Regulatory agency consideration of pharmacogenomics

Experimental Biology and Medicine

Volumn 233, Issue 12, 2008, Pages 1498-1503

  Pendergast, Mary K ^a  

^a Pendergast Consulting   (United States)

Author keywords

Federal regulation; Food and drug administration; Pharmacogenomics

Indexed keywords

ANALYTIC METHOD; CASE STUDY; CONFERENCE PAPER; DRUG RESEARCH; FOOD AND DRUG ADMINISTRATION; GENETIC ANALYSIS; GENETIC VARIABILITY; GOVERNMENT REGULATION; HUMAN; LABORATORY TEST; PHARMACOGENOMICS; UNITED STATES; VALIDITY; CLINICAL PHARMACOLOGY; CLINICAL TRIAL; DRUG APPROVAL; DRUG INDUSTRY; ETHICS; GENETIC SCREENING; GENETICS; PHARMACOGENETICS; PROFESSIONAL STANDARD; REVIEW;

DRUG;

CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; DRUG INDUSTRY; ETHICAL REVIEW; ETHICS COMMITTEES, RESEARCH; GENETIC RESEARCH; GENETIC SCREENING; GOVERNMENT REGULATION; HUMANS; PHARMACEUTICAL PREPARATIONS; PHARMACOGENETICS; PHARMACOLOGY, CLINICAL; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 58749091151     PISSN: 15353702     EISSN: 15353699     Source Type: Journal    
DOI: 10.3181/0806-S-207     Document Type: Conference Paper

References (21)

1. U.S. Food and Drug Administration. Pharmacogenomics and its role in drug safety. FDA Drug Safety Newsletter. Winter, 2008. Available at: http://www.fda.gov/cder/dsn/2008-winter/pharmacogenomics.htm.
2. U.S. Food and Drug Administration. Draft drug-diagnostic co-development concept paper. April 8, 2005. Available at: http://www.fda.gov/cder/genomics/ pharmacoconceptfn.pdf. FDA opened Docket No. 2004N-0279 to receive comments on this topic. The FDA sometimes collapses the clinical validity and clinical utility questions into one construct, which they call ''clinical test validation.''
3. The CDRH's Office of In Vitro Diagnostics has extensive regulatory information on its website. U.S. Food and Drug Administration. Office of In Vitro Diagnostics. 2008. http://www.fda.gov/cdrh/oivd/officeinfo.html. All medical devices are classified into one of three classes: I, II, and III. Class I devices have only general controls such as proper labeling and good manufacturing practices. Class II devices receive pre-market clearance based on scientific studies, and they are sometimes required to meet pre-specified regulatory requirements. Class III devices receive pre-market approval based on the most extensive scientific data and studies.
4. CMS was petitioned to create a genetic testing specialty under CLIA and to establish standards for proficiency testing, but rejected the petition on August 15, 2007. A copy of the denial letter can be found at http://www.dnapolicy.org/resources/CMSresponse8.15.07.pdf.
5. Woodcock J, Lesko LJ. Pharmacogenomic-guided drug development: regulatory perspective. Pharmacogenomics 2:20-24, 2002.
6. In May 2002, FDA proposed a new regulatory process, called the ''safe harbor'' through which companies could voluntarily submit exploratory pharmacogenomic data to FDA. The report of the May 16-17, 2002 workshop was published. Lesko LJ, Salerno RA, Spear BB, Anderson DC, Anderson T, Brazell C, Collins J, Dorner A, Essayan D, Gomez-Mancilla B, Hackett J, Huang S, Ide S, Killinger J, Leighton J, Mansfield E, Meyer R, Ryan S, Schmith V, Shaw P, Sistare F, Watson M, Worobec A. Pharmacogenetics and pharmacogenomics in drug development and regulatory decision making: Report of the first FDA-PWG-PhRMA-DruSafe Workshop. J Clin Pharmacol 43:342-358, 2003.
7. U.S. Food and Drug Administration. Draft guidance for industry: pharmacogenomic data submissions. November 3, 2003. Available at: http://www.fda.gov/OHRMS/DOCKETS/98fr/2003d-0497-gdl0001.pdf. The opportunity to view the draft guidance and to comment on it was published in the Federal Register. Federal Register 68:62461, 2003. The docket created for comments on the draft guidance was Docket No. 2003D-0497.
8. The report of that workshop, which focused on the more practical aspects of voluntary genomic submissions and their review, and on the types of data that would be required not voluntary was published. Salerno RA, Lesko LJ, Pharmacogenomics in drug development and regulatory decision-making: the Genomic Data Submission (GDS) proposal. Pharmacogenetics 5(1):25-30, 2004.
9. Notice: guidance for industry on pharmacogenomic data submissions. Federal Register 70:14698, 2005. The final guidance can be found at http://www.fda.gov/cder/guidance/6400fnl.pdf. Pharmacogenomic data submissions, companion guidance, August 2007. The guidance can be found at http://www.fda.gov/cder/guidance/7735dft.pdf.
10. See http://www.fda.gov/cder/genomics. On April 7, 2008, FDA posted a new guidance document, Guidance for industry: E15 definitions for genomic biomarkers, pharmacogenomics, pharmacogenetics, genomic data and sample coding categories. This guidance arises out of the activities of the International Conference on Harmonization, which is a joint effort of the United States, Japan, and the European Union. Available at http://www.fda.gov/cber/gdlns/ iche15term.pdf.
11. Lesko LJ. Personalized medicine: regulatory perspective. January 8, 2008. Slides ofaspeechavailable at: http://www.fda.gov/cder/genomics/presentations/ Lesko-PCAST-Jan08.pdf.
12. U.S. Food and Drug Administration. Draft drug-diagnostic co-development concept paper. April 8, 2005. Available at: http://www.fda.gov/cder/genomics/ pharmacoconceptfn.pdf. FDA opened Docket No. 2004N-0279 to receive comments on this topic.
13. The FDA's first effort to regulate IVDMIAs was in a ''Draft guidance for industry, clinical laboratories, and FDA staff: in vitro diagnostic multivariate index assays'' released on September 7, 2008. FDA opened a public docket to receive comments on the draft guidance. Docket No. 2006D-0347. After reviewing comments and holding a public meeting, FDA published a second draft guidance with the same name on July 26, 2007. The second draft can be accessed at http://www.fda.gov/cdrh/oivd/guidance/1610.pdf. FDA has not finalized the guidance.
14. U.S. Food and Drug Administration. FDA news: FDA clears breast cancer specific molecular prognostic test. February 6, 2007. Available at: http://www.fda.gov/bbs/topics/NEWS/2007/NEW01555.html. Agendia submitted data to FDA to validate this intended use. The studies determined that the test was ''useful in predicting time to distant metastasis in women who are under age 61 and in the two earliest stages of the disease (Stage I and Stage II) and who have tumor size equal to or less than five centimeters and no evidence that the cancer has spread to nearby lymph nodes (lymph node negative).''
15. 21 CFR 866.6040.
16. The special controls for this type of device are in FDA's guidance document entitled ''Class II Special Controls Guidance Document: Gene Expression Profiling Test System for Breast Cancer Prognosis.'' U.S. Food and Drug Administration. Class II Special Controls Guidance Document: Gene expression profiling test system for breast cancer prognosis. May 9, 2007. Available at: http://www.fda.gov/cdrh/oivd/guidance/1627.html.
17. Clinical laboratories that are uncertain of the regulatory requirements for a medical device can meet with the FDA for a ''pre-IDE'' meeting. Office of In Vitro Diagnostic Device Evaluation and Safety. Overview of IVD regulation. February 3, 2005. Available at: http://www.fda.gov/cdrh/oivd/regulatory- overview.html.
18. U.S. Food and Drug Administration. Table of valid genomic biomarkers in the context of approved drug labels. April 7, 2008. Available at: http://www.fda.gov/cder/genomics/genomic-biomarkers-table.htm.
19. Katasanis SH, Javitt G, Hudson K. Public health: a case study of personalized medicine. Science 320:53-54, 2008. The authors do not explain whether the tests were performed using the approved Roche kit or were performed as LDTs.
20. Genetics & Public Policy Center. Marketing of unproven tests a threat to public health. 2008. Available at: http://www.dnapolicy.org/news.release. php?action=detail&pressrelease-id=94&print=1.
21. Berg AO et al. Recommendations from the EGAPP Working Group: testing for cytochrome P45 polymorphisms in adults with nonpsychotic depression treated with selective serotonin reuptake inhibitors. Genet Med 9(12):819-825, 2007.