Real-World Data in healthcare decision-making in rare neurological diseases

Rubrik: Pharma-Markt

(Treffer aus pharmind, Nr. 05, Seite 437 (2023))

Leunikava I | Sendersky V | Barnes A | Rolph H | Union K | Chakraborty S

Real-World Data in healthcare decision-making in rare neurological diseases / Leunikava et al. • Healthcare decision-making · Leunikava I¹, Sendersky V², Barnes A³, Rolph H⁴, Union K², Chakraborty S² · ¹UCB Pharma, Monheim (Germany), ²UCB Pharma, Brussels (Belgium), ³Weave Research Ltd, London (UK), ⁴Helen Rolph Research Ltd., Surrey (UK) und
RCTs are labelled as the “gold standard” tool to test health technologies as they are conducted under experimental controlled conditions and on homogeneous populations to minimize potential for bias. However, despite their strengths, RCTs have substantial limitations. They are very costly, and although they may take years to complete, they may not be able to assess long-term effects or to detect long-term adverse events. The evidence generated in RCTs does not show the full value of new health technologies with all possible real-world benefits for patients and their caregivers (aspects they are going to experience the most may stay undetected) ...