Due Diligence for a New Drug Indication

Jenna Brager, PhD, RN, MS

Senior Executive Director of Regulatory Affairs for MyMD Pharmaceuticals, Inc.

As a biotech company progresses through the various stages of drug development and commercialization, understanding how its potential study drug brings value to the industry, stakeholders, and the overall strategic development plan needs to remain in the forefront.¹ Considering a new indication for a drug program is not only exciting but also a significant undertaking. During the preliminary stages of consideration, a broad, high-level screening of opportunities should be performed to determine what has already been done in the specific therapeutic area as well as how the drug will be differentiated from treatments currently available.² Here, the goal is to quickly eliminate or move away from opportunities that are not truly aligned with the drug development plan. 

The following steps provide a guide to preparing for a new drug indication:

1. Investigate clinical and pre-clinical trials

   Before designing a new protocol in support of human clinical trials, one must understand the landscape in which the drug was previously tested clinically and pre-clinically (if possible). 

   For example:

   1. How many clinical trials have been completed in the specific therapeutic area?
   2. How many clinical trials are actively recruiting and where?
   3. What will the company’s drug do differently than the current and/or previous trials?

   Before moving forward with a new indication, the company must have a clear path in mind for the drug. It is important to understand the strengths and limitations of previously conducted trials and how current trial design has accounted for these lessons as necessary.   

2. Dive into the literature

   In addition to the information obtained above, it is important to focus on the published literature surrounding the therapeutic indication. 

   For example:

   1. Who are the competitors (nationally and internationally)?
   2. How rich is the literature in the therapeutic indication of interest?
   3. What is the gap in the literature and how does the drug address it?
   4. What is innovative about the drug?
   5. Were the primary and/or secondary outcomes met?

   Exploring the literature will help the company understand how other drugs geared towards the indicated have performed in clinical settings and if various outcomes were achieved. 

3. Confer with the experts

   Communicating with experts in the field not only increases overall buy-in and confidence in the drug development plan, but also leads to potential partnerships (site identification, principal investigator identification, etc.) that is necessary to implement the clinical trial.

   For example:

   1. Are the experts in the field interested in learning more about the drug?
   2. Are they willing to test it on his/her patients?
   3. Is the company’s drug challenging the current ‘gold standard’ treatment?

   If the experts are excited about the potential of the new drug indication, the company is certainly on the right track. Partnering with experts in the field is essential to pushing the drug program forward. This strengthens the application to the Agency as it reiterates that the experts believe that the drug shows promise and is worth testing in a clinical trial setting.  

4. Recognize the FDA’s perspective

   Understanding the path previous drugs followed to come to market while also staying up to date with recent press releases surrounding other drugs in the area of interest will help guide one’s drug program.

   For example:

   1. What drugs were recently approved for this therapeutic indication?
      1. How similar or dissimilar is the company’s drug?
   2. What companies are currently manufacturing these drugs?
   3. Have there been recent recalls or issues announced by the FDA surrounding the specific indication?

   Recognizing where the FDA currently stands should be a primary factor in the company’s plan to push a drug forward.  This will help ensure that the company is most appropriately aligned with the Agency.

Summary

Gaining a thorough understanding of the landscape prior to initiating a new drug development program is essential to a company’s success. Growing a strong knowledge base of the therapeutic indication requires evaluation of the cost of manufacturing (e.g. tablet, capsule, infusion), previous clinical trials (efficacy) and preclinical trials (toxicities), and understanding the most recent published literature. The company should communicate with current experts in the field, and review the regulatory pathways and the FDA’s perspectives such as pediatric indications, Orphan Drug Designation, right to try legislation, and compassionate use criteria.  Long-term considerations will include the need for Phase 4 post marketing clinical trials and specific FDA regulated safety monitoring programs. These are the key steps to performing due diligence for a new drug indication.

References

1. https://www.iqviabiotech.com/
2. https://www.biopharma-excellence.com/2018-2-13-a-guide-to-scientific-due-diligence-how-to-set-up-an-efficient-process/

Dr. Jenna Brager, PhD, RN, MS, is the senior executive director of regulatory affairs at MyMD Pharmaceuticals. Prior to joining MyMD, Dr. Brager served as the Director of Clinical Research for LifeBridge Health and was a clinical investigator at Johns Hopkins Hospital. Dr. Brager received her B.S. in Nursing from the University of North Florida, graduating cum laude and received her Ph.D. from the Johns Hopkins University School of Nursing.

Dr. Brager welcomes comments, questions, and requests for future blog topics. Please email jbrager@mymd.com.