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Observational studies are patient-centered


Patient centricity is the process of designing a service around the patient. For us, it means focusing a study on patients and patient needs. Only in the last few years, with the creation of PCORI (Patient-Centered Outcomes Research Institute) in 2010, pharma companies started having a patient-centric vision more than a product-centric one. Nowadays, there is an increasing emphasis on the need for patient centricity that require the adoption of new approaches to designing and conducting clinical studies (randomized or observational), with the aim of improving recruitment, retention and compliance.

The age-old problem facing researchers conducting trials is how to attract, engage and retain patient volunteers to participate continuously throughout all phases of a drug trial. This problem doesn't appear to stem from lack of interest or motivation to join drug trials, but from a paucity of information, lack of involvement in reporting outcomes, and restricted access to data generated from drug trials. Another pressing issue in the research and development of new drugs is the ever-increasing cost of development, which is reflected in the higher cost of medicines when they are authorized for sale. Furthermore many patients are unaware of the existence of clinical trials that they can be involved in.

One solution is for pharmaceutical companies and contract research organizations (CROs) to communicate regularly with Patient Advocacy Groups that are engaged to review materials relating to clinical trials. They scrutinize the information to ensure that it is relevant to the patient and that the terminology used is easy to understand. It is important to ask patients to provide qualitative data about their experiences with taking the drug. This is despite patients being in the best position to describe how a certain drug affects them and impacts their quality of life. In other words, patient involvement allows cross-validation of the quantitative results with qualitative, subjective data.

In this context, which is the best way to achieve patient-centered outcomes? Randomized controlled trials (RCTs) are the “gold standard” for evaluating efficacy and safety of new drugs, but observational studies better outline patients’ experiences. In the aim of answering this question, we have chosen a review article to examine the relative merits of RCTs and observational studies in the setting of chronic myeloid leukemia (CML), particularly chronic phase chronic myeloid leukemia (CP-CML), a disease in which life expectancy is expected to approximate that of patients without CP-CML. This is being made possible through the development of small-molecule tyrosine kinase inhibitors (TKIs) in the past decade. This molecule has transformed the CP-CML in a chronic illness and consequently clinical management of this disease is changing. While data on safety, toxicity, adherence and QoL are largely derived from randomized controlled trials (RCTs), other more patient-centered outcomes, such as health-related quality of life (QoL), adverse event (AE) profiles and treatment adherence, are not revealed by this kind of study, but they are becoming increasingly important. This review suggests that integration between clinical trials and observational studies could be a further step to foster patient centricity and rise benefits for all stakeholders, patients first of all.

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