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Extreme Makeover: Clinical Trials Edition

Posted on December 4th, 2017 by in Pharma R&D

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In a piece featured in the PharmaTimes, Katrina Megget points out the sad state of affairs when it comes to clinical trials, highlighting some appalling stats on the frequent failure to reach enrollment targets (in some cases, not finding any patients at all) and the tendency for studies to go on far longer than anticipated. These problems aren’t a mere annoyance—they cost pharmaceutical companies an enormous amount of money. But change is in the air.“In many ways, the industry has reached a tipping point, and it is waking up to the idea of actively seeking out efficiencies in research and development,” says Megget. She cites trial design innovations, approval and set-up process tweaks, the use of new technology to improve recruitment and participation, data sharing and cutting red tape as ways that clinical trials in particular can become more efficient.

There’s little question that clinical trials are due for re-design. The traditional Phase 1, 2, 3 model is becoming less tenable as precision medicine grows more prevalent. In this informative short video, biotech giant Roche explains how the standard trial structure isn’t suited for today’s targeting of specific mutations, demonstrating two alternative designs, the Umbrella Study and the Bucket (or Basket) Study. The more expansive Umbrella Study tests the impact of different drugs on various mutations in one type of cancer: “Patients are selected based on the genetic mutation most prominent in their tumor and treated with a number of medicines known to target this specific mutation.” The Bucket type, on the other hand, tests “the effect of a single drug or drug combinations on a specific mutation in a variety of cancer types.” Another alternative mentioned is the Adaptive Study design, which allows researchers to make changes during the study, based on how patients are responding.

New approaches to clinical trial design also call for new approaches to patient recruitment. Successfully finding, recruiting and retaining the right patients is a constant headache in trials (the NIH reports that over 80% of US clinical trials fail to keep to enrollment timelines). However, the digital revolution is opening up new avenues for recruitment. In an article on innovation in clinical trials, ClinEdge, an agency that provides patient recruitment services, spotlights digital health (e.g. wearables, mobile apps) and digital media (e.g. Facebook, YouTube) as two technologies that are helping with both patient recruitment and retention. For example, the creation of a free asthma health app made it possible to quickly and easily recruit thousands of participants for an asthma study. Meanwhile, social media, which enables researchers to reach out to huge numbers of people (often using demographic targeting), has already proved to be far more effective than traditional recruitment methods.

Wearables and the digital world are full of possibilities—and challenges—as they bring clinical trials into the Big Data age. Whether gaining patient information from an app, a Fitbit, genetic profiles or many other growing sources of data out there, researchers now must contend with sorting through, processing and making sense of all that information. Elsevier has long been using cutting-edge technology to enhance researchers’ ability to find information in large databases and is now collaborating to share data and help build knowledge networks. Being able to mine all this Big Data has big implications for clinical trials.

In an interview with Applied Clinical Trials, James Streeter of the Oracle Health Sciences Business Unit says, “Analysis of the bigger, more diverse real-world data sets can shed light via visualizations on unknown relationships among clinical trial factors. For instance, in relation to patient centricity, safety, risk-based monitoring and genomics. Taken together, the ability to amalgamate, organize and analyze real-world data sets with structured data sets can only enhance those questions we know to ask of our clinical trials. But additionally, this capability can provide new, provable indications of hidden relationships that can precipitate better support, new hypotheses and provoke new, potentially life-saving questions that we didn’t think to ask before.”

So for all the well-deserved concerns about the difficulties and costs of traditional clinical trials, the upside is that the landscape is changing. New trial designs, recruitment strategies and technologies could all be leading to shorter, yet more effective trials. Sanofi even announced earlier this year that it is launching digital clinical trials. The times are indeed changing, and hopefully for the better.


All opinions shared in this post are the author’s own.

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