Use of real-world evidence for oncology clinical decision making in emerging economies.

Real-world evidence (RWE) can provide insights into patient profiles, disease detection, treatment choice, dosing strategies, treatment sequencing, adverse event management and financial toxicity associated with oncology treatment. However, the full potential of RWE is untapped in emerging economies due to structural and behavioral factors. Structural barriers include lack of regulatory engagement, real-world data availability, quality and integrity. Behavioral barriers include entrenched healthcare professional behaviors that impede rapid RWE understanding and adoption. These barriers can be addressed with close collaboration of healthcare stakeholders; of whom, regulators need to be at the forefront given their ability to facilitate use of RWE in healthcare policy and legislation.

Lay abstract Traditionally, randomized clinical trials have been used to provide insights on new medical therapies and continue to remain the gold standard for approval. The-increasing availability of patient level data in the real-world, it is now possible to generate evidence regarding the usage and potential benefits or risks of a medical therapy derived from analysis of real-world data. This evidence is collectively referred to real-world evidence (RWE). randomized clinical trials and RWE are complementary and the area of Oncology especially benefits from RWE to guide clinical decision making across the patient journey. Key benefits include cancer screening and diagnosis, optimal treatment choices (including personalized medicine) and disease management such as dosing and treatment of side effects. In recent times, RWE generation in oncology has been prolific in the USA and western Europe. With expansive biopharmaceutical investments into infrastructure harnessing patient-level data and greater local regulatory guidance, oncology patients in emerging economies may now also have the opportunity to benefit from clinical decision making informed by RWE.

The Impact of a Forced Non-Medical Switch of Inhaled Respiratory Medication Among Patients with Asthma or Chronic Obstructive Pulmonary Disease: A Patient Survey on Experience with Switch, Therapy Satisfaction, and Disease Control.

PURPOSE: Budesonide/formoterol pressurized metered-dose inhaler (pMDI) was removed from a Medicare Part D formulary, and patients switched to fluticasone-based dry powder inhaler (DPI) therapies. This study describes the experience, satisfaction, and disease control among patients with asthma or chronic obstructive pulmonary disease (COPD) who switched due to removal from the formulary. PATIENTS AND METHODS: A patient survey was conducted among adults with asthma or COPD who used budesonide/formoterol pMDI for ≥3 months prior to the formulary block and the new medication for ≥3 weeks after switching, recruited by providers in a research panel. Survey comprised both validated instruments (PASAPQ, OEQ, ACQ-6, and CAT) and stand-alone questions. Patient characteristics, switch experience, device and treatment satisfaction, onset of effect, and disease control were compared between disease (asthma and COPD) and medication (once and twice daily) cohorts. Minimal significance for group differences: P≤0.05. RESULTS: Among 100 patients, 93% received communication from their doctor or nurse about the switch and 73% received training on using the new inhaler. Patients used their new treatment for an average of 7 months prior to completing the survey. Patient satisfaction with the new therapy was high (PASAPQ; mean overall satisfaction: 6.2 for asthma; 6.0 for COPD; P=0.338). However, asthma was not well controlled (ACQ-6) in 62% of patients with asthma, and 56% of patients with COPD reported high/very high impact of their illness on their lives (CAT). Sixty-eight percent and 70% of patients with asthma and COPD, respectively, required reliever medication (≥3 puffs) most days during the week prior to the survey. There were no significant differences in disease control (ACQ-6, CAT) between once-daily and twice-daily treatments (P>0.05 for both asthma and COPD). CONCLUSION: Even when reporting satisfaction with their new medication, objective measures showed substantial morbidity, regardless of DPI device or dosing regimen.