Modernizing Clinical Research
January 29, 2015
This week, the US House of Representatives’ Energy & Commerce Committee released a discussion draft of the legislation to advance the committee’s 21st Century Cures plan to accelerate the pace of cures. Quintiles was invited to provide testimony to the subcommittee last July in support of the 21st Century Cures and Modernization of Clinical Trials.
1. Accelerate patient recruitmentFinding the right patients for the right trials, especially with increasingly complex inclusion/exclusion criteria of more targeted therapies, is one of the biggest time challenges in the drug development process today. Slow recruitment can add years and millions of dollars to the discovery process.
These obstacles can be overcome through the use of master protocols and adaptive designs to target therapies to the right patients, to identify responding/non-responding patients faster, and reduce redundancy of new or separate trials. Quintiles currently uses biostatistics experts to help establish methodologies for such adaptive design models, which allows researchers to answer critical questions about dosing, patient populations and study design before investing time and resources in a new clinical trial.
My recommendation to Congress: Encourage the FDA to set goals for more frequent use of master protocols and adaptive designs to maximize identification of drugs that work in the patient population without having to duplicate efforts across multiple sponsors. Congress could consider requiring a certain percentage (perhaps 10%) of therapies entering Phase II to include master protocols and adaptive designs and that regulatory standards and approval criteria be clarified to encourage multiple biopharma companies to collaborate on the use of these tools.
3. Standardize and Harmonize
Redundancies and inefficiencies in the clinical trial start-up process adds substantial time and cost to the trial process. These include Institutional Review Board (IRB) approval, contracting, and lack of data standards. We can reduce this waste without impacting the integrity of the research by creating reciprocal or central IRB approval, and establishing industry data standards, common trial documents, and regulatory harmonization. In our experience at Quintiles, the use of central IRBs alone can cut the time to start an investigative site from more than 100 days to just 45.
My recommendations to Congress: Congress should look for opportunities to support standardization and harmonization of these processes. That may include:
4. Support alternative development pathways
The ultimate goal of this effort, and the healthcare industry overall, is to find more efficient ways to bring lifesaving drugs to market. One important solution is to create alternative development pathways for serious unmet medical needs. This would reduce development time as well as FDA review/approval timelines. At Quintiles, we have found that better data analytics, use of genomics and biomarkers for more precise identification of sub-populations, patient registries, and other modes of collecting and analyzing real world data in targeted patient populations can help shorten development and delivery time while still maintaining the integrity of the results.
My recommendation to Congress: Congress could encourage FDA to pilot alternative development pathways similar to the Adaptive Licensing approach that the European Medicines Agency (EMA) is now piloting, whereby treatments could be tested and approved for limited use/ populations, while ongoing studies would still be required.
One of the driving priorities in the industry today is to modernize the clinical trial process as part of the broader effort to increase the pace of drug development. The 21st Century Cures Initiative demonstrates the power of cross-industry collaboration, and the many opportunities that policymakers and regulators have to further drive efficiencies that will help us all deliver better, faster trials and therapies to patients who need them. It is my hope that by working collaboratively across government, academia and industry stakeholder groups, we can identify and implement solutions that will save lives, cut costs, and streamline the process of bringing lifesaving treatments to those most in need.