New cancer drugs in clinical studies can be life-saving. Currently large groups of patients are receiving the newest drugs, but these drugs are often only effective for a small percentage of patients. To study which patients really benefit from the treatment, doctors and researchers need access to the clinical data and tissue samples that patients provide in clinical studies.
Lack of reliable biomarkers
New breast cancer drugs often work very well in a small group of patients with specific tumour characteristics. For instance, adding immune therapy to chemotherapy in case of local triple negative breast cancer, reduces the risk of mortality by approximately 5 percent after 5 years. This means that a small group benefits greatly from the treatment. Nevertheless, the latest immunotherapy is usually given to all patients, due to the lack of reliable biomarkers that can predict in advance who will benefit from the drug.
An international group of 53 breast cancer experts and patients representatives is therefore calling for better cooperation between academic institutions and the pharmaceutical sector. The results have just been published in the medical journal JAMA Oncology.
20 years of breast cancer medication
The research team, led by UZ Leuven and KU Leuven, examined what happens to tumour samples and clinical data of breast cancer patients that participate in clinical trials for new cancer drugs. This shows that there have been far fewer joint studies between academic researchers and the pharmaceutical sector in recent years. They then analysed 20 years of approved breast cancer medication in the US. They examined how many tumour samples were collected during clinical trials, how much biomarker research followed from this, and whether this led to a refinement of the target group for the drug. The conclusion: none of the biomarker research on the samples led to a narrower scope of application for the drug.
With our research, we want to highlight that commercial interests alone should not determine what happens to tumour samples and clinical data
prof. dr. Hans Wildiers
The researchers argue that academic researchers should also have access to this valuable data and samples to fully exploit their scientific and clinical potential.
Smaller market share
When breast cancer patients participate in a clinical trial, they often provide their clinical data and tumour samples to the company conducting the trial. These data are a treasure trove of information for additional scientific research into biomarkers. Such a biomarker can show whether or not a patient will benefit from new cancer drugs such as immunotherapy. But academic doctors and researchers are getting little opportunity to look for those biomarkers, because often only the pharmaceutical company conducting the clinical trial has direct access to the clinical data and samples.
Molecular research could demonstrate which smaller subgroups of patients really benefit from the drug, but this could reduce the market share of the new cancer drug and thus be financially disadvantageous for the pharmaceutical company.
Specific recommendations
In the article the researchers also formulate concrete recommendations. For example, they are requesting changes to the informed consent forms for clinical trials, so that patients can explicitly give their consent for academic research. They advocate for shared ownership of tumour samples, joint research agendas and an active role for patient organisations. Collaboration is essential to accelerate biomarker research, enable targeted use of medication, avoid unnecessary toxicity and protect the affordability of cancer care.
Prof. dr. Hans Wildiers, breast cancer specialist at UZ Leuven and first author of the study: “Both pharmaceutical companies and academic researchers aim to find better treatments for cancer patients. But the priorities of companies and doctors may be conflicting. The pharmaceutical industry tries to find treatments for large groups of patients, for financial reasons. As academic researchers, we want to define as much as possible smaller groups of patients: groups with certain tumour characteristics that clearly benefit greatly from a new targeted cancer therapy. As long as there are no good biomarkers, we have to use new cancer drugs for all patients in the study, while we know that often only 5 to 10 percent of patients benefit from it. Such medication can cause a lot of side effects and cost society a lot of money. With our research, we want to highlight that commercial interests alone should not determine what happens to tumour samples and clinical data.”
A lot of patients don’t realise that their doctors do not have access to their tumour samples or data from clinical studies
prof. dr. Christine Desmedt
Prof. dr. Christine Desmedt, head of the Laboratory for Translational Breast Cancer Research at KU Leuven and senior author of the study: “Of course we want patients to continue participating in clinical trials: these ensure that their chance of survival or recovery increases. But a lot of patients don’t realise that their doctors do not have access to their tumour samples or data from clinical studies. We need to make this more transparent and organise it better. We know that not only breast cancer researchers, but all oncology researchers worldwide are facing this problem. Our study is a first step towards putting the issues on the table and exploring constructive collaborations.”
The study has recently been published in JAMA Oncology and is the result of a unique collaboration between breast cancer specialists, researchers, legal professionals and representatives of international patient organisations from more than 40 countries. The recommendations will soon be discussed on international forums with representatives from the European Medicines Agency (EMA) and the pharmaceutical industry.