5 Trends Poised To Accelerate Drug Development

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

In November 2014, Tufts Center for Drug Discovery announced it takes $2.6 billion to push a drug through development and marketing approval — a process that often spans longer than 10 years.

This eye-opening amount of time and money was, of course, met with some skepticism from consumer groups and the media, which argued that the Tufts research — partially funded by drug makers — could simply be trying to justify rising drug prices. (Both The Wall Street Journal blog (WSJ) and The New York Times published pieces highlighting the ways critics approached the news release.)

But critics aside, it’s figures such as these that are inspiring the industry to find new ways to speed drug development. The solutions will vary depending on the type of treatment and the resources available to the companies developing these drugs. However, there have been a few trends taking shape in the industry that are expected to have a big impact on drug development, starting with (though definitely not limited to):

1. Biomarker Identification: The FDA’s Drug Development Tools Qualification Program was established in 2012 to identify useful and appropriate biomarkers, clinical outcomes, assessments, and animal models. However, the agency recently announced that it needs help validating and identifying “promising” biomarker candidates. Identifying these biomarkers would not only help a company identify prospective patients for a certain treatment, but it would also help separate out those patients who might be harmed or would not see any effect from a particular experimental drug. With knowledge like this, researchers could replace a drug’s clinical efficacy endpoint or push a drug more quickly through the testing phase, says Regulatory Affairs Professionals Society. The FDA’s comment request document can be found here in the Federal Register. (I’d also imagine the identification of biomarkers could be particularly important for companies and patients given the amount of chatter about “Right To Try” laws these days. The better a company can identify a drug’s potential effects in patients, the fewer unpleasant surprises in the course of compassionate use and, in turn, the fewer delayed trials.)
2. Multi-Arm Trials: According to a list of 2015 trends and predictions released by Cytel, multi-arm trials are a way the clinical space can make the trial process more efficient. Gone are (or should be) the days of the two-arm trial, as this format clings to the incorrect notion that researchers are only allowed to ask one question in a clinical trial, Cytel says. Given the increasing attention to personalized medicine and patient centricity, we’re clearly living in an age where a trial should be structured to match the complexity of researchers’ questions so they can more quickly confirm what they need to know and get the medicine more efficiently into patients’ hands.
3. Adaptive Early Phase Development: In its list of trends and predictions, Cytel also identified Phase 1/2a exploratory trials for proof of concept and dose-finding as an essential tool to help researchers learn how a drug will affect patients. Adaptive development can provide researchers with actionable information about the drug’s performance even before it reaches Phase 3 trials. The more knowledge gained early on, the better able to tackle problems that could arise and delay a clinical trial. BMC Medical Research Methodology recently published “Three steps to writing adaptive study protocols in the early phase clinical development of new medicines,” for those of you interested in learning more.
4. Big Data: It’s hardly surprising that Big Data would make the list. To date, Big Data seems to be just an alluring concept the industry is still struggling to make actionable. However, in January, Tufts identified Big Data as being one of the top 2015 trends in its annual report. According to Tufts, sponsors and CROs will be turning to Big Data to streamline research/protocol designs, select and engage patients, and improve regulatory submissions — all of which stand to shave some time off of Tufts’ estimated 10 years for drug development. Like Cytel, Tufts also hits on the rising importance of adaptive trial designs to improve quality and success rates. The Tufts 2015 Outlook can be found here.
5. 3-D Printing: There’s been a lot of buzz about 3-D printing over the last year, and quite often accompanying that discussion is a reference to the company Organovo, a high-profile and active player in the space. The company’s goal is to create living human tissue (with a particular focus on the liver currently) and test drugs on this tissue to determine toxicity, eliminating the need to use petri dishes of cells and animals in early-stage drug testing. In a Life Science Leader feature, Organovo’s CEO, Keith Murphy, says, “Three-dimensional bioprinted tissues can help pharmaceutical companies speed up the drug discovery process allowing R&D teams to test new and promising drugs on functional human tissues during hit-to-lead [H2L] and lead optimization stages of drug development. This will help identify potential toxicity and efficacy issues before drugs ever enter clinical studies.” As Murphy tells the WSJ, it often takes a drug entering a clinical trial for a company to realize it’s toxic — a costly discovery that can quickly halt development. (In fact, just this week I read about Eli Lilly’s latest issues with its long-acting insulin treatment, Peglispro. Because it caused an increase in liver fat and other toxicity concerns, the company has pushed back a regulatory filing until after 2016. Yikes.) 3-D printing is still a relatively small market, but it is pegged to grow 14 percent from $425 million to $485 million in 2015.