure control plan
- Biohazardous waste/regulated medical waste disposal policy, procedures, or permits
- Standard operating procedures (SOPs) for use and handling of the particular study agent, as well as spill and containment procedures
Clinical trial sites should carefully prepare these SOPs and combine with staff training for anyone who will be handling the gene therapy based investigational product.
Getting Registered With a Central IBC
Unless you are at a large academic medical center, likely your research site does not have its own institutional biosafety committee, so sites will need to register with an independent IBC. Sponsors conducting gene therapy trials pick their central IBC much in the same way they pick a central IRB; so you will want to partner with an experienced independent IBC who has plenty of experience conducting integrated IRB/IBC reviews. The registration process is reasonably straightforward and involves the independent IBC submitting paperwork to the National Institutes of Health, Office of Science Policy to register your site. Plan ahead since the OSP registration process can take upwards of eight weeks. If you plan to register with multiple independent IBCs, the OSP process must be repeated for each additional independent IBC provider you decide to work with.
Once your facility is ready, SOPs are written and implemented, and you have registered with an independent IBC, you are ready to start taking in gene therapy trials!
Get Noticed by Sponsors
Research sites actively seeking gene therapy trials, and more recently mRNA based COVID trials, can improve their overall portfolio of research and position their sites for an increased number of clinical trials. Here are some common things you can do to get yourself noticed:
- Add cell and gene therapy capacity language on your research site’s website to let sponsors know you are ready and able to conduct gene therapy research
- Proactively reach out to the cell and gene therapy centers of excellence at sponsors and CROs
- Include documentation in your response to site feasibility questionnaires to let sponsors know you are registered with an IBC and ready to take on their next trial
- Partner with an independent IBC who will actively promote your site to sponsors and CROs who are looking to place their gene therapy research at sites how are pre-registered and ready to get started quickly
Innovative treatments using gene therapy are growing and public perception of genetically engineered therapeutics is changing thanks to the mRNA COVID-19 vaccines now among us. Make sure your site and your participants can take advantage of these new, cutting-edge investigational products. Get ready for the rush!
At Advarra we know Gene Therapy Research. We offer the industry’s only truly integrated IBC and IRB service with optimized collaboration; and, with Advarra’s worldwide presence we have reviewed more protocols than any other integrated IRB and IBC. Our exclusive network of Gene Therapy Ready sites is standing by to rapidly start-up your gene therapy research. All backed by our amazing biosafety consulting team ready to help you from site selection through to market approval. Contact us today to learn why when you think Gene Therapy think Advarra!
Sites — Is your site part of the Advarra’s Gene Therapy Ready network? Get signed up today.
Sponsors/CROs — Looking to place your next Gene Therapy treatment or vaccine trial? Contact us to learn more about Advarra’s Gene Therapy Ready network and how we can support your next trial with integrated central IRB and IBC review.