Three “Givens” That Will Always Drive Successful Drug Development

Ken Getz, Associate Professor and Director of Sponsored Research, Tufts CSDD

Ken Getz, Associate Professor and Director of Sponsored Research, Tufts CSDD

There is much to celebrate as we reach ACRP’s 40th anniversary. The clinical research enterprise has been remarkably productive, and has delivered important medical therapies across a wide range of disease conditions. Research and development pipelines have grown steadily during the past four decades, fed by such innovation drivers as high-throughput screening, proteomics, and genomics. Rising numbers of promising new medical therapies demand greater capacity from, and coordination among, a global community of clinical research stakeholders.

The next 40 years look as exciting as the last. Trends and changes are unfolding that will profoundly impact how our enterprise manages its innovations and how it will operate and perform, including the growth of precision medicine; the consolidation of research sponsors and service providers; the convergence of clinical research and clinical practice; the use of wearable and mobile devices and new technology solutions; and more sophisticated uses of large and growing structured and unstructured data and information for scientific and management purposes.

Perennial “Givens” for the Next 40 Years

Other well-wishers in this issue of Clinical Researcher touch on and predict where these exciting developments will take us, and the changes and challenges that must be anticipated along the way. I wanted to share some consistent themes, or “givens,” that will be as important—if not more so—in the next 40 years as they have been in the past four decades. Throughout my career as an observer of the clinical research enterprise, I generally invoke these three “givens” when I riff on the implications of any new research that I have conducted:

  1. Relationships drive a successful clinical research enterprise

    No matter how hard we try to drive speed and efficiency with new technology solutions and process improvements, or how tightly we manage study timelines, success always rests with the quality and caliber of our relationships. The time and care that we take to build expectations and solidify as a team; to establish alignment; to clearly delineate roles and responsibilities; to convey respect and establish trust; to invite and accommodate timely input; to give partners the support and room that they need to excel—time and time again, these are the characteristics that define drug development success.

    Research from a variety of sources (e.g., ACRP, Avoca, the Clinical Trials Transformation Initiative, CenterWatch, TransCelerate, Tufts Center for the Study of Drug Development, etc.) has shown that there are a number of essential and integral relationships that are neglected and often dysfunctional. Sponsor relationships with contract research organizations (CROs) and sponsor/ CRO–investigative site relationships are two such areas. Recent attention on improving the relationship between clinical research professionals and patients/advocacy groups/the public/healthcare providers/payers—and impassioned efforts to establish and drive higher levels of engagement and partnership with these communities—will go far in helping the clinical research enterprise to achieve success in the future.

  2. Transparency and disclosure are essential

    Stakeholders throughout the clinical research enterprise readily agree that transparency and disclosure are the critical building blocks of trusting and lasting relationships. Moving forward, we must commit ourselves to improving transparency and disclosure, and to raising our standards to as high a degree as possible. This includes not only increasing the amount of and access to data and information, but also to providing it in languages and terminology that can be best understood by communities of varied and diverse levels of comprehension.

    Industry-wide behaviors often belie our general agreement with this “given.” To name but a few areas, operating and financial data often are not shared between sponsors, CROs, and investigative sites; public and private sector conflicts-of-interests are not consistently and adequately disclosed; poor compliance and incomplete and highly technical information in ClinicalTrials.gov renders this public information source insufficient; and although we are obligated to do so, most sponsors are not providing clinical trial results to their study volunteers.

  3. Balance must be achieved between scientific excellence and feasible execution

    Scholarly research has consistently shown that scientific and logistical complexity is inversely related to high performance and efficiency. As drug development programs have become more complex, so too have they become costlier, riskier, and longer in duration.

    Partners in any relationship succeed when their expected contributions are feasible and achievable. For our study volunteers, feasibility includes not only access to clinical trials, but also convenience and comfort. Time will be well spent challenging great drug development science at the outset, so that it can be viably executed and all partners in a given program can succeed in supporting it.

    Today’s study designs have the highest relative number of procedures and eligibility criteria on record. Our operating models frequently involve complex and fragmented, poorly coordinated global teams of internal and contract service personnel. In response, some of the impacts include how contract and budget negotiations are protracted; study start-up and overall cycle times are extended well beyond initial plan; recruitment and retention rates continue to decline; change orders and protocol amendments increase; and investigative site performance varies greatly, with a high percentage failing to enroll or under-enrolling study volunteers.

    No doubt the results of our scholarly research will continue to fuel my riffs and rants on these “givens.” They are key principles that will go far in delivering successful drug development programs regardless of individual strategies and tactics employed. Further, through training, certification, education, communication, and networking, ACRP will play a major role in helping to ensure that these “givens” are addressed!

    Best wishes and congratulations to ACRP on its 40th anniversary.

Kenneth A. Getz, MBA, (Kenneth.getz@tufts.edu) is the chair of the nonprofit Center for Information & Study on Clinical Research Participation and director of sponsored research and an associate professor at the Tufts Center for the Study of Drug Development, Tufts University School of Medicine. He is also the founder and owner of CenterWatch and a co-owner of the Metrics Champion Consortium.

[DOI: 10.14524/CR-16-4012]