(2 of 4) Transitioning from Clinical Research to FDA Review: Clinical Data Application Assembly Team

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Are you planning, or currently in, the process of building your submission clinical data package for a New Drug Application (NDA) or Biologics License Application (BLA)? It can be a complicated process, and in this blog series, we are discussing some of the most important considerations to keep in mind while preparing for FDA submission of clinical data and analytics. Our last post covered the process of preparing the application deliverables to showcase all of the safety and efficacy evidence on your new biologic or pharmaceutical. This time, we are looking specifically at the team members you will need to assemble to ensure the process is smooth and flawless.

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(1 of 4) Transitioning from Clinical Research to FDA Review: Preparing Submission Deliverables

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The process of developing new pharmaceuticals and biologics is long-drawn, pricey, and heavily regulated. After drug discovery and years of pre-clinical and clinical research, a regulatory authority, such as the U.S. Food and Drug Administration (FDA), must review all animal and human data regarding a product’s safety, efficacy, and biological mechanisms. Successfully achieving market approval requires not only a safe and effectual therapy but also accurately presenting all of the evidence to prove it works and is safe for use. In this blog series, we will be reviewing some of the most important components necessary for filing a New Drug Application (NDA) or a Biologics License Application (BLA): the deliverables, the people, the timeline, and the budget. Today, let’s start with the deliverables.

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Is Enrichment Beneficial for ALS Research? We Say “Yes!”

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Earlier this year, the FDA released updated guidelines on the types and use of study population enrichment methods. They believe the use of enrichment can potentially provide increased efficiency in the clinical trial process. Our experts at Princeton Pharmatech have proven these benefits, at least in the development of Edaravone, a product approved in 2017 for treatment of amyotrophic lateral sclerosis (ALS). Read more

Six Factors that Determine if a REMS is Needed

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Yesterday, April 4th, 2019, the FDA issued new industry guidance for Risk Evaluation and Mitigation Strategy (REMS). This final guidance clarifies how the FDA applies six factors in determining whether a REMS is required for a particular drug and what type of REMS might be necessary (i.e. what specific elements or tools should be included as part of the REMS) to ensure that the benefits of a drug outweigh its risks. If the drug has been shown to be effective but is associated with a specific serious risk, REMS might be required to manage and mitigate a specific risk. Read more

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

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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