The TMF is the official record of the clinical study for Regulatory Authorities, be it…
Pediatric Hematology Oncology is a relatively recent area of study. Only in the past 50 years have substantial advances taken place in the understanding of immunology, biochemistry, enzymology, genetics, molecular genetics, histology and pathology of the different childhood malignancies.
The majority of the research and therapies studied and approved to date have been targeted to cancers in the adult population. As such there is a compelling requirement for pediatric clinical research to support the expansion of clinical treatments for childhood cancers.
Since the late 1960s Pediatric Hematology Oncology has developed into a vast and complex specialty to study the alteration of the functions of the elements of blood, bone marrow, solid tissues and organs. The terms childhood cancer or pediatric cancer are used interchangeably to refer to malignancies that occur from birth to 18 years of age.
In 1973 the sub-specialty of Pediatric Hematology Oncology was approved by the American Board of Medical Specialists. In 1974 the American Board of Pediatrics had devised a certification in Pediatric Hematology Oncology and by the year 2000, 1,602 Pediatric Hematology Oncologists were certified.
Today this specialty comprises experts ranging from practitioners, researchers and scientists across many disciplines, all working toward one common goal and that is to continue to improve childhood survival rates and provide adequate treatment options to patients.
Predominant Types of Pediatric Cancers in Canada:
The most common types of pediatric cancer found in Canadian children are leukemia (33%) followed by brain and central nervous system (CNS) (20%) and lymphoma (11%). Other common types of cancers diagnosed in children include tumors of the peripheral nervous system (Neuroblastoma), tumors of muscle, bone or soft tissue (sarcomas), and tumors of the kidney (Wilms tumor).
Treatment Landscape in Canada:
Medical practitioners and scientists are always looking to improve and advance treatment and discover new treatments that can effectively treat the disease, and provide minimal side effects and relief of symptoms. Before a new treatment can be made widely available it must be tested and studied in a clinical trial to ensure it is safe and effective, and possibly better than an existing treatment.
Approximately 900 children are diagnosed with cancer every year in Canada, where the incidence is highest among infants under the age of one (248 per 1,000,000) and lowest among children between 10-14 years (123 per 1,000,000). However, pediatric cancers account for the second leading cause of death by disease among Canadian children.
For many types of childhood cancer, treatment based on a clinical trial protocol gives a survival advantage. In Canada, approximately 80% of children living with cancer are either enrolled in a clinical trial or receiving treatment according to a registered protocol.
There are 17 pediatric cancer centers across Canada offering care and effective treatment to children with cancer. There are many different types of treatments available where each treatment will depend on the type of cancer and how advanced it is. Common treatments include surgery, chemotherapy, radiation therapy, immunotherapy, targeted therapy and stem cell transplant. Some treatments are comprised of intensive multimodal therapy therefore providing a combination of chemotherapy, radiotherapy and/or surgery, for example while other treatments include only one modality.
Between 1995 and 2000 the median time between first contact with a health care professional and the start of treatment was 17 days.
Advancement in diagnosis, adequate treatment options and disease management, has seen the outlook for these children improve tremendously over time. As improvements continue, processes to ensure pediatric cancer patients start treatment much sooner and median time between consecutive events to diagnosis and initiation of treatment have significantly reduced further over the past 20 years.
Cure rates have for the most part improved with the use of multiple treatment modalities, new treatment therapies and better supportive care. The 5-year survival rate of pediatric patients is as high as 80%, which means that 80% of patients are expected to survive for at least 5 years following diagnosis. According to Cancer Care Ontario this is a 20% improvement compared to rates of 20 to 30 years ago when many children did not survive.
Mitigating the Impact of the Disease on Children and Their Families:
Survivors are at risk for acute, chronic and/or long-term health problems as a result of their cancer and its treatment (e.g. second cancers, reduced fertility, psychological disorders). Management of the disease along with the side effects exhibited by each child during and after treatment have quite an impact on the patients and their families. Nonetheless, continued research is aimed at advancing treatments and agents to decrease toxicity and improve prognosis and quality of life.
It is important to note that Canadian pediatric treatment centers offer services and resources for families and patients. Furthermore, there are innumerable resources, programs, organizations, and foundations that are dedicated to helping families and patients in Canada and serve as a conduit for providing tools and guidance, such as the: Canadian Cancer Survivor Network; Childhood Cancer Canada; Canadian Cancer Society; Cancer Assistance Program; Kids Cancer Care Alberta; and Cancer Care Ontario.
Trial Sponsors’ Priorities:
Selection criteria used by Sponsors of clinical CROs is primarily focused on finding the right match between the two organizations. Findings indicate that Sponsors are most concerned by factors such as: the CRO’s overall experience in the study’s therapeutic area and experience in the same indication (61%); can the Sponsor work with the CRO (63%); and the perception by the Sponsor of the CRO’s dedication (62%).
For patient centric trials, pediatric or otherwise, CRAs with the necessary therapeutic expertise play an important supporting role. As the site personnel’s primary point of contact with the Sponsor, CRAs are positioned to best represent the values of the clinical trial team to sites and study participants. When site personnel are advocating on behalf of a patient’s questions or concerns, the CRA is an ally who conveys these concerns to the country lead for further discussion with the clinical trial team.
With an experienced, expert and supportive CRO team, site personnel can more effectively adapt clinical trials to suit the needs of their study participants, resulting in a continued access to cutting edge oncology study drugs for participants.
This Niche Canadian Oncology CRO – Consistently Goes Above-and-Beyond:
With 23 years unparalleled Canadian oncology experience Scimega Research has an optimal operations design critical to its ability to consistently go above-and-beyond for every one of your studies.
Scimega is a Canadian niche oncology CRO that serves small to medium size biotech companies in clinical development of cutting-edge oncology therapies. We consistently deliver our services with a strategic focus on Sponsor program success with clinical trial start-up times 50% faster than the North American average, premium quality data, and trial sites that are often among top enrollers in our Sponsors’ global oncology studies.
Dimah Nassar, Clinical Research Associate
Cancer Care Ontario. https://www.cancercareontario.ca/en/cancer-facts/cancer-rates-increasing-among-children-ontario Accessed 18Nov2020.
Cancer.net: https://www.cancer.net/cancer-types/childhood-cancer/about-clinical-trials Accessed 18Nov2020.
Government of Canada: (https://www.canada.ca/en/public-health/services/chronic-diseases/cancer/cancer-children-canada-0-14-years.html Accessed 16Nov2020
Ma X, Liu Y, Liu Y, et al. Pan-cancer genome and transcriptome analyses of 1,699 pediatric leukemias and solid tumors. Nature. 2018. doi: 10.1038/nature25795 Accessed from ScienceDaily: St. Jude Children’s Research Hospital. Genomic analysis underscores need for precision therapies that target pediatric cancer. Published February 28 2018. https://www.sciencedaily.com/releases/2018/02/180228131126.htm Accessed 16Nov2020
National Cancer Institute- Childhood Cancers (NCI): https://www.cancer.gov/types/childhood-cancers Accessed 16Nov2020
Nature: Pediatric Research: https://www.nature.com/articles/pr2002282 accessed 17Nov2020