Fewer than half of new drugs have data on their comparative benefits and harms against
existing treatment options at the time of regulatory approval in Europe and the USA. Even …

Synthetic antisense oligonucleotides (ASOs) and duplex RNAs (dsRNAs) are an
increasingly successful strategy for drug development. After a slow start, the pace of success …

Replacing RCTs with real world data for regulatory decision making: a self-fulfilling prophecy? |
The BMJ Skip to main content Intended for healthcare professionals Access provided by Google …

Background Bevacizumab—a humanized monoclonal antibody—has been widely used to
treat patients with hereditary hemorrhagic telangiectasia (HHT), but no randomized trial has …

RNA sequencing has emerged as a promising approach in cancer prognosis as sequencing
data becomes more easily and affordably accessible. However, it remains challenging to …

The RASopathies are a group of clinically defined developmental syndromes caused by
germline variants of the RAS/mitogen‐activated protein (MAPK) cascade. The prototypic …

Abstract Spinocerebellar ataxia type 3 (SCA3), also known as Machado–Joseph disease
(MJD), is a neurodegenerative disorder caused by a polyglutamine expansion in the ATXN3 …

Background To assess uncertainty in regulatory decision-making for orphan medicinal
products (OMP), a summary of the current basis for approval is required; a systematic …

While current guidelines generally recommend single endpoints for primary analyses of
confirmatory clinical trials, it is recognized that certain settings require inference on multiple …

Recently licensed cell and gene therapies have promising but highly uncertain clinical
benefits. They are entering the market at very high prices, with the latest entrants costing …