Benefiting from Innovative Clinical Trial Designs

Benefiting from Innovative Clinical Trial Designs

Clinical trial success rates have been known to be low for some time now, but that does not imply they will remain low forever. As more tools become available to clinical trial sponsors, technical operators and even patients, future clinical success may look nothing like it has over the last decade.

Taking steps to change old fashioned protocols were panelist that gathered for today’s panel on clinical trial innovation trends at the BIO Investor Forum (San Francisco). The panel session, moderated by Komathi Stem of ReThynk Consulting, showcased some of the latest approaches currently being used to increase success and improve productivity in clinical development. Two of the panelists are currently focused on using biomarkers in trials for cancer and neurology treatments and a third is concentrating on digital methods to optimize clinical benefit and compliance:

The discussion started off on the topic of digital tools in clinical development. Pear Therapeutics is a 5AM, Jazz Ventures backed company creating “digital pharmaceuticals”. What that means is that they make user-friendly, customizable software apps for patients taking a specific drug or for patients responsive to digital intervention. Areas of focus where such technology can work are addiction, schizophrenia, pain, post-traumatic stress disorder, anxiety, depression, and sleep.

Beth Rogozinski says that one of the motivations behind the usage of IT in the clinical setting is that patients are more honest with entering data into an app on a mobile device than interfacing with a doctor. The early rollout for these technologies is for subjective Q&A to be entered on a device, but new apps will soon be analyzing voice and other biometric data to detect physical changes in patients.

Patient Reported Outcomes (PROs) will also be captured more and more by mobile device interaction as the field matures. The hope is that there will be an integration of data input such that PRO data is compiled only once (at time of patient entry) and not re-entered into multiple databases.

One takeaway from all these alternative protocols is that companies should strive to “capture it all” when it comes to clinical data – you never know what data you will need one day as new questions about a therapy or the disease progression itself arises.

Melissa Paoloni brought up “Platform trials” as an alternative, adaptive means for clinical development. She believes that these trial paradigms, such as the I-SPY design, will eventually be used outside oncology. An interesting trait that these studies have is that they are potentially never ending – as new trial arms start when old arms are completed. They are disease-focused rather than single drug-focused. As there are many sub-populations even within certain cancers, there will always be a search for a better therapy. The trick is coordinating the new therapy entrants into the consortium of pharmaceutical, biotechnology and diagnostics companies, then managing all the logistics.

Aidan Power added to the biomarker discussion pointing out that a trial must have a validated biomarker plus utility for the specific indication. The challenge in Alzheimer’s, (for example with tau, amyloid imaging, etc.) is how well the diagnostic ties to efficacy. Early on you may not know how correlated these are. Further complicating things in co-development, is that incentives on a financial level may be diverge for the small biomarker company vs the drug developer’s incentives and timelines.

The session was also supplemented with a slide presentation on just how much biomarkers can impact success in the clinic. Below is a summary slide of our recent work on biomarkers, co-published with Amplion and Biomedtracker (download report here: www.bio.org/iareports).Looking at this chart, it is no wonder companies are looking to stratify patient populations to yield homogenous trial participants.

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