Diversity in Oncology Clinical Trials: Current Landscape for Industry-Sponsored Clinical Trials in Asia.

INTRODUCTION: There has been a growing recognition on the importance of diversity in clinical trials. Existing research has highlighted a significant demographic imbalance. Amidst this renewed focus on diversity, it is crucial to acknowledge that Asia comprises over half of the world's population. Given the region's demographic significance, we sought to compare various characteristics and growth rates for trials with sites in Asia against those without any sites in Asia. METHODS: We performed comprehensive analyses of industry-sponsored phase 2 and 3 oncology trials registered at Clinicaltrials.gov, using drugs or biologics as investigational agents and executed between 1 January 2018 and 31 December 2022. We applied the compound annual growth rate (CAGR) as an analytical tool to track the trial growth rates over this 5-year period. RESULTS: We identified 894 industry-sponsored phase 2 and 3 cancer studies with available study location data. Out of these, 415 trials (46.42%) had study sites in Asia. Notably, these trials with sites in Asia were also more likely to be phase 3 trials (39.76% vs 6.47%, p < 0.001), include female and paediatric populations, and be randomised trials. Interestingly, lung and stomach cancers were more commonly studied in these trials, while myeloma was less commonly studied. The number of trial sites for liver cancer was not significantly higher for Asia, even though the incidence of the disease is much higher in this region. Despite an overall declining trend in the number of clinical trials in the last 5 years, we observed a transitional positive increase in the CAGR from 2020 to 2021 for trials with sites in Asia. However, East Asia, specifically China, exhibited a disproportionate overrepresentation in these trials. CONCLUSIONS: There are notable characteristics of clinical trials with sites in Asia. Comprehending these disparities may aid in the strategic planning to enhance a balanced representation of ethnicities in trials.

Cancer incidence and mortality trends in Asia based on regions and human development index levels: an analyses from GLOBOCAN 2020.

OBJECTIVE: As Asian countries transition socially and economically to higher Human Development Index (HDI) levels, cancer trends are expected to shift to those seen in the Western World. A strong correlation also exists between HDI levels and age-standardized rates (ASR) for the incidence and mortality of cancer. However, there are very few reports on the trends in Asian countries, particularly in Low and Middle-Income Countries (LMICs). In this study, we have investigated the relationship between socioeconomic developments in Asia (determined using HDI levels of countries) and cancer incidence and mortality in these nations. METHODS: The GLOBOCAN 2020 database was used to study the cancer incidence and mortality data for all cancers combined and those most commonly diagnosed in Asia. The difference in data was analyzed based on region and HDI level. Further, the predictions for cancer incidence and mortality in 2040 according to the GLOBOCAN 2020 were analyzed using the updated HDI stratification described in the UNDP 2020 report. RESULTS: Asia has the highest cancer burden compared to the other regions worldwide. Lung cancer carries the highest cancer incidence and mortality rates in the region. Inequitable distribution of cancer incidence and mortality is seen across regions and HDI levels in Asia. CONCLUSIONS: Inequalities in cancer incidence and mortality can only be expected to increase unless innovative and cost-effective interventions are urgently implemented. An effective cancer management plan is needed in Asia, particularly in LMICs, prioritizing effective cancer prevention and control measures for health systems.

Educational needs, perception, and perspectives of oncologists regarding compassionate use programs in Asia.

OBJECTIVE: This study was conducted to capture the educational needs, perceptions, and perspectives of oncologists towards Compassionate Use Programs (CUPs) in Asia, with the aim of gathering insights related to unmet needs for physician and patient assistance. METHODS: The participants responded to a voluntary, self-administered, closed-ended questionnaire through an online platform between 29 April 2020 and 17 June 2020. RESULTS: A total of 111 oncologists provided informed consent to participate in the study. Of these, 102 respondents fully completed the questionnaire and were included in the analyses. Maximum respondents (35.3%) had 10-20 years of experience after specialization with 19.6, 23.5, and 21.6% respondents having <5, 5-10, and ≥20 years of experience, respectively. Practice type plays a statistically significant role in the awareness of the existing compassionate program (p = .0066). While many respondents seem clear on the application process for CUP set in place by pharmaceutical companies, a higher number of respondents are unclear about the country regulations and processes for applying to CUPs set in place by regulatory authorities. Most respondents (75.5%) reported that there are no resources or training provided to them regarding CUPs. There was a significant association between the clarity of the application process for CUP set in place by the sponsors and the number of applications submitted (p = .0321). CONCLUSIONS: Our study brings light on various issues faced by physicians in accessing CUPs especially related to the lack of education and training on utilizing CUPs. There are significant unmet needs related to improving the clarity for the application process, providing resources and related training, particularly for oncologists who do not have previous experience with CUPs.

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.

Real-world treatment patterns and outcomes of patients with stage IV squamous cell carcinoma of the head and neck.

AIM: To understand the treatment patterns and outcomes for stage IV squamous cell carcinoma of the head and neck, patients receiving second-line or later drug therapy. MATERIALS & METHODS: Real-world data were collected from 1152 patients in the USA, France, Germany and the UK through a retrospective chart analysis and patient-reported outcomes were collected using validated questionnaires in a subgroup of patients. RESULTS: Forty-four percent of patients had stage IVA/B disease. A total of 77, 19 and 3% of patients had received two, three and four plus lines of active drug treatment. Platinum- and cetuximab-based regimens were common at early treatment lines. Time to progression was short (5.2 months post first line), survival rates low and patient-reported health status poor. CONCLUSION: Novel therapies that could improve clinical and patient-reported outcomes would address a significant unmet need.

Continuous infusion of amphotericin B: preliminary experience at Faculdade de Medicina da Fundação ABC.

CONTEXT AND OBJECTIVE: Intravenous amphotericin B deoxycholate (AmB-D) infusions, usually given over 4 hours, frequently induce nephrotoxicity and undesirable infusion-related side effects such as rigors and chills. There is evidence in the literature that the use of AmB-D in the form of continuous 24-hour infusion is less toxic than the usual four-hour infusion of this drug. Our objective was to evaluate the efficacy and safety of continuous infusion of AmB-D for the treatment of persistent fever in neutropenic patients with hematological malignancies after chemotherapy. DESIGN AND SETTING: Observational retrospective analysis of our experience with continuous infusion of AmB-D, at Faculdade de Medicina da Fundação ABC and Hospital Estadual Mário Covas in Santo André. METHODS: From October 2003 to May 2004, 12 patients with hematological malignancies and chemotherapy-induced neutropenia received 13 cycles of continuous infusion of AmB-D. RESULTS: The median dose of AmB-D was 0.84 mg/kg/day (0.33 to 2.30 mg/kg/day). Concomitant use of nephrotoxic medications occurred in 92% of the cycles. Nephrotoxicity occurred in 30.76% of the cycles, hypokalemia in 16.67%, hepatotoxicity in 30% and adverse infusion-related events in 23%. All patients survived for at least seven days after starting continuous infusion of AmB-D, and clinical resolution occurred in 76% of the cycles. CONCLUSIONS: Continuous infusion of AmB-D can be used in our Institution as an alternative to the more toxic four-hour infusion of AmB-D and possibly also as an alternative to the more expensive liposomal formulations of the drug.

Continuous infusion of amphotericin B: preliminary experience at Faculdade de Medicina da Fundação ABC

CONTEXTO E OBJETIVO: Pacientes neutropênicos com febre persistente podem apresentar infecções fúngicas com freqüência. A administração de anfotericina B deoxicolato tem sido padrão para estes pacientes, no entanto sua infusão endovenosa, usualmente administrada em quatro horas, pode levar a nefrotoxicidade, hepatotoxicidade e efeitos adversos relacionados à infusão, como tremores e calafrios. A literatura evidencia que o uso de anfotericina B deoxicolato em infusão contínua de 24 horas pode ser menos tóxica em relação à administração usual. O objetivo do estudo foi avaliar a eficácia, segurança e toxicidade da anfotericina B infusional contínua em pacientes onco-hematológicos após quimioterapia com neutropenia febril persistente. TIPO DE ESTUDO E LOCAL: Estudo observacional e retrospectivo de nossa experiência com anfotericina B deoxicolato em infusão contínua de 24 horas, na Faculdade de Medicina da Fundação ABC e Hospital Estadual Mário Covas, em Santo André. MÉTODOS: No período entre outubro de 2003 e maio de 2004, 12 pacientes com neoplasias hematológicas e neutropenia febril induzida por quimioterapia receberam 13 ciclos de anfotericina B deoxicolato infusional. RESULTADOS: A dose média da infusão foi de 0,84 mg/kg/dia. O uso concomitante de outras drogas nefrotóxicas ocorreu em 92% dos ciclos. Foram observados nefrotoxicidade em 30,76%, hipocalemia em 16,67%, hepatotoxicidade em 30% e efeitos adversos relacionados à infusão em 23% dos ciclos. Todos os pacientes sobreviveram aos sete primeiros dias após o início do tratamento e a resolução clínica ocorreu em 76% dos ciclos. CONCLUSÃO: A infusão contínua de anfotericina B é exeqüível para uso em nossa instituição como alternativa à infusão em quatro horas (mais tóxica) e possivelmente às caras formulações lípidicas desta droga.