Getafix Pharmaceuticals is a small start-up pharmaceutical company developing a compound it hopes to market as soon as possible for treating a very rare form of pediatric brain cancer. You are working in the Regulatory Affairs Department and the CEO brings you into his office to advise the leadership team on what it would take to get that compound to market.
PART I: What would you advise the Getafix Pharmaceuticals Leadership Team at that first meeting? How can the Regulatory Affairs team be most helpful here? Are there any special considerations?
PART II: With nonclinical studies completed, planned, and ongoing, what advice would you give Getafix Pharmaceuticals R&D Leadership in order to best place them for IND submission?
PART III: Getafix Pharmaceuticals is now planning to submit an initial IND submission to FDA. Outline operational and strategic considerations Getafix should take into account when preparing to submit its initial IND to the FDA. Indicate whether you would recommend a Pre-IND meeting with FDA, and if so, describe the contents of required meeting material and potential goals of the meeting. Describe documentation required for initial IND submission.
PART IV: Getafix Pharmaceuticals has maintained an effective IND and and is ready for an NDA. Outline operational and strategic considerations Getafix should take into account throughout NDA preparation. Within your plan, provide a brief introduction describing the purpose of the NDA and its review by the FDA. Describe documentation required for initial NDA submission and outline post-approval activities Getafix would need to perform to keep the drug on the market in the United States. Also, consider the possibility of an Advisory Committee Meeting and outline steps needed to prepare for this during NDA preparation.
Part V: Congratulations! The SolveIT drug was approved by the FDA due to your assistance, and has been on the market for 3 years, with profits rising over $100Million, soaring GetaFix Stock. Unfortunately, GetaFix has recently received a warning letter citing failure to report anaphylactic reactions to SolveIT at a high rate, at a recent medical conference – cell phone evidence caught the head of Medical Affairs discussing off-label uses of the drug, and a recent promotional advertisement violated fair balance requirements. What are the requirements of these violations and how would you recommend Getafix respond to this warning letter? What are the impacts of warning letters on drug companies?
Each answer should be answered in more than 2 paragraphs, references should be at end of each question.