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In the News: June 2021 Regulatory and Development Updates

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program.

Decades-Old Antidepressants Being Repurposed to Treat Cancer 

At Camargo, we have worked with hundreds of investigators who have found new targets for established drugs.  It is exciting to find new uses of drugs with known safety because these drugs can reach new groups of patients in a relatively short timeframe. Such may be the case for a 1950s class of drugs that may have potential in the treatment of cancer. 

Monoamine oxidase inhibitors (MAOIs) such as prochlorperazine, phenelzine, clorgyline and mocolobemide were once popular antidepressants.  Although still available as generics, for the most part they have been replaced by newer drugs with fewer side effects. But sometimes, a side effect can be exploited for the treatment of another disease. 

Researchers at the Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have observed that MAOIs might help the immune system attack cancer.  In the announcement of their findings, the researchers noted the advantage of repurposed drugs: “What’s especially exciting is that this is a very well-studied and safe class of drug, so repurposing it for cancer isn’t as challenging as developing a completely new drug would be.” 

The UCLA researchers measured monoamine oxidase A (MAOA) gene expression in tumors from cancer patients and found that high MAOA expression was associated with shorter survival times and turned to a mouse model to investigate these observations with promising results. Researchers at the University of Southern California in conjunction with the National Cancer Institute are currently conducting a Phase II clinical trial of the MAOI phenelzine in prostate cancer patients and have announced in an interim study publication that it has thus far demonstrated efficacy. 

Report Published on the FDA’s Use of Patient Experience Data in Regulatory Decision-Making 

As required by components of the 21st Century Cures Act and the FDA Reauthorization Act, the FDA has sought to promote Patient-Focused Drug Development considerations into regulatory decision making, including product application approvals. The requirements include periodic assessments of the FDA’s use of patient experience data, the first being due this year, 2021. The FDA itself did not produce the assessment but instead contracted the Eastern Research Group to conduct the assessment and produce a report. 

The assessment sought to answer several questions: 

  • How does the FDA use patient experience data in regulatory decision-making? 
  • How do FDA staff, applicants, and other stakeholders describe the FDA’s use of patient experience data in regulatory decision-making? 
  • What good practices and opportunities for improvement exist for the use of patient experience data in regulatory decision-making? 

So how did the FDA do? Overall, the report indicates that the Agency has made a good initial effort but that there is a need to further clarify and expand the role of patient experience data in regulatory decision-making. 

Rather unsurprisingly, one point made in the report was that while the FDA often focuses on clinical endpoints, patients are often more concerned with quality of life-related issues. The report is a useful read to keep current on a topic of interest at the FDA and in drug development as a whole. Camargo is active in the advancement of patient experience data in drug development—see our recent white paper on the topic.

FDA Publishes Draft Guidance on Adverse Drug Experience Reporting for INDs and BA/BE Studies 

In June, the FDA issued an updated (draft) guidance titled “Sponsor Responsibilities—Safety Reporting Requirements and Safety Assessment for IND and Bioavailability/Bioequivalence Studies.” Notably, the guidance states early on that the content from the 2012 final guidance remains largely unchanged and that the new guidance, once finalized, will supersede the 2012 final guidance. However, until that time, the 2012 final guidance continues to represent FDA’s current thinking about on the subject. (That is, the still current thinking is nine years old.) The draft actually combines the 2012 final guidance and a 2015 draft guidance: “Safety Assessment for IND Safety Reporting.” 

Most of the revisions are to the contents of the 2015 draft guidance, specifically including: 

  1. Planned unblinding of safety data and the implications for trial integrity, 
  1. Increased flexibility regarding the party reviewing aggregate safety information for IND safety reporting purposes, 
  1. Clarification regarding the scope and methodology of aggregate analyses, and 
  1. Clarification regarding the plan for safety surveillance, including what elements should be included in the plan. 

The guidance also addresses the reporting of serious adverse events (SAEs) during clinical investigations under an IND and during BA/BE studies not conducted under an IND. The required reporting process is often a source of confusion for IND sponsors new to drug development. For example, discussions of five definitions (“adverse event,” “adverse reaction/suspected adverse reaction,” “unexpected,” “serious,” and “life-threatening”) take up roughly a page each. 

A review of the consolidated draft guidance in light of the “current” guidance is time well invested. Camargo can help with interpreting the guidances or with the actual reporting process. 

Co-Authors:

Ken Phelps
President and Founder

Bill Stoltman, JD
Vice President, Regulatory Operations

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