Clinical Trial Design and Enrollment

Clinical trials, a critical component in the development of innovative treatments, are especially critical in oncology, a field in which first line therapies aren’t always effective at delaying the disease progression and often aren’t curative. To ensure the continued development of effective oncology treatments, the cancer community must address the problems of poor patient recruitment and retention and inefficient trial design and execution.

We have developed three work streams to improve clinical trial design and access, focused in the following areas:

  1. Improve the pediatric oncology drug development pipeline
  2. Clinical trial access and navigation
  3. Clinical trial design to reach additional patients

OUR APPROACH

Improve the pediatric oncology drug development pipeline

  • Cancer remains the leading cause of death by disease for children in the U.S.
  • While over 1,000 new therapeutics are currently being tested in clinical trials for adult oncology indications, very few are being evaluated simultaneously in pediatric patients. This is despite legislation requiring pediatric evaluation of approved products.
  • Drugs that fail to achieve proof-of-concept or approval for an adult indication frequently have their development curtailed regardless of whether or not they may be of utility in a pediatric cancer population. Pediatric patients have unique needs, including distinct biology from adult cancers, drug formulation changes, dose modifications, and the evaluation of toxicities that may present differently than adults.

Goal:  To identify and support opportunities to improve the system for pediatric oncology treatment development

CLINICAL TRIAL ACCESS AND NAVIGATION

  • Less than 10% of adult cancer patients will ever participate in a clinical trial;
  • Approximately half of cancer-related clinical trials fail to reach their accrual target;
  • The majority of patients indicate they would be amenable to participation in clinical trials, but cite challenges focused on lack of awareness and understanding as the greatest barriers to participation.

Goal: To identify and pursue opportunities to deliver a comprehensive and user-friendly clinical trial resource to patients and their doctors

Program: Oncology Clinical Trial Information Commons (OCTIC)

CLINICAL TRIAL DESIGN

  • Patients in the community oncology setting have less knowledge of, and access to clinical trials.
  • Even if patients get to trial enrollment, the travel demands and time commitment to stay on a trial are a challenge.
  • There are examples of success in reaching more and diverse patient populations that we can learn from and expand.

Goal: To engage key groups (i.e. community oncology practices, patient groups, cancer centers, biopharmaceutical and biotechnology companies) to learn successful strategies and identify new sectors and partners to make game-changing improvements in clinical trial design and reach.

WORKING GROUP MEMBERS

  • Peter Adamson, M.D., Chair, Children’s Oncology Group; Alan R. Cohen Endowed Chair in Pediatrics; Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Children’s Hospital of Philadelphia (CHOP)
  • Nilo Azad, M.D., Associate Professor of Oncology, Johns Hopkins University
  • Carol Brown, M.D., Surgeon; Director, Office of Diversity Programs in Clinical Care, Research, and Training, Memorial Sloan Kettering Cancer Center (Board Member)
  • Craig Lipset, Head of Clinical Innovation, Worldwide Research & Development, Pfizer
  • Frank Prendergast, M.D., Ph.D., Advisor, WellNovation
  • Kim Thiboldeaux, CEO, Cancer Support Community (Board Member)
  • Karen Winkfield, M.D., Ph.D., Associate Director for Cancer Health Equity, Wake Forest Baptist Comprehensive Cancer Center

RELEVANT RESOURCES

Video Module: Understanding the oncology research and development pipeline
Video Module: Harnessing innovation to improve and save lives
Commitments made during the Biden Cancer Summit to improve clinical trials