Reference no: EM13705509

cite references appropriately giving credit where credit is due.

Question 1-
What hurdles impacted research into therapies for rare diseases leaving those suffering from these disorders with few treatment options until recent years? Describe FDAs legislative initiative to encourage sponsor companies to conduct research for rare disorders. What are the key components of this legislation? Has FDAs approach been successful?


Question 2-
A number of regulatory documents developed in support of Investigational New Drug Applications (INDs) and New Drug Application (NDAs) contain information that will be incorporated/and updated in during the regulatory approval and post-approval process. List four key elements documented within various sections of the IND and NDA that find their way into the package insert/labeling of approved drugs. List the names of the documents and/or sections of the documents where the information is initially incorporated linking these with applicable section of the required labeling contents for the approved drug.

Question 3-
List three different types of supplemental NDAs and provide a definition for each. Which supplemental NDA typically receives a higher priority than others with respect to Agency review?



Question 4-
List two initiatives undertaken by FDA to allow for expanded access to investigational drugs, one that focuses on decreasing development timelines and one that addresses review cycle. Briefly describe these initiatives.




Question 5-
Identify and describe two FDA regulatory initiatives designed to ensure proper labeling information for children. How these initiatives are similar, how are they different? Provide your assessment as to which approach you believe is more effective at achieving its goal.


Question 6-
How do The New Drug Application (NDA) and the Abbreviated New Drug Application (ANDA) differ in terms of regulatory requirements? How are they similar? Which of these applications (if any) require no clinical trials?



Question 7-
How should a sponsor address a major post-marketing manufacturing change with FDA?


Question 8-
What steps should an Sponsor organization take to meet minimum GCP requirements internally (within the Sponsor's Company) with respect to the conduct of clinical studies and externally in ensuring compliance with investigational sites participating in clinical trials. Please be specific.

Question 9-
What types of post-marketing approval activities might be required by the FDA for a sponsor who has received approval for an NDA application? What impact might the use of surrogate endpoints reported in an NDA application have on post-market approval requirements?
Question 10-
Briefly describe the timing of sponsor post-approval safety reporting requirements to FDA. FDA's focus on safety extends beyond approval and includes ensuring that consumers also have a voice in reporting issues related to safety. What direct-to-consumer effort has recently been employed by the agency to encourage adverse reaction reporting?

Question 11-
List three key labeling requirements and briefly define these requirements.


Question 12-
List five (5) required elements of an informed consent form. Under what circumstances might an informed consent form need to be revised? Explain the meaning of the statement, "Informed Consent is not a form, but a process."

Question 13-
What is a Case Report Form? What advantages does an electronic Case Report Form have over a traditional paper-based case report form? Which document typically gets developed first, the protocol or the CRF? Why?


Question 14-
Identify and briefly describe direct-To-consumer (DTC) advertisement types discussed during class. Outline the case for and against DTC advertising.