Despite a large number of people globally being affected by rare kidney diseases, research support and health care policy programs usually focus on the management of the broad spectrum of chronic kidney diseases without particular attention to rare causes that would require a targeted approach for proper cure. Hence, specific curative approaches for rare kidney diseases are scarce and these diseases are not treated optimally, with implications on the patients’ health and quality of life, on the cost for the health care system, and society. There is therefore a need for rare kidney diseases and their mechanisms to receive the appropriate scientific, political and policy attention to develop specific corrective approaches. A wide range of policies are required to address the various challenges that target care for rare kidney diseases, including the need to increase awareness, improve and accelerate diagnosis, support and implement therapeutic advances and inform the management of the diseases. In this paper, we provide specific policy recommendations to address the challenges hindering the provision of targeted care for rare kidney diseases, focusing on: awareness and prioritization; diagnosis; management; and therapeutic innovation. In combination, the recommendations provide a holistic approach aiming for all aspects of rare kidney disease care to improve health outcomes, reduce the economic impact, and deliver benefits to society. Greater commitment from all the key stakeholders is now needed, and a central role should be assigned to rare kidney disease patients to partner in the design and implementation of potential solutions.
Analytics for clinical development strategy: A unified approach to optimizing return on investment in R&D
Despite the rapid scientific advances with new targets, modalities, and biomarkers, the return on invested capital (ROIC) in novel therapeutics remains low....