Results from Expanded Access Programs: A Review of Academic Literature.

BACKGROUND: Although expanded access is an increasingly used pathway for patients to access investigational medicine, little is known on the magnitude and content of published scientific research collected via expanded access. METHODS: We performed a review of all peer-reviewed expanded access publications between January 1, 2000 and January 1, 2022. We analyzed the publications for drugs, diseases, disease area, patient numbers, time, geographical location, subject, and research methodology (single center/multicenter, international/national, prospective/retrospective). We additionally analyzed endpoints reported in all COVID-19-related expanded access publications. RESULTS: We screened 3810 articles and included 1231, describing 523 drugs for 354 diseases for 507,481 patients. The number of publications significantly increased over time ([Formula: see text]). Large geographical disparities existed as Europe and the Americas accounted for 87.4% of all publications, whereas Africa only accounted for 0.6%. Oncology and hematology accounted for 53% of all publications. Twenty-nine percent of all expanded access patients (N = 197,187) reported on in 2020 and 2021 were treated in the context of COVID-19. CONCLUSIONS: By summarizing characteristics of patients, diseases, and research methods described in all scientific literature published on expanded access, we provide a unique dataset for future research. We show that published scientific research on expanded access has surged over the past decades, partly due to COVID-19. However, international collaboration and equity in geographic access remain an issue of concern. Lastly, we stress the need for harmonization of research legislation and guidance on the value of expanded access data within real-world data frameworks to improve equity in patient access and streamline future expanded access research.

How Do Shifts in Patients with Mental Health Problems' Formal and Informal Care Utilization Affect Informal Caregivers?: A COVID-19 Case Study.

(1) Background: This study investigated how potential shifts in patients' formal and informal care utilization during the COVID-19 pandemic impacted their informal caregivers in terms of their subjective burden, psychological wellbeing, and happiness. (2) Methods: A retrospective cohort study design was employed for a panel of Dutch informal caregivers of persons with mental health problems (n = 219) in June 2020. Descriptive statistics and differences between means were determined for the patients' informal and care utilization and informal caregivers' subjective burden, happiness, and psychological wellbeing. Three mediation analyses were conducted using the PROCESS macro. (3) Results: Informal caregivers reported significantly worse happiness and subjective burden scores during the COVID-19 pandemic compared with before the lockdown. There were minimal shifts in patient's care utilization reported, with the exception of a decrease in significant emotional and practical support provided by the informal caregiver. In the mediation analyses, there was not a significant indirect effect of shifts in patients' formal care utilization on informal caregivers' subjective burden, psychological wellbeing, and happiness through shifts in patients' informal care utilization. (4) Discussion and conclusion: Whilst we found that shifts in patients' care utilization during the first wave of the pandemic did not affect the informal caregiver in the short term, it is unclear what the long-term impact of the pandemic might be on informal caregivers. More research should be conducted to understand the implications of short- and long-term impact of substitution on informal caregivers of persons with mental health problems, with special consideration of the COVID-19 context and uptake of e-health technology.

[Fewer cancer diagnoses during the COVID-19 epidemic according to diagnosis, age and region]. / Onderdiagnostiek bij kanker door de COVID-19-crisis naar diagnose, leeftijd en provincie: Een cruciale rol voor eerstelijnszorgverleners.

The COVID-19 crisis and the intelligent lockdown have led to a situation in which the Dutch health care system was locked for months for non-COVID-19 patients. Patients did not dare to go to their general practitioner, general practitioners were reticent about hospital referrals, and diagnostic and treatment trajectories were delayed or adapted. This also concerned cancer patients. The severity of the underdiagnosis for these patients mainly depends on their prognosis. In this study, we evaluated the magnitude of underdiagnosis using data from the Netherlands Cancer Registry and the Dutch registry of histo- and cytopathology (PALGA). From the week of the first COVID-19 diagnosis in the Netherlands, a decrease of 20-40% in the number of cancer diagnosis was noticed. This decrease was observed in nearly all cancer types, including cancers such as lung cancer (average 23%), head-neck cancer (average 36%) and hematology (average 26%), for which a missed or delayed diagnosis can be life threatening. Therefore, we want more attention for the observed underdiagnosis and the role of the primary care givers, including the general practitioner and dentist. Furthermore, it is of crucial importance that patients with symptoms or complaints feel no hesitation to visit their caregivers.