The Benefits of a Regulatory Science Approach for the Development of Drugs

A recent question came to Processa CEO Dr. David Young ...

You’ve often spoken of a “Regulatory Science Approach” for the development of drugs. What is the benefit of doing this, and can you give an example of how this translates to one of your pipeline candidates?

His response illustrates how this approach will benefit drug development, along with examples of this approach helping in the past.

FDA’s NDA (New Drug Approval) process requires a sponsor to demonstrate that the clinical benefit of a drug significantly outweighs the risk of approving the drug or of not approving the drug. Given the complexity of all the factors that can affect both a patient and the ability of a drug to successfully treat a medical condition, there are an endless number of clinical designs and development programs that can investigate the efficacy and safety of a drug.

The PCSA Regulatory Science Approach evaluates all the potential designs and paths to determine the development program(s) with the highest probability of demonstrating an FDA approvable benefit-risk profile for a drug. In addition, the PCSA approach evaluates how efficient the development programs are in terms of time and cost.

An example of the use of our Regulatory Science Approach is with Next Generation Capecitabine (the combination of PCS6422 and capecitabine, capecitabine - an approved anti-cancer drug with a market > $1B). As we obtained data, we continue to evaluate which clinical trials and development programs provide us with the highest probability of successfully demonstrating an FDA approvable benefit-risk profile.

Since the increased potency and alteration in capecitabine metabolism did not last as long as desired in the first 2 cohorts of our Phase 1B trial, we modified the design of the trial and have moved forward with an amended protocol without altering the projected timeline for the initiation of our next efficacy/safety trial. In addition, the protocol modifications should increase the probability of approval by identifying a dose optimization process that could determine the optimal next Generation Capecitabine dosing regimen for each individual patient, similar to what the FDA has proposed in their Oncology Project Optimus Program.

Additionally, I think that you could also look at how our Regulatory Science Approach affected our last FDA approval of Acthar at Questcor Pharmaceuticals. Acthar was originally approved in 1952 and had over 50 approved indications on-label in 2007 but it was not approved for the indication that it was used the most, infantile spasms. The FDA rejected the sNDA application for Infantile Spasms. Our team used our Regulatory Science Approach to obtain a new label with 19 indications (maintaining 18 indications on the label) including the new approval of infantile spasms by providing the clinical evidence supporting that the benefits of the drug outweighed the risk of administering the drug or not approving this drug in patients with each of the 19 medical conditions.