Clinical Research Introduction
Much research is done to identify new and better ways of treating cancer while preserving quality of life.
When this research involves testing treatments on patients, it is called a cancer clinical trial or clinical research study.
Clinical trials assess
- value of existing treatments
- modifications to reduce toxicity
- potentially promising new treatments
- new approaches to assessing treatments
Investigators recruit patients who meet predetermined criteria, deliver treatment and collect data over a defined time period for analysis. They usually cannot predict whether individual participants will benefit from the approach under investigation.
Participants are carefully monitored during and after treatment, and this may involve more tests and clinic visits than might otherwise be expected. However this close monitoring provides the most valuable evidence to improve care.
When clinical research shows a treatment is not dangerously toxic, follow-up research is done. The treatment is compared with standard therapies, and adjusted to minimize side effects.
A reliable clinical trial may require hundreds or thousands of participants in its latter stages. This is because researchers must find small, but important, differences between the new and standard treatment.
To show a 10% (1:10) improvement in effectiveness of a therapy, the trial requires 1,000 patients. To show a 5% (1:20) improvement, 4,000 patients would be needed.
Clinical Trial Design
Clinical trials are designed by medical and research experts. Frequently, experts from many different hospitals work together on clinical trial design.
Major childhood cancer centres collaborate on research design and management with national or international childhood cancer study groups. For example: the Children’s Oncology Group (COG) in North America and the UK Childhood Cancer and Leukaemia Group (CCLG).
Many childhood cancer centres also design their own research studies. This is particularly common in investigating rare, underfunded cancers like retinoblastoma.
Clinical trials and research studies are led by a research team, including doctors and specialist research nurses. The research protocol should clearly identify who has designed the study, the principal researchers and collaborating centres/groups.
Within research groups and individual treatment centres, clinical trials and clinical research studies are approved by a Research Ethics Board or Institutional Review Board. These boards may include research scientists, doctors, nurses, trustees, patient representatives and members of the public. This body has a responsibility to
- protect participants from harm
- assure participants understand their treatment is a research protocol
- ensure a rigorous scientific approach to obtain reliable evidence
If unacceptable side effects emerge, the Committee should take appropriate action to ensure patients are protected. Similarly, if a treatment is found to be less effective than a standard therapy, the Committee should act to ensure patients receive best care.
Funding and Payment
Clinical research may be funded by government or a National Cancer Institute. Research is also routinely funded by pharmaceutical and biotechnology companies, universities, hospitals, research foundations and charities.
Funders may have vested interests in results. For example, effectiveness of modified or new therapy will impact sales for a pharmaceutical sponsor. A widely published researcher may generate more funding for a university or hospital. Protocols should include information about who is funding the research, and any conflicts of interest.
Clinical investigators are sometimes paid to conduct specific research. To prevent bias, they should not accept payment except for impartial research support.