Abstract

Cancer become epidemic, it caused 9,5millions deaths in 2018 and the disease incidence yearly increase. Anticancer drugs costs have been increasing and its alarming worldwide healthcare systems, so evidence-based medicine in this area impact directly on financial aspects, patient access and best healthcare practice. Lately Worldwide Health Agencies such as FDA, EMA and ANVISA (Brazilian Health Agency) have made flexible requirements for drug approval. Phase 2 and Non-randomized studies can be used as single reference to new drug approval, and mainly in oncology, usually those trials use surrogate outcomes as the primary endpoints, such as progressive-free survival (PFS) and objective response rate (ORR). Those surrogate endpoints have major impact on evidence-based assessment, they are not patient-important outcomes. Patients have to be active on their healthcare decisions, and those outcomes are barriers to shared decision making. Moreover, outcomes based on PFS can be biased due to many causes: First, there are no standard for measurement (commonly use radiological parameters); Second, progression is subjective, and the measurement can be different among researchers; Third, frequency of assessment can influence the results and the last, there is no relationship that the progression control can generate benefit to patient. In oncology, the majority of papers published use those surrogate endpoints and when we focus on new technologies approved by the FDA, it is more significant. How do the physicians deal critically with this study limitation? How physicians’ critical point of view would be influenced by pharmaceutical industry marketing (always attending in their office and medical conferences)? On cancer and rare diseases treatment, there is also the patients’ association pressure regarding new technologies. Patients with advanced cancer are facing death and they want fast and effective solutions for their health problems. This society pressure for ‘unmet medical needs’ are in many times used as argument for approval of drugs with phase 2 trials or even non-randomized studies, considered still experimental. New technologies access is important, and we face an EBM Manifesto critique, the clinical trials cost and duration. Clinical trials and real-world data could be associated to generate information about efficiency and safety of new drugs. In my opinion, the insertion of real-world data in the process of post market is important and could be mandatory to pharmaceutical industry, the public presentation of those data would be essential (as it was stated in AllTrails initiative) and if the rule was not fulfilled, it could result, for example, in a big fine or the drug registry suspension. Currently, when a new drug enters in the Market, patients and sometimes also physicians don’t know the risks and uncertainty regarding those new treatment choices, in many cases this drug haven’t yet confirmatory data or they are based on non-randomized studies. Patients should be clearly informed, I suggest patient education about that uncertainty of using some new medicines and also training for physicians on shared decision making. Transparency is the key for good healthcare practices based in evidence.