Jumo Health Leads Roundtable Discussion with Health Care Professionals to Address RACE Act Impact on Bolstering Treatment Options
On August 18, 2020, the RACE Act went into effect. The RACE (Research to Accelerate Cures and Equity for Children Act) Act amended the Pediatric Research Equity Act (PREA) and requires that life science companies that are developing a targeted cancer therapy for adults must also develop the drug for children with cancer if the molecular target or mechanism of action of the drug is relevant driver to a pediatric cancer. A new drug application (NDA) or biologics license application (BLA) for the study of a new drug in adults submitted on or after August 18, 2020 must include plans for studying the drug in pediatric cancer as well.(1,2)
For children and young adults under 19 years old, cancer is the number one cause of death by disease.(3) Studies have demonstrated that between 30-60% of solid tumors in children with relapsed, refractory, or high-risk cancer have potentially actionable molecular alterations.(4)
Historically, tumors have been defined by histologic subtype. With the availability of next generation sequencing and other molecular diagnostic technologies, we can now identify changes in the genetic and molecular make-up of the cells in a tumor, and thus, tumors can now be stratified by a molecular target rather than just by organ of origin. These molecular targets or alterations can appear in tumors in different organs and of different tumor types. The majority of new cancer drugs over the last decade are designed to target one or more of these molecular alterations instead of the organ. Children’s tumors often appear in different organs than adults but still may have the same genomic mutation as an adult tumor (e.g., ALK alterations in lung cancer and neuroblastoma are quite different but result in same mechanism of activation, thus the drugs developed for lung cancer can work in neuroblastoma as well and other rare tumors). Unfortunately, the majority of these new targeted therapies are developed only for adult patients, and it is very difficult for physicians to access these new, promising therapies in trials and for “off-label” use for children with tumors that have the relevant biology.
The RACE Act mandates changes to this paradigm!
Jumo Health, an industry leader in addressing the challenges of health literacy and its impact on outcomes, convened a panel of 9 industry experts in oncology for a roundtable discussion to explore some of the opportunities and critical challenges with the successful implementation of the RACE Act.
Some key takeaways from that discussion:
- Hospital Expertise. Children with cancer between infancy and 15 years old are almost always seen at a c enter with pediatric expertise or at a children’s hospital that is affiliated with Children’s Oncology Group (COG). However:
- These centers vary in size and resources
- And, thus, they vary in their ability to offer patients relevant clinical trials including Pediatric MATCH. (NCI-COG Pediatric MATCH trials are designed to determine the efficacy of targeted therapies in children and adolescents with solid tumors with specific genetic mutations that have progressed beyond standard of care. Since launch the study has expanded to include 13 different therapy arms matching patients with therapies based on a molecular alteration in their tumors. Pediatric Match is taking place in about 200 children’s hospitals, university medical centers and cancer centers)(5,6)
- Molecular testing gap. Importantly, not all children are getting access to molecular testing which is required for getting access to molecularly targeted therapies and trials studying these drugs. Again, this is a function of the size and location of the center and is further constrained by reimbursement considerations.
- Adolescents and young adults. Adolescents and young adults have experienced less improvement in survival than younger children.(7) There are few hospitals that have centers focused on the specific needs of adolescents.(7) They are often not seen at COG sites, but rather in adult academic and community centers. Adolescents have reduced access to clinical trials and are often not candidates for most adult trials and may not have information or access to pediatric trial. In addition, given their age, there are other health considerations that must be addressed in the context of a treatment or trial.
- Off label data void. Many drugs are being used off label (off label therapies are drugs that are already FDA approved for a target in at least one indication, but are studied or used for non-approved indications) and yet the data is not being captured, aggregated and analyzed in a way that leads to more trials and treatments. However, access to an off-label therapy can be very challenging often due to issues with reimbursement.
- Innovation in study design. The American Society of Clinical Oncology (ASCO) is running a trial called, TAPUR (Targeted Agent and Profiling Utilization Registry), that is studying the off-label use of targeted therapies. Patients as young as12 years old can enroll. Pediatric oncologists often use targeted therapies off label if the proper dosing is available for children with tumors with relevant mutations who have relapsed or are refractory to treatment. TAPUR could be a model/avenue for older children and adolescents for access to off label therapies and data collection. While this trial could be a way for children to access targeted therapies in a trial setting allowing longitudinal data collection, the RACE Act is focused on novel therapies not off-label drugs.
While the roundtable identified the issues and addressed near term opportunities, important topics for discussion remain, such as:
- Should a clinical trial protocol, similar to, but not competitive with Pediatric MATCH, be designed and implemented for industry funded, interventional studies? What should this look like?
- Should different age groups be addressed differently?
- How can we increase molecular testing for children with cancer? Can testing be done earlier in the course of disease? (Early testing in adults with cancer has been demonstrated to have a significant impact on outcomes.)
- Given the rarity of childhood cancers with actionable biomarkers, new models for collaboration, data sharing, and clinical trials will be required. What will these look like?
- There is a need for culturally sensitive educational resources across age cohorts. Regardless of the type of trial or approach, new models of education will be essential for all these efforts. Pharma will need to play a key role in supporting physicians as they educate patients and families about molecular testing and new types of drugs and trials for treating childhood cancer.
Despite these questions and challenges, the stakes are high and we must bring academia, industry and advocacy organizations together in order to ensure a successful implementation of the RACE Act.
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About the Panel
Jennifer Levin Carter, MD, MPH, MBA
Dr. Carter is currently Managing Director of JLC Precision Health Strategies, LLC., a strategy and innovation consulting company partnering with executives and investors to advise data-driven healthcare and life science companies on their commercial and financing strategy, and on the development of novel products and services. Dr. Carter is an entrepreneur, executive and thought leader in precision medicine and drug development focused on improving patient access to emerging technologies and therapeutics. In 2018, she founded TrialzOWN, Inc., a healthcare company in stealth, and was CEO until its prelaunch acquisition by the Flagship Pioneering company, Integral Health, (now Valo Health), in March 2019.
Prior to TrialzOWN, Dr. Carter was the Founder and President of N-of-One®, Inc. She served as acting-CEO from 2008-2012, and Chief Medical Officer from 2012 until its acquisition by Qiagen (Market Cap $8B) in 2019. At N-of-One, Dr. Carter led the creation of award-winning solutions that delivered novel treatment strategies to hundreds of thousands of patients with cancer globally. She is currently on the Board and/or strategic advisor to multiple venture and Private Equity-backed public and private precision medicine, healthcare services, digital health and life science companies. She graduated Phi Beta Kappa, Summa Cum Laude with distinction with a BS in Molecular Biophysics and Biochemistry from Yale University, an MD from Harvard Medical School, an MPH from Harvard School of Public Health, and an MBA from the Sloan School at Massachusetts Institute of Technology.
Susan Cohn, MD
Susan L. Cohn, MD, is a highly respected expert in pediatric cancers and blood diseases. She is a leading authority on neuroblastoma, a cancer of nerve cells and the most common type of cancer found in infants. Dr. Cohn is actively researching several aspects of neuroblastoma. She leads clinical trials for children with newly diagnosed neuroblastoma and is also one of the few pediatric oncologists in the United States who is conducting Phase I clinical trials of promising treatments for patients with relapsed disease. Her team has established the only 131I-meta-iodobenzlyguanidine (MIBG) treatment program in Illinois. She also is conducting research in the laboratory focused on developing more effective treatments for children with this aggressive cancer. Dr. Cohn’s research has received generous support from the National Institutes of Health/National Cancer Institute, the National Institute of Neurological Disorders and Stroke, the St. Baldrick’s Foundation, and Alex’s Lemonade Stand Foundation.
Dr. Cohn has held several leadership positions in national pediatric cooperative clinical research groups, including serving as the first chair of the Children's Oncology Group (COG) Neuroblastoma Disease Committee. She is the co-Chair of the International Neuroblastoma Risk Group (INRG) Task Force, and a past member of the Board of Directors for the American Society of Clinical Oncology (ASCO) and was ASCO Treasurer from 2012-2015. She currently serves on ASCO’s Conquer Cancer Foundation Board of Directors and the St. Baldrick’s Foundation Board of Directors. In 2016, she received the American Society of Clinical Oncology (ASCO) Pediatric Oncology Award, and in 2019, she received the American Association of Cancer Research (AACR) Joseph H. Burchenal Memorial Award for Outstanding Achievement in Clinical Cancer Research. A prolific author, Dr. Cohn has published more than 200 peer-reviewed journal articles, several book chapters, and has co-edited two books. She also serves on the editorial boards for the Journal of Clinical Oncology and Pediatric Blood and Cancer. Dr. Cohn has been selected as a top doctor by Chicago magazine and Top Cancer Doctor by Newsweek and Castle Connolly.
Lia Gore, MD
Dr. Gore is at the Center for Cancer and Blood Disorders and is Professor of Pediatric Hematology/Oncology/Bone Marrow Transplant-Cellular Therapeutics University of Colorado School of Medicine.
Dr. Gore is a board-certified pediatric hematologist/oncologist who specializes in phase I clinical trials with special interests in acute leukemias and bone marrow transplant. Her research focuses on clinical-translational research in novel cancer therapeutics and new oncology drug development.
Dr. Gore received her medical degree from the George Washington University School of Medicine and Health Sciences in Washington, DC, where she also completed her internship in Pediatrics at Children’s National Medical Center. Dr. Gore completed a residency in pediatrics, a fellowship in pediatric hematology/oncology, and a post-doctoral fellowship in leukemia biology at the University of Colorado Program and Children’s Hospital Colorado. She sees pediatric patients at Children’s Hospital Colorado and adult patients at the UC Health/University of Colorado Cancer Center.
Dr. Gore is a member of the American Association of Cancer Research (AACR), the American Society of Clinical Oncology (ASCO), American Society of Hematology (ASH), the American Society of Pediatric Hematology/Oncology (ASPHO), and the Children’s Oncology Group (COG), and serves as an advisor to the US FDA and several European Pediatric Oncology organizations.
Doug Hawkins, MD
Dr. Hawkins is Group Chair of the Children's Oncology Group (COG). Prior to becoming COG Group Chair in 2020, he was the Chair of the COG Soft Tissue Sarcoma Committee, overseeing the conduct of biology studies and clinical trials for rhabdomyosarcoma and other soft tissue sarcomas across North America. He was a member of the COG Bone Tumor Steering Committee.
Dr. Hawkins had focused on clinical research, particularly in the treatment of pediatric sarcomas. He was the chair of two COG clinical trials, one for Ewing sarcoma and another for rhabdomyosarcoma. He chairs the international EURO EWING Consortium External Advisory Board and also chairs the the international Frontline and Relapse in RhabdoMyoSarcoma Study (FaR-RMS) Data Monitoring Committee.
Katherine Janeway, MD
Katherine Janeway, MD, MMSc is a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Her clinical title is Senior Physician. She is an expert in caring for children, adolescents and young adults with sarcomas. She’s been named Top Doctor by Boston Magazine in 2020.
Dr. Janeway’s research is focused on genomics, precision oncology and bone sarcomas. She leads clinical trials in osteosarcoma and Ewing sarcoma both as an independent investigator and as the Chair of the Children’s Oncology Group (COG) Bone Tumor Committee. Dr. Janeway’s work has uncovered the genetic errors or mutations that cause and promote difficult to treat childhood solid malignancies. She has leveraged this understanding to expand treatment opportunities and improve clinical trials for children with advanced childhood solid malignancies. For example, she is part of the leadership team of the first and largest national precision oncology trial in the United States, the National Cancer Institute-COG Pediatric MATCH trial.
In recognition of her achievements demonstrating the utility of sophisticated tumor profiling tests in the pediatric oncology clinic for improved treatment opportunities and clinical trials, Dr. Janeway took on the role of Director of Clinical Genomics at Dana-Farber Cancer Institute in 2018. Dr. Janeway’s research group is currently focused on better characterizing the clinical impact of genomic sequencing. The main project in her laboratory addressing this question is the iCat2/GAIN consortium study which has enrolled and sequenced over 500 patients with difficult to treat childhood solid cancers. Her group is also contributing to efforts to share and aggregate both sequencing and clinical data to enable a deeper understanding of childhood cancers. In particular, the group has several data generation and sharing projects aiming to better characterize the genetic events giving rise to and sustaining osteosarcoma and Ewing sarcoma. With these projects they hope to identify clinical and genomic factors that explain prognosis and treatment response and resistance.
John Maris, MD
Dr. John Maris is Giulio D’Angio Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania and the Children’s Hospital of Philadelphia. He is a physician-scientist who has focused for over three decades on the childhood cancer neuroblastoma with the dual goals of improving patient outcomes and also using the disease as a model to understand cancer in general. His group has discovered all of the known neuroblastoma susceptibility genes and his group has also identified many of the oncogenic drivers of the disease. Dr. Maris has steadfastly sought to translate these discoveries to the clinic using precision medicine approaches and more recently by co-leading a multi-institutional St. Baldrick’s Foundation-Stand Up to Cancer Pediatric Cancer Dream Team project to bring the fields of genomics and immunology together to combat childhood cancers, and more recently a Beau Biden Moonshot Center Award to extend this rapidly evolving area of research.
Dr. Maris is an internationally recognized practicing pediatric oncologist who cares for children with refractory neuroblastoma from around the world, typically in the context of early phase clinical trials. Dr. Maris has been continuously funded by the National Institutes of Health and many other funding bodies. He currently holds a National Cancer Institute Outstanding Investigator Award and has received several prestigious awards including election into the American Society of Clinical Investigation, the Oski award for outstanding pediatric oncologists, and the Berwick award at Penn for melding basic and clinical teaching, and the William Osler Patient Oriented Research Award at Penn.
Yael Mosse, MD
Dr. Mossé is a tenured Associate Professor at The Children's Hospital of Philadelphia and the Perelman School of Medicine of the University of Pennsylvania. Her clinical and research specialty is neuroblastoma, a cancer that starts in the nerve tissues of infants and young children. She received her M.D. from the Sackler School of Medicine, Tel Aviv, Israel, in 1997, joining the Children's Hospital residency program that year, followed by fellowship training at the same hospital starting in 2000. She trained in the laboratory of John Maris, MD and now has her own independent NIH-funded research program where she investigates the hereditary predisposition and progression of neuroblastoma with a focus on drug development. She garnered international attention for her discovery of germline and somatic mutations in the ALK (anaplastic lymphoma kinase) gene that occur in some neuroblastomas.
Changes, or mutations, in the ALK gene play a role in the growth of cancer cells and has also been linked to lung cancer and lymphoma. Her group has shown that these mutations can be specifically targeted with a drug called crizotinib, a finding that has led to integration of ALK testing and ALK inhibition in the next Children’s Oncology Group Phase 3 trial for patients with newly diagnosed high-risk neuroblastoma. The ALK mutation occurs in about 14 percent of patients with the most aggressive form of neuroblastoma, and ALK expression is found on 90 percent of neuroblastomas. Recent early-phase research conducted in the lab showed that an antibody targeting ALK expression kills neuroblastoma cells, an alternative therapeutic strategy being developed in the lab.
Theodore Laetsch, MD
Dr. Laetsch is an Associate Professor of Pediatrics at the University of Pennsylvania / Children's Hospital of Philadelphia and chair of the Rare Tumors Committee for the Children’s Oncology Group (COG). He received his MD from the University of California, San Francisco and completed his internship, residency and was chief resident in Pediatrics at the Children's Hospital of Colorado. He was a fellow in pediatric hematology-oncology at the Children's Hospital of Philadelphia before joining the faculty at the University of Texas Southwestern Medical Center.
In 2020, he returned to the Children's Hospital of Philadelphia, where he is the inaugural director of the very rare malignant tumor program and leads the developmental therapeutics program. Dr. Laetsch is active in early phase drug development nationally, as a member of the Pediatric Subcommittee of the FDA Oncologic Drugs Advisory Committee, Pediatric NCI/COG MATCH Target and Agent Prioritization Committee, and national/international chair of three Children’s Oncology Group therapeutic studies.
Joshua Schiffman, MD
Dr. Schiffman is the CEO and Co-Founder of PEEL Therapeutics, delivering evolution-inspired medicine to patients. He also co-founded ItRunsInMyFamily.com, an online family history collection tool. Dr. Schiffman is a Professor of Pediatrics and Investigator at Huntsman Cancer Institute at the University of Utah and recently served as the Medical Director for the Family Cancer Assessment Clinic. Dr. Schiffman graduated from the Brown University School of Medicine in 2000, followed by clinical training in Pediatrics and Pediatric Hematology-Oncology at Stanford University from 2000-2008. His academic research and now commercial efforts focus on genomic development of cancer in children and studying animals that naturally are protected from cancer, like elephants. Dr. Schiffman is Board Certified in Pediatric Hematology-Oncology.
Richard L. Schilsky, MD, FACP, FSCT, FASCO
Dr. Schilsky, a board-certified internist and medical oncologist, is the Executive Vice President and Chief Medical Officer of the American Society of Clinical Oncology (ASCO), the world’s largest professional organization representing physicians who care for people with cancer. In this role, Dr. Schilsky is involved in professional and patient education, quality improvement, policy and advocacy, communications, clinical research and development of CancerLinQ, ASCO’s rapid learning system for oncology. Prior to joining ASCO, Dr. Schilsky was a faculty member at the University of Chicago from 1984-2012. He served as Director of the University of Chicago Cancer Research Center (1991-99), as Associate Deanfor Clinical Research (1999-2007) and as Chief of the Section of Hematology-Oncology (2009-2012).
Before joining the U of C faculty, Dr. Schilsky served as Assistant Professor of Medicine at the University of Missouri-Columbia School of Medicine from 1981-1984. An international expert in gastrointestinal malignancies and cancer pharmacology Dr. Schilsky served as Chairman of the Cancer and Leukemia Group B, an NCI-sponsored national cancer clinical trials group from 1995-2010. He has served on a number of peer review and advisory committees for the National Cancer Institute (NCI) including as a member and chair of the NCI Board of Scientific Advisors and as a member of the Clinical and Translational Research Advisory Committee. Dr. Schilsky also served as a member and chair of the Oncologic Drugs Advisory Committee of the Food and Drug Administration.
He presently serves as a member of the Board of Directors of the Reagan-Udall Foundation for the FDA, a member of the Board of Directors of Friends of Cancer Research and as a member of the National Cancer Policy Forum of the National Academy of Medicine. Dr. Schilsky has served as a member of the Board of Directors of ASCO and of the Conquer Cancer Foundation of ASCO and as ASCO President 2008-2009. Dr. Schilsky also plays a leading role in several international cancer research organizations currently serving as Chairman of the WIN Consortium, as chairman of the Protocol Review Committee for the European Organization for Research and Treatment of Cancer (EORTC) and as co-chair of the Scientific Advisory Board of the Ontario Institute for Cancer Research. Dr. Schilsky earned his M.D. at the University of Chicago Pritzker School of Medicine in 1975. Following a residency in Internal Medicine at the University of Texas Southwestern Medical Center and Parkland Memorial Hospital, he received training in Medical Oncology and Clinical Pharmacology at the National Cancer Institute (NCI) from 1977 to 1981.
2. PRA Health Sciences: The RACE Act: Your next steps start here.
3. PRA Health Sciences White Paper; RACE Act Prompts More Pediatric Trials
4. Grobner SN et al. (2018) The landscape of genomic alterations across childhood cancers. Nature. 555:321-340.
6. Children’s Oncology Group
7. Trama, A et.alI Is the cancer survival improvement in European and American adolescent and young adults still Lagging behind that in children? AYA Oncology Review. Pediatric Blood Cancer. 2019.
About Jumo Health
Jumo Health develops age appropriate, educational resources for patients and their care circle for use throughout their medical journey.
By working with providers, manufacturers, and advocacy groups, we ensure our resources are available at the moment of diagnosis, during a treatment regimen, or while participating in a clinical trial. With the belief that an informed patient is a compliant patient, Jumo Health designs practical solutions using popular mediums. With experience providing resources to more than 70 countries in 80 languages, covering more than 200 topics, our mixed media solutions range from comic books to animated videos, are evidence based and peer reviewed, and pay careful attention to health literacy and reading comprehension barriers.