ABSTRACT
Improving patient care and advancing scientific discovery requires responsible sharing of research data, healthcare records, biosamples, and biomedical resources that must also respect applicable use conditions. Defining a standard to structure and manage these use conditions is a complex and challenging task. This is exemplified by a near unlimited range of asset types, a high variability of applicable conditions, and differing applications at the individual or collective level. Furthermore, the specifics and granularity required are likely to vary depending on the ultimate contexts of use. All these factors confound alignment of institutional missions, funding objectives, regulatory and technical requirements to facilitate effective sharing. The presented work highlights the complexity and diversity of the problem, reviews the current state of the art, and emphasises the need for a flexible and adaptable approach. We propose Digital Use Conditions (DUC) as a framework that addresses these needs by leveraging existing standards, striking a balance between expressiveness versus ambiguity, and considering the breadth of applicable information with their context of use.
Subject(s)
Information Dissemination , HumansABSTRACT
PURPOSE: To estimate the resource gap in the polymerase chain reaction (PCR) monitoring for patients with chronic myeloid leukemia (CML) in low- and middle-income countries (LMICs). METHODS: We developed a model of demand and supply of PCR monitoring of CML patients in 60 LMICs. PCR testing was assumed to use Cepheid's GeneXpert® IV system. We included costs of GeneXpert® instruments, uninterrupted power supplies, warranties, calibration kits, test cartridges, and shipping. We calculated the country-specific monetary gap in PCR monitoring, stratified by country priority defined as the availability of tyrosine kinase inhibitors (TKIs) through The Max Foundation initiatives. RESULTS: The 5-year gap in PCR monitoring was $29.1 million across all countries, 22% ($6.4 million) in countries with all five TKIs available, 20% ($5.7 million) in countries with four TKIs available, 50% ($14.5 million) in countries with three TKIs available, 8% ($2.2 million) in countries with two TKIs available, and 1% ($0.3 million) in countries with one TKI available. The gap was highest in South Asia (52%; $15.1 million) and lowest in Latin America (6%; $1.9 million). Excluding labor costs, the bulk of the resource needs (86%; $25.2 million) were for procurement of BCR-ABL cartridges. CONCLUSION: Removing the 5-year gap in PCR monitoring capacity for CML in LMICs will require the mobilization of significant resources and will likely lead to better treatment outcomes and reduced treatment costs through optimization of treatment, discontinuation of therapy in appropriate patients, and facilitation of clinical research. Development of streamlined monitoring guidelines for resource-limited countries should be considered.
ABSTRACT
BACKGROUND: Cancer is a major burden of disease in low- and middle-income countries (LMICs) yet financial barriers limit access to life-saving oncology drugs. Medical donation and other drug access programs can help improve patient access to essential medicines, such as quality assured oncology drugs in LMICs. However, there are no published examples of the conduct of pharmacovigilance with donated medical products intended for use in LMICs where pharmacovigilance is weak. We describe a partnership between a pharmaceutical company and a non-governmental organization as a case example that addresses the challenges in performing pharmacovigilance with donated medicines in LMICs. The Max Foundation's direct to patient model is designed to improve global access to quality assured oncology drugs through access programs such as the Glivec® (generic name: imatinib) International Patient Assistance Program (GIPAP). RESULTS: Between 2013 and 2016, in the course of managing the GIPAP program, The Max Foundation was made aware of 13,039 instances of adverse events (AEs). These AEs were reported to The Max Foundation by physicians, patients, and caregivers. The Max Foundation reported these AEs to Novartis through the AE reporting tool within its Patient Assistance Tracking System (PATS). Physicians were the reporters for 58% of the AEs while the remainder of the AEs were reported directly by patients or caregivers. The overall rate of reported AEs remained relatively steady for the years 2013 through 2016 at 92, 95, 86, and 97 AEs reported per 1000 persons who received Glivec® per year, respectively. The vast majority of adverse events (85%) were reported from countries where The Max Foundation has a MaxStation, i.e., where The Max Foundation staff interact directly with physicians and patients at clinics or over the phone. AE reporting rates were consistently higher in all years studied from countries where The Max Foundation has a MaxStation. While India accounted for the largest number of reported adverse events in 2016 (1990), Bolivia had the highest rate of reported adverse events at 484 AEs per 1000 patients. CONCLUSIONS: International patient assistance programs that provide access to medicines can have an important role in assisting pharmaceutical companies in fulfilling their pharmacovigilance obligations. Adverse event information collected through PATS can potentially contribute to the overall body of knowledge on the safety of medicinal products.
