Caregiver-Reported Economic Impacts of Pediatric Rare Diseases-A Scoping Review.

: Rare diseases are conditions that are individually rare but collectively common. These diseases can incur significant direct and indirect costs with a combination of high medical expenses, loss of income, and additional non-medical costs. Despite this, research into the economic cost for families of children with a rare disease is lacking.

Interventions to support young adults and families with the healthcare transition between paediatric and adult nephrology health services: A systematic scoping review.

Background: Healthcare transition can be challenging for young people and families living with chronic kidney conditions, including those with rare renal disorders who often have multi-systemic conditions, those who have undergone kidney transplantation, and those who face intense treatments like dialysis. Comprehensive, holistic healthcare transition interventions are required, encompassing physical, psychosocial, sexual, educational and vocational support.

Aim: This manuscript presents a systematic scoping review synthesising the healthcare transition interventions to support youth and families within nephrology services.

Antibiotic Development: Lessons from the Past and Future Opportunities.

The challenge of antimicrobial resistance is broadly appreciated by the clinical and scientific communities. To assess progress in the development of medical countermeasures to combat bacterial infections, we deployed information gleaned from clinical trials conducted from 2000 to 2021. Whereas private sector interest in cancer grew dramatically over this period, activity to combat bacterial infections remained stagnant.

2023 in review: FDA approvals of new medicines.

An analysis of all new entities approved by both the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) identified the approval of 69 new entities in the year 2023, 50 % more than in the previous year. Oncology drugs tied with congenital and infectious diseases drugs for the most approvals. Although orphan and priority approvals continued at a high pace, the rate of fast-track approvals continued to decline.

WANTED DEAD OR ALIVE: New Thinking to Incentivize Drug Development.

Despite unprecedented advancements in our understanding of disease process and targeting opportunities, our continued ability to discover and develop new medicines is fundamentally in doubt. These challenges reflect a growing recognition of the escalating risks of drug development (in terms of dollars and time) by established pharmaceutical companies and investors. Such hurdles are surmountable and require new thinking.