In completing Writing Assignment students will have the opportunity to:
- Demonstrate an understanding of the basic principles of what encompasses safety science with respect to drug/biologics development
- Demonstrate an ability to extract and interpret safety-relevant information form the publicly available FDA Drug Approval Package Information
- Demonstrate an ability to describe/analyze safety information for an approved drug/biologic/device
For this assignment, you are a Sr Manager of Regulatory Affairs at a small, emerging biotechnology company that is looking to develop a new, inhaled treatment for Parkinson’s Disease. Your company is very small and inexperienced and so far, none of the nonclinical safety testing or clinical testing of this new drug has begun. As your team members prepare to start nonclinical safety testing and then clinical testing of the drug, it is your task to provide your colleagues with a summary of how nonclinical and clinical data is used to inform the development of safety information about your new Parkinson’s disease drug. You also need to provide your colleagues with background information on the regulatory aspects of safety monitoring, using the safety guidance provided by FDA and ICH.
During your research to learn more about drugs that treat Parkinson’s, you discover that there is already a marketed product for Parkinson’s that is also administered by inhaler. The already approved drug is called Inbrija (also known as CVT-301 – the research code name). This is great news! You can use this similar, already-approved drug as a comparison for your investigational inhaled therapeutic for Parkinson’s.
There is a lot of information your company can learn about how to safely develop your planned novel Parkinson’s therapeutic by studying the FDA’s published NDA approval information located on the FDA’s database of approved products: (Links to an external site.). Specifically, you can learn a lot about the safety information that was included in the NDA for Inbrija by reviewing information contained in the following FDA Application review Files:
Your task is to provide the following information to your colleagues (incorporation of figures and graphs HIGHLY encouraged):
- Part 1: Background information:
- Overview of Safety Basics: A description of the basic principles of generating safety information about drugs/biologics from nonclinical and clinical studies that are relevant to a drug’s development. You may wish to include a description of why safety monitoring is a critical component of nonclinical and clinical development of a drug so that your colleagues fully understand the importance.
- Regulatory information: Describe for your colleagues the different ICH and FDA guidance documents that are available to help you navigate the regulatory requirements with respect to safety monitoring from the nonclinical and clinical development perspectives. The ICH’s M3(R2) (Links to an external site.) is a useful tool that you can describe for your colleagues to help them understand nonclinical safety requirements that must be completed prior to and during clinical testing of the drug. You can also navigate to the FDA’s Safety Reporting requirements for INDs (Links to an external site.) and describe some of the requirements for clinical safety monitoring.
- Part II: Pulling out relevant examples from the Inbrija FDA approval package
- Nonclinical safety information: Using the “Pharmacology Review” file for Inbrija’s NDA approval package, describe what kinds of safety pharmacology and general toxicology studies were run. You can describe the number of studies in each nonclinical safety category as well as what species they were run in and the basic results/findings of these studies. You are encouraged to include tables and figures from the Pharmacology Review in your written summary, just be sure to include citations.
- Clinical safety information: Using the “Medical Review” file, describe some of the safety information that was generated as part of the clinical trials that were implemented when Inbrija was being developed. You should include a brief list/description of all the clinical trials that were run.
- Part III: Conclusions: This section should be at least one paragraph and should work to summarize next steps for your company with respect to developing the nonclinical and clinical safety program for your novel Parkinson’s disease therapeutic.
Expert Solution Preview
Introduction: In this assignment, we will be discussing how nonclinical and clinical data is used to develop safety information about a new drug for Parkinson’s disease. The task is to provide a summary of the safety monitoring process, including the regulatory aspects of safety monitoring, by using the safety guidance provided by FDA and ICH. Furthermore, we will use the approved drug Inbrija as a comparison for our investigational inhaled therapeutic for Parkinson’s disease.
Part 1: Background Information:
Nonclinical and clinical studies are two critical components of drug development that generate safety information relevant to a drug’s development. Safety monitoring is vital during the development stage to ensure the safety and effectiveness of the drug. The US Food and Drug Administration (FDA) and ICH have provided regulatory guidance to assist drug companies in monitoring the safety of the drugs. For nonclinical testing, ICH’s M3(R2) guidance can be used. For clinical safety monitoring, FDA’s Safety Reporting Requirements for INDs is a useful tool.
Part II: Pulling out relevant examples from the Inbrija FDA approval package
Nonclinical safety information: As per the Pharmacology Review file for Inbrija’s NDA approval package, several safety pharmacology and general toxicology studies were conducted. These studies were performed on different species of rodents and dogs, and the findings indicated that the drug was safe for clinical testing.
Clinical safety information: As per the Medical Review file, there were five clinical trials for Inbrija, and all were phase 3 randomized, double-blind, placebo-controlled studies that analyzed the efficacy, safety, and tolerability of the drug when compared with the placebo.
Part III: Conclusions
In conclusion, our small and emerging biotech company will have to go through nonclinical safety testing and clinical trials to ensure the safety and efficacy of the new investigational inhaled therapeutic for Parkinson’s disease. We can use the ICH’s M3(R2) and FDA’s Safety Reporting Requirements for INDs guidance documents to ensure regulatory compliance during the safety monitoring process. We can also refer to the information provided for Inbrija during its approval process as a useful resource for our nonclinical and clinical safety program.