ON TARGET
COMPLIMENTARY NEWS LETTER OF TARGET HEALTH® INC.
30 July 2007
I. WHAT'S NEW?
Target Health Web-Based Software
For Clinical Trials
II. QUIZ - (Fill
In The Blanks)
Stem Cells May Mend Broken Hearts [mice]
III. HISTORY OF MEDICINE
Ancient Invention Increasingly Becomes High-Tech Marvel
IV. ORPHAN DISEASE
Consequence of Treatment Interruption in Gaucher Disease
V. EPIDEMIOLOGY
Is Being
Fat a Contagious Disease?
VI. CARDIOLOGY
Defibrillators Highly Effective in Hypertrophic Cardiomyopathy
VII. REGULATORY AFFAIRS
Financial
Disclosures
VIII.
Target
Health Web-Based Software For Clinical Trials
Target e*CRF®,
EDC Made Simple™ Version 3.1, now provides the randomization schedule without
the need of Interactive Voice Response Systems (IVRS). You can also program
most edit checks without the need to be a programmer and build most forms in an
hour or 2. If it is in the Library, the time is reduced to a minute. Target
Document® comes with a Trial Master File format already built in, and Target
Encoder® accelerates MedDRA and WHO-DRUG coding. All are 21 CFR Part 11
compliant and also fulfill EU Directive standards.
For more information about
Target Health, please contact Dr.
Jules T. Mitchel.
Visit our Blog and Website.
II. QUIZ (Fill In The
Blanks)
Stem Cells May Mend Broken
Hearts [mice]
Stem cells may
help repair damaged 1) ___ after a heart attack. A study, done on mice, shows
that stem cells play a limited, but significant role in 2) ___ damaged hearts.
However, it remains unclear whether it is heart cells that are doing the
repair, or cells from elsewhere in the 3) ___. Richard Lee and colleagues of
the
ANSWERS: 1) tissue;
2) repairing; 3) body; 4) protein; 5) muscle; 6) aged; 7) heart; 8)
regeneration
III. HISTORY OF MEDICINE
Ancient Invention Increasingly Becomes High-Tech Marvel
Sandpaper, a seemingly
simple tool for smoothing out rough surfaces, is much more complex than meets
the eye and has increasingly become a high-tech marvel, according to an article
in the July 23 issue of Chemical & Engineering News. Whether honing medical
implants or shaping-up jet turbine blades, improved sanding techniques are
smoothing the way toward technological progress. First used by the Chinese as
early as the 13th century, sandpaper has evolved from an amalgam of crushed
seashells bonded to parchment paper into a highly sophisticated process. The
design of modern sandpaper, technically called a 'coasted abrasive,' involves a
complex interplay between the chemical properties of the abrasive material,
adhesive, and the backing material. Abrasives, or the gritty particles on
sandpaper, can range from natural minerals, such as garnet or emery, to
synthetic materials, such as fused aluminum oxide or silicon carbide. Choosing
an abrasive can be a tricky task, as many materials can chemically react with
the material being sanded or are too expensive for practical use. But today,
computer programs can take the guesswork out of making sandpaper by modeling
how an abrasive will perform, while the electron microscope has been used to
optimize the structure of the tiniest abrasives. With the development of
stronger materials, scientists have correspondingly developed stronger
abrasives and improved bonding agents to allow for more aggressive sanding.
From simple woodwork to high-end machining, there's no alternative to
sandpaper.
IV. ORPHAN DISEASE
Consequence of Treatment
Interruption in Gaucher Disease
According to an
article published in the Journal of Pediatrics (2007;151:197-201), a study was
performed to document the effects of interrupting enzyme replacement therapy
(ERT) for at least 1 year in a group of children with type 1 Gaucher disease.
For the study, all children with type 1 Gaucher disease who were treated at 2
pediatric centers and who were required to suspend ERT for at least 1 year were
studied before, during, and after treatment interruption. Hemoglobin and
platelet levels, organomegaly, growth, and bone manifestations were monitored.
Results showed that 5 of 32 children experienced treatment interruptions.
Before ERT, all children had splenomegaly, 4 children had hepatomegaly, 4 children
had growth retardation, 3 children had skeletal manifestations, 3 children had
thrombocytopenia, and 1 child had anemia. After 1 to 7 years of ERT, all
children were growing normally, none had skeletal manifestations, organomegaly
had decreased or disappeared, and hematologic features had improved. After 15
to 36 months of ERT interruption, splenomegaly recurred or worsened in all
children, hepatomegaly and hematologic features recurred or worsened in 4
children, serious bone manifestations developed in 4 children, and 3 children
experienced growth retardation. After at least 11 months of resumed ERT in 4
children, 2 had hepatomegaly, 2 had splenomegaly, and all had persistent
skeletal manifestations. It was concluded that interruption of ERT in children
with type 1 Gaucher disease should be avoided because it can cause recurrent
organomegaly, growth delays, and skeletal manifestations that do not resolve
after treatment reinstatement.
V. EPIDEMIOLOGY
Is Being Fat a Contagious
Disease?
The prevalence
of obesity has increased substantially over the past 30 years. As a result, a
study published in the New England Journal of Medicine (2007;357:370-379), was
performed to analyze the nature and extent of the person-to-person spread of
obesity as a possible factor contributing to the obesity epidemic. The study
evaluated a densely interconnected social network of 12,067 people assessed
repeatedly from 1971 to 2003 as part of the Framingham Heart Study. The
body-mass index (BMI) was available for all subjects. Longitudinal statistical
models were used to examine whether weight gain in one person was associated
with weight gain in his or her friends, siblings, spouse, and neighbors.
Results showed discernible clusters of obese persons (BMI 30) were present
in the network at all time points, and the clusters extended to three degrees
of separation. These clusters did not appear to be solely attributable to the
selective formation of social ties among obese persons. A person's chances of
becoming obese increased by 57% if he or she had a friend who became obese in a
given interval. Among pairs of adult siblings, if one sibling became obese, the
chance that the other would become obese increased by 40%. If one spouse became
obese, the likelihood that the other spouse would become obese increased by
37%. These effects were not seen among neighbors in the immediate geographic
location. Persons of the same gender had relatively greater influence on each
other than those of the opposite gender. It was concluded that Network
Phenomena appear to be relevant to the biologic and behavioral trait of
obesity, and that obesity appears to spread through social ties. And yes, these
findings have implications for clinical and public health interventions.
VI. CARDIOLOGY
Defibrillators Highly
Effective in Hypertrophic Cardiomyopathy
Recently, the
implantable cardioverter-defibrillator (ICD) has been promoted for prevention
of sudden death in hypertrophic cardiomyopathy (HCM). However, the
effectiveness and appropriate selection of patients for this therapy is
incompletely resolved. As a result, a study published in the Journal of the
American Medical Association (2007;298:405-412) was performed to study the
relationship between clinical risk profile and incidence and efficacy of ICD
intervention in HCM. The investigation was based on a multicenter registry
study of ICDs implanted between 1986 and 2003 in 506 unrelated patients with
HCM. To be included into the study, patients had to have been judged to be at
high risk for sudden death and received and ICD. Mean follow-up was 3.7 years.
Measured risk factors for sudden death included family history of sudden death,
massive left ventricular hypertrophy, nonsustained ventricular tachycardia on
Holter monitoring, and unexplained prior syncope. The main outcome measure was
appropriate ICD intervention terminating ventricular tachycardia or
fibrillation. Of the 506 patients, most were young (mean age, 42 years) at
implantation, and most (439 [87%]) had no or only mildly limiting symptoms.
Results showed that ICD interventions appropriately terminated ventricular
tachycardia/fibrillation in 103 patients (20%). Intervention rates were 10.6%
per year for secondary prevention after cardiac arrest and 3.6% per year for
primary prevention. Time to first appropriate discharge was up to 10 years,
with a 27% probability 5 years or more after implantation. For primary
prevention, 18 of the 51 patients with appropriate ICD interventions (35%) had
undergone implantation for only a single risk factor. Interestingly, likelihood
of appropriate discharge was similar in patients with 1, 2, or 3 or more risk
markers. The single sudden death due to an arrhythmia (in the absence of
advanced heart failure) resulted from an ICD malfunction. ICD complications
included inappropriate shocks in 136 patients (27%). Based on the study
results, it was shown that in a high-risk HCM cohort, ICD interventions for
life-threatening ventricular tachyarrhythmias were frequent and highly
effective in restoring normal rhythm, and that an important proportion of ICD
discharges occurred in primary prevention patients who had undergone
implantation for a single risk factor. According to the authors, a single
marker of high risk for sudden death may be sufficient to justify consideration
for prophylactic defibrillator implantation in selected patients with HCM.
VII. REGULATORY AFFAIRS
TARGET HEALTH
excels in Regulatory Affairs and works closely with many of its clients
performing all FDA submissions. TARGET HEALTH receives daily updates of new
developments at FDA. Each week, highlights of what is going on at FDA are
shared to assure that new information is expeditiously made available.
Financial Disclosures
On February 2,
1998, FDA published a final rule requiring anyone who submits a marketing
application of any drug, biological product or device to submit certain
information concerning the compensation to, and financial interests of, any
clinical investigator conducting clinical studies covered by the rule. Under
the applicable regulations (21 CFR Parts 54, 312, 314, 320, 330, 601, 807, 812,
814, and 860), an applicant is required to submit to FDA a list of clinical
investigators who conducted covered clinical studies and certify and/or
disclose certain financial arrangements as follows:
If FDA
determines that the financial interests of any clinical investigator raise a
serious question about the integrity of the data, FDA will take any action it
deems necessary to ensure the reliability of the data including:
For more
information about our expertise in Regulatory Affairs, please contact Dr. Jules T. Mitchel or Dr.
Glen Park.
VIII. TARGET HEALTH
TARGET HEALTH
INC. (www.targethealth.com)is a
full service eCRO with full-time staff dedicated to all aspects of drug and
device development. Areas of expertise include Regulatory Affairs, comprising,
but not limited to, IND, IDE, NDA, PMA and 510(k) submissions, execution of
Clinical Trials, Project Management, Biostatistics and Data Management, Web
Trials, utilizing Target e*CRF®, our proprietary Internet-based Clinical Trial
System, and Medical Writing. TARGET HEALTH's Pharmaceutical Advisory Dream
Team assists companies in strategic planning from Discovery to Market
Launch. Let us help you on your next project.
TARGET HEALTH INC.
261 Madison Avenue
24th Floor
New York, NY 10016
Phone: (212) 681-2100; Fax (212) 681-2105
http://blog.targethealth.com
www.targethealth.com
Dr. Jules T. Mitchel,
President
Ms Joyce Hays, CEO
©2007 Target Health Inc. All rights reserved
