Publications by authors named "J Matthias von der Schulenburg"
Article Synopsis
- Health technology assessment (HTA) for pharmaceuticals, especially rare disease treatments, involves complexities and uncertainties due to faster approval processes.
- Real-world evidence (RWE) is being proposed to support HTA decisions, but its collection and application come with significant methodological challenges.
- The paper emphasizes the importance of rigorous study design and analysis in using RWE, and offers suggestions to enhance its effectiveness in HTA, highlighting its growing role in determining coverage and pricing.
Objective: No consensus exists on the ideal methodology to evaluate the economic impact and value of new, potentially curative gene therapies. We aimed to identify and describe published methodologic recommendations for the economic evaluation of gene therapies and assess whether these recommendations have been applied in published evaluations.
Methods: This study was conducted in three stages: a systematic literature review of methodologic recommendations for economic evaluation of gene therapies; an assessment of the appropriateness of recommendations; and a review to assess the degree to which the recommendations were applied in published evaluations.
Background: Strokes cause an estimated annual health care burden of 170 billion euros across Europe. Atrial fibrillation is one of the major risk factors for stroke and increases the individual risk 4.2-fold.
View Article and Find Full Text PDFIntroduction: Until recently, adjuvant treatment options for higher stage resectable cutaneous melanoma were limited. Two studies with a similar set-up, published 2017, led to registration of targeted therapy for BRAF-mutated melanoma with dabrafenib and trametinib as well as of the immunotherapy with nivolumab irrespective of BRAF-mutation status. Both options have been positively assessed in Germany since 2019 for the adjuvant treatment of BRAF-V600 mutated melanoma.
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