Miguel Bronchud: The importance of well-designed Randomized Controlled Trials
Miguel Bronchud, Co-Founder of Regenerative Medicine Solutions, shared a post on LinkedIn:
“I am not the only ‘veteran cancer doctor’ to have stressed over many years the importance of well-designed Randomized Controlled Trials (RCTs). I did so over three decades ago, when the advent of recombinant human growth factors like G-CSF (Filgrastim) and peripheral blood stem cells (CD34+) allowed us ‘to push conventional cytotoxic chemotherapy to the limits’ to check if more intensive treatments would allow us more effective curative treatments – in spite of obvious but manageable increases in side effects.
Our efforts then and patients resilience proved positive in several randomized clinical trials in blood malignancies – but unfortunately not effective enough in most solid tumors. I was also among the first to defend the new cancer research paradigm- looking for molecular cancer drivers could lead to the development of new more effective cancer treatments.
Now there are many more ongoing challenges in our field, including the fragmentation of targeted therapies often called ‘personalized treatments’, high associated treatment costs , or the need to normalize conversations with patients or healthcare providers regarding the cost effective added or lack of sufficient value of treatments , often with significant but small gains, or to again support well-designed Randomized Controlled Trials (RCTs), with reasonable survival endpoints and clinical follow up. Without forgetting the inequalities of access to new expensive treatments. There is nothing wrong with trying to improve communication with patients, or to promote critical thinking in oncology. At present 85% of trials are funded by industry, but many oncologists and government officials responsible for funding science say it cannot be done any other way, because only pharmaceutical companies have the large amounts of money necessary. We cannot leave the responsibility of allowing or facilitating the administration of new drugs only to regulatory bodies, or to political-technical agencies and evaluation committees not always open to clinical scrutiny. Regulators have a very difficult job because they are trying to balance a number of competing priorities. There is a constant tension between approving treatments quickly, so that they reach patients as soon as possible, but also ensuring that they are good treatments. And we cannot ignore that prevention and early detection remains a more rational approach than treatment of advanced disease. And we should not forget that new imaging or molecular diagnostic tests including genomics can add to costs, for example: What is the real impact of pan-tumor Next-generation sequencing (NGS) panel testing on clinical decision making for patients with advanced solid tumors?