Working for a large biotech company I was assigned to a product development team that was given the challenge of getting a first-in-man drug into a Phase-1 trial using the minimalist amount of resources and shortest amount of time.  The project was named after the 1980s TV series hero MacGyver who applied his scientific knowledge to ordinary items (e.g. Swiss Army knife) to create for him and others a means of escape from impending doom and in the process save the world. 

The team success would be measured against already established budget, resource and timeline metrics for other first-in-man Investigative New Drug applications (IND). As the regulatory representative on the team, my challenge was to ensure that there was just enough regulatory compliance with current regulations and guidance’s that the IND would be accepted and the clinical trial not put on hold. No regulatory “nice-to-haves” would be acceptable.

Examples of nice to haves:

• Using the recommend upper number of test animals in the preclinical studies
• Using the recommend upper number of patients in each dose cohort in the Phase-1 trial
• Full laboratory panels plus additional labs (e.g. possible biomarkers)
• Phase-1 trial design with a placebo or active control arm

The MacGyver project team beat all established metrics. The budget was 40% less than the established metric and the time from starting the IND enabling preclinical studies to filing the IND was less than five months (the metric was 12 months).  At the end of the FDA 30-day review clock for the IND there were no reviewer questions and the Agency gave the company the green light to start the Phase-1 trial.

On the surface the project looked successful and could provide a possible new model for moving drugs into the clinic.  However, this was a case were the project was a success but the model was a failure. In the “Lessons Learned” document, that is written by the project team and filed with upper management, it was discussed that the budget savings were only temporary.  Additional money needed to be spent to cover the commitments made to the FDA to provide additional nonclinical and analytical studies. Also, the project had to be put on hiatus on completion of the Phase-1 to allow catch-up time for the assay and drug manufacturing development that usually would have been done prior to going into the clinic. 

Bottom-line, even with solid clinical safety and proof of concept data from the Phase-1 trial the project couldn’t move forwarded because of the “short-cuts” taken.  To move into the Phase-2 more than twice the budget had to be allotted and seven months added to the timeline. Also resources from other drug product development projects had to be pulled-in to keep the project going.

The MacGyver project really demonstrated to the project team and upper management that, while the minimalist model could get an IND deliverable that is acceptable to the FDA, the tradeoff would be in time and budget to take the next step.

I’m interested in hearing about your experience with other “MacGyver type” models and their success.

Taylor Burtis

Tags: Product Development, Project Management

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