Non interventional drug studies in oncology: Why we need them?

Abstract

Oncology is a highly researched therapeutic area with an ever expanding armamentarium of drugs entering the market. It is unique in how the heterogeneity of tumor, patient and treatment factors is critical in determining outcomes of interventions. When it comes to decision making in the clinic, the practicing physician often seeks answers in populations with obvious deviations from the ideal selected populations included in the pivotal phase III randomized controlled trials (RCTs). While the randomized nature of the RCT ensures its high internal validity by removing bias, their ‘controlled’ nature casts a doubt on their generalizability to the real world population. It is for this reason that trials done in a naturalistic setting post the marketing authorization of a drug are increasingly required. This article discusses the importance of non interventional drug studies in oncology as an important tool in testing the external validity of controlled trial results and its value in generation of new hypothesis. It also discusses the limitations of such studies while outlining the steps in their effective conduct.

Herbal Medicines, a Prominent Component in Complementary and Alternative Medicine Use in Gastrointestinal Field

“…Herbs and supplements group was the most common CAM modality in this study, a common fi nding which is seen in similar studies ( 2,3 ). We have recently conducted a research on 160 patients suff ering from functional dyspepsia.…”

“…Th is point becomes extremely remarkable when we review high prevalence of counterfeit medicaments distribution especially for those regions without precise regulations and supervision for herbal remedies. On may remain unidentifi ed and unrecognized in the restrictive design of a controlled trial ( 3 ). We believe that this new analysis allowed us to reduce the infl uence of the described biases, showing that azathioprine and/or anti-TNFα therapy delayed progression of phenotype in patients with Crohn's disease.…”

Medical Science Liaisons in Real-World Evidence Studies: Experience of AstraZeneca Russia

“…PREVENT study (ClinicalTrials.Gov ID: NCT02155998) was targeted at the population of adult male patients with locally advanced prostate cancer with high and very high risk of recurrence who had undergone surgery or radiotherapy within 3 months prior to enrollment, consented to participate in this non-interventional study, and who were being treated for prostate cancer in the oncology institutions/departments in Russian Federation. Observational studies in oncology are generally recognized as problematic, 8 and on top of this, inclusion criteria in this particular study were quite tough (required prostate cancer risk stratification). As a result, we faced delayed recruitment and the urgent need to undertake additional steps.…”

A retrospective study of trifluridine/tipiracil in pretreated metastatic colorectal cancer patients in clinical practice

“…Compassionate use is important for patients, to give them another chance; it is also important for physicians in order to obtain useful and fit data for clinical practice beyond obvious deviations from the ideal selected populations included in randomized-controlled trials [23]. Based on our single-center experience, we reported the efficacy and the safety results for a consecutive cohort of 85 chemorefractory, heavily pretreated mCRC patients treated with trifluridine/tipiracil in compassionate use in our institution.…”