Use the template provided and choose one of the arguments below to write a Citizen’s Petition based on your Journal Club article: GENE THERAPY
Assignment Question
Background: One of the ways by which industry and the general public (i.e., any interested person) can influence the commercial/regulatory framework is via Citizen’s Petitions. Citizen’s Petitions are requests that are formally submitted to the FDA Commissioner to ask the Commissioner to establish, amend, or revoke a regulation or order or take or refrain from taking any other form of administrative action. Essentially, one is making an appeal (with some statement of grounds/rationale) to request that the FDA take a certain action/uphold a certain position on an issue. If interested, you can read a bit more about Citizen’s Petitions here: https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=10.3Links to an external site. and https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?FR=10.30Links to an external site.
Your assignment: Use the template provided and choose one of the arguments below to write a Citizen’s Petition based on your Journal Club article: GENE THERAPY. See below with directions Make a case for why the FDA’s expedited programs for regenerative medicines (from the reading assignment: “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions: Guidance for Industry”) are adequately designed and good for promoting the development of cell, gene, or tissue engineering products (choose according to the subject of your journal club article). Utilize the safety and/or efficacy data presented in your Journal Club article to support your case. OR Make a case for why the FDA’s expedited programs for regenerative medicines (per the reading reading assignment: “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions: Guidance for Industry”) are inadequate to facilitate the FDA’s thorough review and regulation of cell, gene, or tissue engineering products and/or may lead to the development of unsafe/ineffective cell, gene, or tissue engineering products (choose according to the subject of your journal club article). Utilize the safety and/or efficacy data presented in your Journal Club article to support your case.
NOTE: You will need to request a specific action from the FDA – which will require some thought and creativity on your part. You can be as specific as you like with the topic of your petition, as long as it fits within the context of one of the points above. For example, you could request that FDA grant (if it is a case that wouldn’t otherwise qualify)/deny (if it may qualify and you believe it isn’t safe/wise for the pathway to be available to it) regenerative medicine advanced therapy (RMAT) designation to the product/products like the one you covered.
You could also seek a policy change – perhaps a pathway for more in-depth review/even more abbreviated review due to the complexity/simplicity of product. If you are unsure about your chosen request for the petition, I will be glad to review a draft statement of your requested action and give you some feedback – provided that you email me your question >1 week before the due date. You may also find it helpful to refer to other sources or additional course materials (e.g., some of the scientific rationale, explanations of therapeutic promise offered by different regenerative medicines, and/or or critiques of the FDA’s review process) in formulating a convincing argument. To successfully complete this assignment you will expected to: Draft a petition with all required sections (per the template provided) Clearly articulate a requested action that is in line with the scope of the assignment Construct a sound case to support the requested action, which: Incorporates the journal club topic Is logically written and relevant to the requested action Format, grammar, and overall presentation are also graded aspects of this assignment.
Remember, the FDA Commissioner is the intended audience; write this as if the Commissioner were actually reading it. The assignment should be submitted as a well-written document with clearly expressed points, typed in Microsoft Word using 11-point Arial font. You can just edit the text within the provided template. However, if you choose to amend the template format, be sure that your assignment is adequately spaced, spell checked, and maintains one-inch margins. This assignment can reasonably be completed within 5 pages but I will accept up to a total length of 15 pages – remember: a lengthy assignment does not necessarily equate to a quality assignment; extra information will not earn you extra points (in fact, it may cause you to lose points)!
Note: Be sure to complete this assignment in line with the Code of Conduct and applicable Ethics policies. All references must be documented accordingly.
