Regulatory Process for Biological

Anonymous
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Question Description

I need 5 pages paper about regulatory process for biological, I have attached the general question and how to write the paper, but the specific question ( Scenario) is this:

You are a primary reviewer in CBER at the U.S FDA. A biotech company is submitting an application to license a viral-vector hemophilia therapy. They are using an adeno-associated virus vector expressing the factor VIII gene. The vector transduces cells and persists as episomes within the cell. Clinical trials have been performed to demonstrate and efficacy of the approach in Europe and in the United States.

Also, I have attached some important questions that the answers should be written in the paper and an example, so you can take a look. Also, the reference should be the same as in the example.

Regulatory Process for Biological
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Regulatory Process for Biological
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Example Assignment Paper Rebecca Sheets BIOL 583 2018 Introduction/Scenario: I am a representative of an applicant; my organization is part of a complex public-private partnership to develop a global HIV/AIDS vaccine. Funding has been granted by our organization to an HIV/AIDS vaccine researcher in China who has developed a DNA plasmid prime-viral vector boost system. He has taken this to Phase 2 in trials in China using vaccines manufactured in China. He is working with a clinical trials network that has sites throughout the U.S. and in some other countries. This network is also funded by my organization. Results of a clinical trial performed by another network demonstrated modest efficacy of a viral-vector prime-protein boost. [1,2] Because of those results, the clinical trials network we fund and with which the Chinese researcher is working wants to add a protein boost to his regimen. The challenge is that the protein is being manufactured in the U.S., so the applicant will be the U.S. partner: my organization. We will seek authorization to import the protein into China so that the Chinese researcher may conduct a Phase 1 clinical trial of this 3-component candidate vaccine regimen. The protein will also be studied in clinical trials in the U.S. alone or as a component in other candidate regimens. An initial Phase 1 firstin-human trial of the protein alone will be conducted in the U.S. and a pre-IND meeting with the U.S. FDA was held to guide that. This paper focuses on the process in China to gain authorization to conduct a Phase 1 clinical trial there using the Chinese researcher’s prime-boost regimen with the US protein. This would be the initial first-in-human trial of this complete combination of 3 components combined in a single regimen, although there will be clinical experience with each component separately and 2 of the 3 components combined. Possibility for a meeting between the Chinese regulators and the applicant Based on publicly available information in English, e.g., the Chinese 1 Food and Drug Administration’s (CFDA) website, it is not clear whether there is a possibility for a meeting between the Chinese regulators and our partnership (funding organization and applicant, clinical trials network, Chinese researcher). [3] However, we were able to obtain a meeting with the CFDA prior to filing a submission to request authorization to conduct clinical trials in China with a vaccine candidate produced in the U.S. to be imported to the Chinese clinical trial site. We refer to this as a pre-IND meeting, although it isn’t technically called that in China. We were not able to obtain a similar meeting with the Chinese national control lab, the National Institute for Food and Drug Control (NIFDC). Nonetheless, the CFDA did convey expectations that the NIFDC would expect to receive specimens for testing and lot release control prior to CFDA authorizing the clinical trial. [see 4 for requirements for authorization] 1 Known in China as the State FDA or SFDA 1 Type of Dossier We will be submitting a request for clinical trial approval following the Application and Approval Procedure for Clinical Trials. [5] The Chinese refer to this as Drug Registration: Biologics Register: Preventive biological products. The requirements come from Board Order No. 17 for Drug Registration. [personal communication from Shao Y] Outline of Dossier The Chinese requirements, specified in Board Order No. 17 [personal communication from Shao Y] are for submission of document as follows: 1. Summary of documents. (1) Name of the new product; (2) Documents of proof; (3) The purpose and basis of the topic; (4) Product description in details, drafting notes and references; (5) Packaging and label design. 2. Summarize the overall findings and evaluation data. 3. Research data on bacteria (viruses) used for production. (1) Source of bacteria (viruses), characteristics and identification information (2) Establishment and testing of seed lot (3) Bacteria (viruses) passages stability data (4) Testing results of the seed lot from National Institutes for Food and Drug Control 4. Research data on cell bank used for manufacturing. (1) Source, properties and identification of cell line (2) Establishment and testing of cell bank (3) Passage stability of cell bank (4) Testing results of the working cell bank from National Institutes for Food and Drug Control (5) Source of the culture medium, added ingredients, and quality standards. 5. Manufacturing processes. (1) Vaccine production data, the theoretical and experimental basis and testing data (2) Reagent composition and formulation and testing data, source and quality standards of excipients / adjuvants. 6. Quality control data, pre-clinical efficacy and safety data (1) Quality control data and registration standards (2) Testing methods and data (3) Comparative study data with similar products (4) Data of antigenicity, immunogenicity and protection testing in animals (5) Data on allergy testing in animals (6) Data on animal safety testing. 7. Manufacturing and draft testing procedures, with drafting instructions and the relevant literature. 2 8. Manufacturing and testing data of samples submitted for clinical trial testing (by CFDA). 9. Preliminary stability testing data. 10. Compliance certification on production, research and animal testing. 11. Clinical trial program, research programs and informed consent draft. Special Program This scenario does not have a special program. However, CFDA has verbally assured that products for HIV/AIDS would be handled in exceptional manners given the public health urgency and lack of existing preventive vaccines for this disease. Pediatric Studies The CFDA does not have (to my knowledge or in English on their website) specific requirements for pediatric studies like those required in the US by PREA or in EMA by the Pediatric Medicine directive. However, adolescents are at high risk of acquiring HIV as they become sexually active. In China, one large population of HIV-positive individuals are those that use injection drugs. Injection drug use can be initiated in childhood, as well as in adulthood. Pregnant women who are not diagnosed with HIV and thus, not afforded access to therapeutics that can prevent mother-to-child-transmission of HIV, might give birth to babies who acquire HIV before or during birth or while breast-feeding after birth. For these reasons, it would be appropriate to perform pediatric studies at some point during the development of HIV/AIDS vaccines. This matter needs to be discussed with regulators in many countries, including China, as development advances. However, given the high risk of failure to prevent HIV acquisition of HIV vaccine candidates, pediatric studies are not yet justified. In this instance, it is too early to have such discussions with CFDA, as clinical trials have yet to be initiated with the combination regimen. Guidances and Regulations Among the many guidances and regulations that are applicable, including all (or virtually all) of China’s Drug laws, the following list highlights some relevant to this case: • • • • Drug Registration (Board Order Number 17); Attachment 3: Biologics Register data classification and reporting requirements; Section 2. Preventive biological products [personal communication with Shao Y] Examination & Approval for Clinical Trials of Imported (incl. from Hong Kong, Macao, and Taiwan) preventive biological products dated 2013-12-06 [4] Special Review and Approval Procedure for Drug Registration by the State Food and Drug Administration [6] SFDA Order No. 28: Provisions for Drug Registration [7] 3 In addition, CFDA follows ICH guidelines, WHO guidelines and recommendations, and would apply their requirements/regulations for HepB vaccine to this product as this is only currently existing recombinant vaccine marketed in China that is sufficiently similar and could serve as a model for requirements, even though it would be quite an inexact model. Other relevant requirements would be those for Good Clinical Practices, Good Manufacturing Practices, and Good Laboratory Practices. Bibliography or Reference List [1] Rerks-Ngarm S, Pittisuttithum P., Nitayaphan S., Kaewkungwal J., Chiu J., Paris R. et al. Vaccination with ALVAC and AIDSVAX to prevent HIV-1 infection in Thailand. 2009 NEJM 361(23):2209-20. [2] Haynes BF, Gilbert PB, McElrath MJ, Zolla-Pazner S, Tomaras GD, et al. Immune-Correlates Analysis of an HIV-1 Vaccine Efficacy Trial. 2012 NEJM 366(14):1275-86. [3] Chinese Food and Drug Administration website in English: http://eng.sfda.gov.cn (accessed 2/1/2018) [4] CFDA document: Examination and approval for clinical trials of imported (incl. from Hong Kong, Macao, and Tiawan) preventive biological products dated 2013-12-06: http://eng.sfda.gov.cn/WS03/CL0769/98156.html; http://eng.sfda.gov.cn/WS03/CL0769/98150.html (accessed 2/1/2018) [5] CFDA: Application and Approval Procedure for Clinical Trials: http://eng.sfda.gov.cn/WS03/CL0769/61659.html (accessed 2/1/2018) [6] CFDA document: SFDA Decree No. 21 Special Review and Approval Procedure for Drug Registration of the State Food and Drug Administration: http://eng.sfda.gov.cn/WS03/CL0768/61646.html (accessed 2/1/2018) [7] CFDA document: SFDA Order No. 28: Provisions for Drug Registration: http://eng.sfda.gov.cn/WS03/CL0768/61645.html (accessed 2/1/2018) 4

Tutor Answer

Perfectsolutions
School: UT Austin

Attached.

Running Head: REGULATORY PROCESS FOR BIOLOGICAL

1

Regulatory Process for Biological

Student's Name:
Institution Affiliation:

Introduction
I am a primary reviewer in, The Centre for Biologics Evaluation and Research (CBER)
one of the U.S main six centers for Food and Drug Administration (FDA) which is part of the
U.S. Department of Health and Human Resources. My organization, according to the section 351
of the public health service Act regulates the permitting of the biological products that are traded
in different states in the United States under applicable federal law protects and advances the
human health by ensuring that the products are safe, effective and available to all the citizens
who need them my organization also provides information to the public to promote the safe and
effective use of the biological products, CBER has the authority to deny licensure, suspend or
cancel if the manufacturer of the products does not comply with the requirements. A certain
biotech company is applying for a viral vector hemophilia therapy license from my organization.
The company is using an adeno-associated virus vector expressing the factor VIII gene which
transduces cells and persists within the cell. The company has performed clinical trials to
demonstrate the validity and efficiency of its gene in Europe and in the United States.
Possibility for a meeting between the U.S regulators and the applicant of the licensing
Based on the information provided by my organization FDA’s Centre for Biologics Evaluation
and Research (CBER) website, it is clear that the organization officials hold meetings,

REGULATORY PROCESS FOR BIOLOGICAL

2

conferences and various workshops about various biologics with the main aim of educating the
general public and seek the opinions from the interested members of the public. The minutes a...

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Anonymous
Excellent job

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