Rare disease clinical trials : challenges and best practices / Giuseppe Remuzzi.

Format:

Video

Author/Creator:

Remuzzi, Giuseppe, author.

Language:

English

Subjects (All):

Rare diseases.

Rare diseases--Handbooks, manuals, etc.

Physical Description:

1 videorecording (59 min.)

Other Title:

Rare disease clinical trials

Place of Publication:

London : Henry Stewart Talks, 2024.

System Details:

video file

Contents:

Rare diseases constitute a heterogeneous group of over 6,000 disorders. In the United States, a rare disease is defined by a prevalence of less than 200,000 cases. Although 6-8% of the world population has a rare disease, interventional research in these diseases is limited and sometimes of poor quality. Recruiting and retaining participants, particularly when diseases are progressive, degenerative, or life threatening and making robust inferences from studies when recruitment is constrained can be challenging. In this webinar, Professor Remuzzi will discuss limitations, validity, and applicability of innovative designs that must be taken into consideration and may enable researchers to address questions that are unlikely to be answered with conventional designs. When research participants are limited, researchers should consider using: 1 a crossover design; 2 n-of-1 trials; or 3 one of the following adaptive designs: a a response-adaptive randomization design, b a ranking and selection design, c an internal pilot design, or d a sequential design. Bayesian analysis may be applied to conventional designs, or to any of these uncommon designs. Several of these approaches may also be used in combination. The choice between methods should be guided by factors related to the intervention, disease, anticipated recruitment duration and success, and current state of knowledge about the treatment.

Notes:

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