FDA Issues New Guidance for Rare Diseases

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Rare Diseases: Natural History Studies for Drug Development Guidance

On March 22nd, the FDA issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development Guidance for Industry. A natural history study describes the course of a rare disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes. Natural history studies can be prospective or retrospective. Read more

What is Network Meta-Analysis (NMA)?

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These comparisons create a web-like analysis called a Network Diagram or Network Comparison. Other names of NMA include Multiple Treatment Meta-Analysis, Mixed Treatments Comparison, Indirect Treatment Comparison, Pair-Wise Meta-Analysis and so forth. NMA has been widely used in technology appraisals for various clinical indications by technology assessment agencies around the world. For instance, in January 2019, the FDA performed a fixed-effect network meta-analysis to investigate risk of MACE associated with Romosozumab treatment. Read more

The Sentinel System for Industrial Use: Strengths and Weaknesses

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Currently, the Sentinel Distributed Database (SDD) is comprised largely of administrative and claims data from health insurers. These large administrative claims databases have become increasingly popular sources of data for comparative safety research (observational studies) because they have several important strengths including a large size, longitudinal data on prescription drug use, generalizability, and low operational costs. Read more

Using the Sentinel System in FDA Submissions

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Recently, FDA has been using RWD from Sentinel System (SS) in drug approval process quite often to evaluate some safety signals in the postmarket setting and also for other purpose.

For past three advisory committee meetings hold in January 2019, SS has been applied in two approval processes (Jan 11 and Jan 17 , 2019) and one (Jan 16, 2019) in proposing potential possibility to use SS database in post-marketing safety study. Read more

What is the FDA’s Sentinel System (SS)?

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Starting with a mini-sentinel pilot program, the Sentinel initiative began in 2008 as a multi-year effort to create a national electronic system for monitoring the performance of FDA-regulated medical products. Working with public, academic, and private entities, the FDA developed the sentinel system and launched in 2016. The Sentinel System obtains information from existing electronic healthcare data from multiple sources to assess the safety of approved medical products. The efforts of integrating the Sentinel System into the FDA’s regulatory programs enables the administration to evaluate some safety signals in the postmarket setting more effectively by using Sentinel’s automated tools. Read more

Ethical and Practical Problems With Double-Blind Control Studies

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Placebos are a staple of double-blind, randomized control studies. This is because they help to reduce study bias. When treating malignant hematologic and oncologic disease, however, there are ethical and practical problems that arise. Read more

Emerging Endpoints: Minimal Residual Disease

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Another emerging endpoint is minimal residual disease. This benchmark has emerged because even those in complete remission are known to experience relapses, which is true even when the patient has been in remission for significant periods of time. Emerging technology can now identify malignancies at levels orders of magnitude lower than what was previously conceivable. Read more

Emerging Endpoints: Metastasis-Free Survival

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Nonmetastatic, castration-resistant prostate cancer is a frequently recurrent form of prostate cancer. The treatment-resistant nature means continuous androgen-deprivation therapy and a resultant decrease in the quality of life of the patient. It also makes for a prolonged assessment period for treatments, which makes the overall survival rate an impractical endpoint. Thus, nonmetastatic, castration-resistant prostate cancer is a textbook example of the need for non-traditional endpoints such as metastasis-free survival. Read more

Ethics and Practice in Experimental Childhood Treatment

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Pediatric cancer is not simply adult cancer in a younger body. Thus, there are particular practical and ethical concerns when it comes to treating adolescents, as well as testing those treatments. In many cases, adolescents are not even eligible for such treatments. Read more

New Breakthroughs Create New Cancer Research Endpoints

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In December 2018 the FDA released new guidelines for clinical trials of cancer drugs and biologics. Such endpoints serve different purposes at different points in time during the trial. During early trials the focus is on efficacy and safety. Later on, trials select endpoints for clinical benefits like improved survival and symptom relief.

For cancer patients, the survival rate is considered the optimum endpoint all other things being equal. Tumor assessment and symptom assessment are two other common endpoint categories.

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