“Expect More – Not The Biggest, Just The Best”
Several times a year we are asked what Target Health does and what are our accomplishments. The following summarizes what has happened over the last 12 months. We would like to thank the loyalty and feedback of our over 2,600 readers, some of whom have been receiving ON TARGET since 1995, when we started as a weekly Fax.
We are very pleased to announce that there are now over 20 products with FDA marketing approval including 14 products (NDA-3; PMA-9; BLA-1; and 510k-1) that used Target e*CRF for their pivotal trials. There is one NDA in dermatology and one PMA in review which we expect approval in 2009. An MAA in Womens Health was submitted in May 2008. Of the 25 drug products that were approved by FDA in 2008, two used Target e*CRF for their Phase II and Phase III trials.
In 2008, Target Health celebrated its 15th year as a New York City-based, full-service e*CRO with full-time staff dedicated to all aspects of Drug and Device Regulatory Affairs, Clinical Research, Biostatistics, Data Management, Internet-based clinical trials (Target e*CRF), Medical Writing, and Strategic Planning. We also provide fully validated software for clinical trials.
A. Regulatory Affairs
1. Regulatory Representation
Represent over 30 companies at FDA from all over the world including Australia, England, France, Germany, Israel, Korea, Switzerland and the US.
2. NDA Degarelix (Ferring Pharmaceuticals)
December 2008: Ferring Pharmaceuticals received both FDA and EMEA approvals for the marketing of its prostate cancer treatment, degarelix, a new GnRH receptor blocker intended for patients in whom androgen deprivation is warranted. Target e*CRF was used for all Phase 2 and Phase 3 studies. In addition to degarelix, Target Health has collaborated with Ferring on 3 marketed NDAs for products sold in the US, Canada and Europe. Two of these programs also used Target e*CRF.
3. BLA Arcalyst (Regeneron Pharmaceuticals)
February 2008: Regeneron Pharmaceuticals, received FDA marketing approval for Arcalyst (rilonacept) Injection for Subcutaneous Use, an interleukin-1 blocker, for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 and older. Target e*CRF was used for all Phase 2 and Phase 3 studies.
4. 510(k) PD2i Analyzer Vicor Technologies Inc.
December 2008: The Vicor PD2i Analyzer is a device for measuring heart rate variability (HRV). The device can use any ECG acquisition System that can digitize raw signals, in order to perform proprietary calculations from HRV and then display the results utilizing a display unit. The objective of the PD2i Algorithm is to make the PD2i Correlation Integrals (CIs) from the input data, and then find the accepted and convergent slopes of the linear scaling regions for each embedding dimension (i.e., the PD2i values).
5. FDA Inspection of Target Health
In May 2008, FDA completed a 4 day, unannounced though not unexpected, inspection of Target Health for an NDA under review. For this program Target Health performed all of the clinical and regulatory strategic planning, toxicology, regulatory, study designs, monitoring of the clinical trials (Phase 1, 2 and 3), data management, including Target e*CRF (EDC), statistics, medical writing and preparation of the NDA. There were 2 pivotal trials and a rescue protocol for treatment failures. The FDA audit also included a detailed review of Target e*CRF and data management. The outcome of the inspection was that from evaluation of the establishment inspection report and the documents submitted with that report, we conclude that you adhered to the applicable statutory requirements and FDA regulations governing the monitoring practices of clinical investigations and the protection of human subjects.
6. FDA Approvables 2008
PMA – SyntheMed, Inc.: SyntheMed received a recommendation for approval from the Circulatory System Devices Advisory Panel of the FDA of REPEL-CV Adhesion Barrier for use in pediatric patients (21 and younger) who are likely to need secondary open heart surgery. Target e*CRF was used for the pivotal trial and Target Health submitted the PMA as an eCopy, monitored the study and performed statistical analyses and medical writing.
NDA – Summers Laboratories Inc.: Target e*CRF was used for the Phase 2 and 3 trials for a product to treat head lice. Target Health represented the company with FDA, monitored the studies, performed statistical analyses and medical writing and prepared the NDA, which was submitted as an eCTD. Target Health was inspected by FDA with respect to this program and did not receive a Form FDA 483.
7. NDA Submission
Digestive Care Inc. submitted an NDA for Pancrecarb for the treatment of digestive systems associated with pancreatic insufficiency. Target Health prepared all submission data sets for the studies and the ISS, wrote the ISS and ISE and wrote most of the study reports submitted as part of the NDA.
8. INDs / CTAs
a. Cushings syndrome
c. Gaucher disease (Canada)
d. Ulcerative colitis (n=2)
9. Orphan Drug
a. Scleroderma (US and EU)
10. FDA Meetings
a. Adhesion prevention
b. Cushings syndrome
c. Emergency contraception
d. Gaucher disease
e. Hereditary angioedema
f. Herpes simplex
g. Hormone replacement therapy
h. Ulcerative colitis
i. Uterine myoma
11. Other Major Approvals Prior to 2008
a. 8 PMAs approved in the diagnostic area which used Target e*CRF for data management. “PMAs submitted within 30 days of LPLV. Flawless FDA BIMO (Bioresearch Monitoring) audits.”
b. PMA approved in the area of periodontal disease for a combination drug/device. Target e*CRF was used for the pivotal trial and Target Health monitored the study and performed statistical analyses and medical writing
12. Electronic Submissions
Target Health has expertise in preparation and publishing of electronic submissions and is an FDA approved vendor for electronic submissions through the Electronic Submissions Gateway (ESG).
B. Clinical Trial Software Packages
Target e*CRF: Target e*CRF has now been used in over 160 clinical trial since 1999. Largest trial to date is over 7,000 patients. There are now 14 Approved Products in the US that used Target e*CRF for their clinical trial programs (3-NDAs, 1-BLA, 9-PMAs and 1-510(k)). In the US, there are currently 1 NDA and 1 PMA under review, both of which have been designated “Approvable.” In Europe, there is 1 MAA under review. This coming year, an NDA will be submitted in the area of Women’s Health as well as an NDA for an Orphan Metabolic Disease. For the latter project, Target Health will also be preparing and submitting the eCTD.
Target Document: Target Document is a USER-FRIENDLY, INEXPENSIVE; HIGHLY SOPHISTICATED, Web-based, document management system that allows authorized users to view, download, and manage any document for their organization. – No More paper – Target Document features include: 1) 21 CFR Part 11 compliance; 2) routing for electronic signatures; 3) email alerts; 5) communication tools.
Target Encoder: Target Encoder is a USER-FRIENDLY, INEXPENSIVE; HIGHLY SOPHISTICATED, Web-based, coding system that allows authorized users to automatically code MedDRA and WHO Drug and other types of dictionaries. Target Encoder is fully integrated with Target e*CRF.
Target e*CTMS: Target e*CTMS is a USER-FRIENDLY, INEXPENSIVE; HIGHLY SOPHISTICATED, Web-based, clinical trial management system. A new clinical trial starts with identification of the sponsor and project name. Investigators, IRBs and users are maintained within the CTMS and can be easily assigned to a project. All staff within a clinical site can be identified with their title and contact information, as well as shipping addresses which could be different from the head office. As the site commits to participate in the clinical trial, a site number can be assigned. Once IRB approval is obtained, and all regulatory documents have been identified as received, an alert can be sent out via email to allow for drug shipment. Target e*CTMS provides many additional features such as: 1) Decision Logs, 2) Meeting Logs with uploading of the meeting minutes, 3) Questions and Answers, 4) status of Regulatory Submissions and Deliverables, and 5) Monitor Site Visit Tracking with document upload.
Target Batch Edit Checks: With Target e*CRF, batch edit checks are now integrated with the electronic query system within the study. Target e*CRF runs the edits and displays the results of those edits through a discrepancy review screen integrated with the query system.
Target e*Pharmacovigilance: Target e*CRF integrates EDC with a pharmacovigilance module by 1) allowing the principle investigators to enter a narrative, 2) allowing the medical monitor to enter a narrative and then have the EDC system generate an approved version of Form 3500A or CIOMS for regulatory submission with the ability to control the original and followup submissions.
Target eClinical Trial Record (Target e*CTR): Target e*CTR allows the clinical study sites to perform direct data entry into any EDC system, and at the same time generates a read-only electronic document, which can be designated as the primary source data (eSource). These data, maintained in a secure, read-only trusted 3rd party environment, are available to the clinical study sites, monitors and regulatory agencies in a human readable format.
C. Academic Collaborations
EDC vendor for 2 NIH grants in Juvenile Rheumatoid Arthritis at the Cleveland Clinic and University of Washington. EDC project completed in the newborn under an HHS grant at Stanford University. Collaboration with the Biotechnology Center at SUNY Stony Brook and UMDNJ (the Medical School of New Jersey).
D. Current Indications
1. Abstract: A Complex Protocol Design To Accelerate Clinical Trial: How To Implement Multiple Database Lock In An Electronic Data Capture System (Bio-IT World, 2008).
2. Mitchel J., Moncrieffe A., Kim Y.J., et al. The Paperless Trial, Past, Present and Future. European Pharmaceutical Contractor, Autumn 2007.
3. Mitchel J., Ghilezan I., Kim, Y.J., et al., The End of the Paper Trail, European Pharmaceutical Contractor Winter 2008; 72-75.
4. Mitchel J, Park G, Lynch S., Pagano R. and Citron M. Phase 1 Clinical Trials and Exploratory Phase 2 Clinical Trials. Book Chapter in Principles in Clinical Oral Health Research, Edited by: William Giannobile (University of Michigan Clinical Center), Brian Burt and Robert Genco (State University of New York at Buffalo) 2009 In Press.
5. Dye BA and Mitchel J. Data Management in Oral Health Research. Book Chapter in Principles in Clinical Oral Health Research, Edited by: William Giannobile (University of Michigan Clinical Center), Brian Burt and Robert Genco (State University of New York at Buffalo) 2009 In Press.
F. Contact Information
Target Health Inc.
261 Madison Avenue, 24th Floor
New York, NY 10016
Phone: (212) 681-2100; Fax (212) 681-2105
Dr. Jules T. Mitchel, President; Ms Joyce Hays, CEO
2008 Target Health Inc. All rights reserved