Don’t let this happen to your company
This is the stock chart for PTC Therapeutics over 5 days. The market cap of company went from approximately $1.2 Billion to about $400 Million — in one day!
This was due to failure to get approval for a FDA drug trial submission. Technically, the FDA refused to even read the application. There was sequence of events that led to this outcome, and most of it could have been prevented.
I have no personal knowledge of this drug trial. But the sequence of events that leads to such outcomes have several commonalities. Here are the steps you can take to avoid them:
Define the clinical end points upfront and work towards them.
Keep them clear and keep them narrow. Define the indication that you want to drug approval for, and aim to meet the clinical trial end points for that indication. At iClinical, we define the analytics for the clinical trial even before the trial starts. This helps pharma companies define the scope of the trial and sticking to it. The pharma companies can now monitor their drug trials from day 1. It will specify the exact metrics for the trial outcomes, if the project physician notices any anomalies — they can either fix it or if it is beyond fixing, they can stop the drug trial.
Outcome: For nonworking trials — cut you losses and stop the trial. For working drugs — speed up the trial and get the drug the market sooner.
Reduce data collection and analysis risks — make all your trial processes real-time
Collect the trial data once and at the source, getting it into the database quickly — a eSource solution would solve both this issues. Once in the database, run real-time analytics and find out how the trial is going. Build in additional alerts and alarms that might reveal cracks in the trial. Communicate the results with the entire team in real-time. Keep the trial physicians informed of the trial progress — keeping them informed will boost morale and increase trial enrollment. Transparency and real time communication will go a long way in speeding up the drug trial.
Deploy periodic checkpoints to check the outcomes
These checkpoints inform you of progress in the drug trial.
The big picture items include: Are we meeting the the clinical trial outcomes? Is the drug working? How are the end points and adverse events tracking, are there any corrective measures required?
On a micro level the analytics would be: Is the trial enrollment on track? Is the data clean? Are the sites following the protocol/SOPs? Are there any protocol deviations — is so how do we fix them?.
The series of timely decisions made on the day to day micro level can add up to a quicker and successful drug submission.
Don’t rush your submission
There are series of bad decisions that lead to bad FDA study submissions. These could either be meeting the stock analysts expectations, making bad data collection and analysis choices, submitting studies that do not clearly demonstrate safety and efficacy end points.
“Give me six hours to chop down a tree and I will spend the first four sharpening the axe.” — Abraham Lincoln
Take the time to study each of the aspects. Get independent outside reviewers to make sure the study design, analysis and the science is bullet proof. If you or your team is not convinced of the trial outcomes/interim analysis, cut your losses and stop the trial immediately. Patience in the right areas (design, analysis and sound science), usually results in successful FDA submissions.
Learn from your mistake (better yet from other people’s mistakes)
There is tremendous precedent for all kinds of drug trials. Design drug trials based on the conclusions of other trials in your therapeutic area, use the data, CRFs and any other assets from these trials. Define the precise data points that you need to collect (don’t over collect data that never gets used), and actionable metrics that point you to the direction of the trial: are we making progress on enrollment?, are the sites enthusiastic about your trial?, can we meet submission deadlines with clean data and accompanying analysis?, is the analysis bullet proof and submission ready. All good questions to ask for a successful study submission.
Here’s hoping you add a few hundred million to your market cap following your next successful FDA study submission.