An Introduction to FDA Clinical Investigator Training Course

The U.S. Food and Drug Administration (FDA) held a Clinical Investigator Training Course or the CITC last month (12/7-8/2021). This  course has been conducted for over ten years. It usually takes place in person, but the COVID-19 pandemic has first pushed the FDA to postpone it and later to provide it virtually due to on-going concerns with public health. (1) The course was delivered online in a novel way, which shows how FDA is adapting to the new situations that present challenges and obstacles for investigators and regulatory bodies.

The 2021 course was mainly focused on topics like COVID-19 (medical products for prevention and treatment, Emergency Use Authorizations (EUA), investigator responsibilities during the pandemic), gene therapy, new trial approaches, drug repurposing, and diverse representation in clinical trials. (1) 

Who may benefit from the course?

The course is primarily recommended for healthcare professionals, who have basic knowledge of clinical studies, regulatory requirements for clinical trials, and the development of medical products, in particular, physicians, nurses, pharmacists, clinical scientists, consultants, employees working in the drug or biologic or device industries, regulatory affairs professionals, and biomedical professionals. (1) The course featured a practical demo of how to apply to the FDA’s new portal and other focused information. If you want to get more information on pharmacy like medical supplies products, you could access to Pharmasources, a vertical e-commerce online platform serving the pharmaceutical industry.

Course overview

The course took place in an abbreviated format. It ran on December 7 and December 8, 2021. This two-day course given by senior FDA experts provided FDA's perspectives.  On the first day, it focused on scientific updates, which involved an introduction to Car T-Cell therapy, gene therapy, oncology clinical trials, COVID-19 treatment, and diversity in trial populations. The second day, focused on operational updates and acceptable scientific and analytic standards in clinical study design and conduct on Decentralized Clinical Trials (DCTs), Digital Health Technologies (DHTs), and investigator responsibilities during COVID-19.   

In this intensive course, participators learned basic clinical trials, the responsibilities of an investigator conducting a clinical trial and the basic concepts of clinical trial design as well as innovative approaches to the conduct of clinical trials related to gene therapy, Car T-cell therapy, the development of medical products for the prevention and treatment of Covid-19.

Highlights of the course

In regards to human gene therapy, the course establishes that these are products that mediate their effects through transcription of transferred genetic material or alteration of genetic sequences, and include products like plasmids, viral and bacterial vectors, ex vivo genetically modified cells, and genome editing products. All of these require substantial evidence of effectiveness and safety to get approved, and this evidence should come from adequate and controlled studies. In addition, FDA offers flexibility in the case of product development for rare diseases, but safety still remains paramount. Because of the variability in product development, the process can involve consulting with FDA early on, (3) such as Pre-IND Consultation Program and INTERACT Meetings with Sponsors for Drugs and Biological Products. (4,5)

Car T-Cell therapies are currently dominating the scene of anti-cancer product development. To date, there are several products that have been approved for clinical use by FDA but toxicity is a significant issue in regards to the therapies. Therefore, current goals involve improving the design to reduce toxicity through suicide genes, on/off switches, antagonists of systemic cytokines, and enhancing efficacy through developing tumor homing and tissue penetration and other aspects. However, safety is still a paramount concern, and some products may require up to 15 years of monitoring after development. (6) 

Drug repurposing is an important topic, as it offers the possibility of treatments for unmet needs, such as rare disorders. It is also worth identifying ineffective and dangerous new uses for existing drugs. Therefore, repurposing requires adequate studies and deserves more clinical attention. (7)

DHTs are becoming more important, in particular, due to the shifts in health associated with the pandemic, and allow several benefits. It is possible to get objective measurements throughout the day while the patients remain in the comfort of their homes, so the data can be collected continuously. At the same time, it puts a higher onus on the investigators to ensure accuracy, precision, and reproducibility, as well as ensure data security and privacy. (8) 

At the same time, an emerging theme in the course was the important role of clinical investigators who must follow regulations and take responsibility for the ethical, medical, and security concerns in each study they carry out. FDA offers many resources for clinical investigators and is open to involve solutions and locate answers. (9)

Significance and impact 

The FDA clinical investigator training course has been a significant feature of the previous year and serves an important purpose, so its revival, even as a virtual event, shows progress. The course offers a quick and concise update on the central issues that concern research.

In a world that continues to fight the pandemic, this is a central topic, as investigators might be able to offer new and better solutions. This is still shown in the selection of COVID-19 as one of the main themes of the course.

There has been significant progress in regards to novel therapeutic  approaches, like gene and cell therapy that become essential and promise a lot of future applications. The focus remains also on cancer and the ethical and regulatory concerns that matter to investigators. FDA serves as both a guide in regards to safety and similar concerns and also helps train professionals in how to make use of all the resources they offer and when FDA can support drug development. In addition to more general information, the course serves as a brief introduction to the mechanisms and tools FDA offers, how these can be used, and how can the organization be contacted. 

Furthermore， the course serves a useful purpose for advancing drug development in a safe way and ensuring that investigators and industry employees in different positions are on the same page. It also reinforces the commitment to safe and ethical development and provides new alternatives in the form of DHTs, for instance, that should be taken more advantage of for better results and better logistics.

In addition，FDA recommends the course  to any professional working in the areas of drug development and clinical trials or healthcare. The registration for the future course or the recording and materials of the course are available at the FDA website. (10)

References

1. NIH. (2021) https://seed.nih.gov/events/20211207/fda-clinical-investigator-training-course-update

2. FDA. (2021) CITIC Agenda https://sbiaevents.com/files2/CITC-2021-Agenda.pdf

3. Xu, L. (2021). Clinical Development of Gene Therapy Products, Clinical Investigator Training Course (CITC)

4. FDA (2020). https://www.fda.gov/drugs/investigational-new-drug-ind-application/pre-ind-consultation-program

5. FDA (2020). https://www.fda.gov/media/124044/download

6. Xu, L. (2021). Clinical Development of Chimeric Antigen Receptor (CAR)-T Cell Therapy in Cancer. Clinical Investigator Training Course (CITC).

7. Stone, H. (2021). Drug Repurposing and Development of New Indications. Clinical Investigator Training Course (CITC).

8. Sacks, L. (2021). Digital health technologies and Decentralized clinical trials. Clinical Investigator Training Course (CITC).

9. Kleppinger, C. (2021). Investigator Responsibilities–Getting It Right During a Pandemic. Clinical Investigator Training Course (CITC).

10. https://www.fda.gov/drugs/news-events-human-drugs/clinical-investigator-training-course-citc-update-12072021-12082021

About the Author:

Lin Zhang, M.D., senior director of a health care industry company in the United States. With the experience in clinical medicine, biotechnology, health industry and other fields, he is responsible for the research and development of plant medicine, functional food and health products. He was a clinician and worked for the National Cancer Institute, FDA and the National Cancer Center of Japan for many years.