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. 

 
When I talk to our biopharma partners, they most often say that reducing the time and expense of developing novel therapies is their most pressing business need — and it’s no wonder why. A recent report from Tufts shows that it still takes more than a decade and roughly $2.5 billion to bring a new drug to market. The FDA approved 41 new molecular and biologic entities in 2014 – a 20% increase from 2013. This is certainly an achievement worth noting, yet the drug development cycle is still too long and too expensive, and we need to modernize this process if we are ever going to meet the goals of delivering better medicines faster, at less cost, to the patients who need them.

As I look ahead into 2015, I see several areas in which we as an industry can improve the clinical research process. I was honored to present some of these ideas in a US Congressional Testimony earlier this past summer, along with experts from Harvard University, Yale University, the Mayo Clinic, and Johnson & Johnson. The event was in support of the 21st Century Cures Initiative, a bi-partisan Congressional effort sponsored by the U.S. Energy & Commerce Committee to identify actions Congress can take to accelerate the pace of cures in America. The end goal of the effort is to generate legislation that will effect changes and drive new cures, faster.

I was excited to participate in this process because we at Quintiles see many opportunities for policymakers and regulators to drive efficiencies that will help us all deliver better, faster trials and therapies to patients who need them. In the session I focused on three areas of opportunity -- patients, processes, and pathways. Below is a summary of the challenges I think Congress can address through legislation, and my recommendations to the panel on how they can begin this journey.

1. Accelerate patient recruitment

Finding 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.

To simplify the recruiting experience, we need to implement processes, technologies, and legislation that will enable better data collection to find the right patients more efficiently, and establish sites in areas where they reside.  At Quintiles we’ve already seen acceleration in patient recruitment through the use of online communities that reach more than three million patients; prime and partner site relationships to improve awareness of where the right patients are; the use of genomics for rapid patient screening and pre-profiling; and analyzing de-identified EMR data from more than 61 million patients.

My recommendation to Congress: Congress can drive further improvements in recruiting by increasing the quality and accessibility of data for researchers. That can be accomplished by:
  • Creating incremental improvements in linkages between Electronic Health Records (EHR) and clinical research databases.
  • Driving EHR interoperability.
  • Examining possible misinterpretations of data privacy rules that hamper use of de-identified data for research.
2. Support adaptive trial design

The traditional clinical trial design model was not built to adapt to new learnings about patient subpopulations, which limits the amount of patient data available from a single trial. Clinical trial productivity is reduced and costs increased by the need for each sponsor to conduct separate development programs in the same patient population for the same indications, for similar molecules. As a result, drug development failure rates are high, time is wasted and patient participation is not optimized.

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:

  • Exploring ways the FDA and the National Institute of Health (NIH) can encourage the use of central IRBs and reciprocal agreements.
  • Looking for opportunities to drive standardization of clinical trial protocols and data collection requirements.
  • Setting goals for FDA around international harmonization.

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.