Drugs

Rare Disease Drug Development Articles

Researchers have published two articles on rare disease drug development in the Journal of Biopharmaceutical Statistics. The first discusses the role of regulatory flexibility in the review and approval process of rare disease drug development. It notes that this issue was recently studied by a National Academy of Sciences, Engineering, and Medicine consensus committee that found that regulatory flexibility plays an important role in the review and approval process of rare disease drug development.

The article says that in general, appropriate regulatory flexibility should be applied under these principles:

they do not change the current process and maintain the same regulatory requirements and standards when possible;
they can address practical challenges that are commonly encountered in drug development without undermining the integrity, quality, and scientific validity of the intended trials;
they will maximize statistical/scientific flexibility; and
they cannot only be applied to rare disease drug development, but also to drug products with normal conditions.

The second article, on using alternative and confirmatory data in support of rare disease drug development, provides an overview of the limitations and major challenges of using alternative and confirmatory data that are commonly encountered in rare disease drug product development. It proposes a novel two-stage hybrid adaptive trial design combining a randomized controlled clinical trial and a real-world study.

The report lists these advantages for such a two-stage study: it does not change the current practice; it maintains the same standard as drug products with normal conditions; it provides a new concept of sample size; it takes care of the issue of no concurrent control group while maintaining scientific validity; it can address inter-patient and intra-patient variabilities; it provides an opportunity for the development of a composite index; it fully uses alternative confirmatory data/evidence, including relevant supplemental data in support of regulatory submissions; the innovative thinking and approaches can be extended to evaluate both safety and efficacy simultaneously; regulatory flexibility maximizes statistical/scientific flexibility; and the novel design and analysis methodologies can also be applied to drug products for common conditions.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome