Pre-Market Approval Application (PMA) Will be Submitted to FDA This Week That Used Target e*CTR® eSource Solution

 

A few months ago, after discussing our eSource solution for clinical trials, a Big Pharma colleague of Target Health said, “You do it first; we know you will not fail; then we will all follow.“

 

That day has come. On Tuesday of this week, Target Health will submit the FIRST FDA marketing application that used direct data entry at the time of the clinic visit. This multicenter study of a novel medical device, was performed at 6 medical centers with 133 subjects. An NDA is planned in Q3 2015 which will include 7 studies, approximately 400 subjects and 35 clinical research sites. Here are some metrics from close to 20 studies:

 

1. In out-patient studies, >90% of data are entered in real time

2. Database changes due to all queries is around 1%

3. Database changes have no impact on study results

4. Sites can see twice as many patients as normal

5. Queries are generated in real time

6. Almost complete elimination of protocol violation prior to treatment

 

A Snowy Morning in February – View From the 24th Floor Offices of Target Health Inc.

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February 2015 – Morning Snow Storm From the 24th Floor Offices of Target Health, 2015 ©Target Health Inc.

 

 

ON TARGET is the newsletter of Target Health Inc., a NYC-based, full-service, contract research organization (eCRO), providing strategic planning, regulatory affairs, clinical research, data management, biostatistics, medical writing and software services to the pharmaceutical and device industries, including the paperless clinical trial.

 

For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 104). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel or Ms. Joyce Hays. The Target Health software tools are designed to partner with both CROs and Sponsors. Please visit the Target Health Website.

 

Joyce Hays, Founder and Editor in Chief of On Target

Jules Mitchel, Editor

 

QUIZ

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Measles

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A child showing a classic 4-day measles rash.

 

Measles, also known as morbilli, or rubeola is a highly contagious infection caused by the measles 1) ___. Initial symptoms typically include fever, often greater than 40 oC (104.0 oF), cough, runny nose, and red eyes. Two or three days after the start of symptoms small white spots may form inside the mouth, known as Koplik’s spots. A red, flat rash which usually starts on the face or ears, and then spreads to the rest of the body typically begins three to five days after the start of symptoms. Symptoms usually develop 10-12 days after exposure to an infected person and lasts 7-10 days. Complications occur in about 30% and may include: diarrhea, blindness, inflammation of the brain, and pneumonia among others. Rubella (German measles) and roseola are different diseases.

 

Measles is an airborne disease which spreads easily through the coughs and 2) ___ of those infected. It may also be spread through contact with saliva or nasal secretions. Nine out of ten people who are not immune who share living space with an infected person will catch it. People are infectious to others from four days before to four days after the start of the rash. People usually only get the disease at most once. Testing for the virus in suspected cases is important for public health efforts. The measles 3) ___ is effective at preventing the disease. Vaccination has resulted in a 75% decrease in deaths from the disease since the year 2000 with about 85% of children globally being vaccinated. No specific treatment is available. Supportive care, however, may improve outcomes. This may include giving oral rehydration solution (slightly sweet and salty fluids), healthy food, and medications to help with the fever. Antibiotics may be used if a bacterial infection such as pneumonia occurs. Vitamin A supplementation is also recommended in the developing world.

 

Measles affects about 20 million people a year, primarily in the developing areas of Africa and Asia, and resulted in about 96,000 deaths in 2013, down from 545,000 deaths in 1990. In 1980, the disease was estimated to have caused 2.6 million deaths per year. Before immunization, in the US between there were three and four million cases occurred a year. Most of those who are infected and who die are less than 4) ___ years old. The risk of death among those infected is usually 0.2%, but may be up to 10% in those who have malnutrition. It is not believed to affect animals.

 

The classic signs and symptoms of measles include four-day fevers (the 4 D’s) and the three C’s – cough, coryza (head cold), and conjunctivitis (red eyes), along with fever and rashes. Koplik’s spots seen inside the mouth are pathognomonic (diagnostic) for measles, but are temporary and therefore rarely seen. Their recognition, before the affected person reaches maximum infectivity, can be used to reduce spread of epidemics.  The characteristic measles rash is classically described as a generalized red maculopapular rash that begins several days after the fever starts. It starts on the back of the 5) ___ and, after a few hours, spreads to the head and neck before spreading to cover most of the body, often causing itching. The measles rash appears two to four days after the initial symptoms and lasts for up to eight days. The rash is said to “stain“, changing color from red to dark brown, before disappearing. Complications with measles are relatively common, ranging from mild complications such as diarrhea to serious complications such as pneumonia (either direct viral pneumonia or secondary bacterial pneumonia), otitis media, acute brain inflammation, and very rarely SSPE (subacute sclerosing panencephalitis), and corneal ulceration (leading to corneal scarring). Complications are usually more severe in adults who catch the virus. The death rate in the 1920s was around 30% for measles pneumonia.

 

Between 1987 and 2000, the case fatality rate across the US was three measles-attributable deaths per 1000 cases, or 0.3%. In underdeveloped nations with high rates of malnutrition and poor healthcare, fatality rates have been as high as 28%. In immuno-compromised persons (e.g., people with AIDS) the fatality rate is approximately 30%. Risk factors for severe measles and its complications include: malnutrition, underlying immunodeficiency, pregnancy , and vitamin 6) ___ deficiency.

 

Measles is caused by the measles virus, a single-stranded, negative-sense, enveloped RNA virus of the genus Morbillivirus within the family Paramyxoviridae. The virus was first isolated in 1954 by Thom as Peebles MD and John Enders MD, who were careful to point out that the isolations were made from patients who had Koplik’s 7) ___. Humans are the natural hosts of the virus; no other animal reservoirs are known to exist. This highly contagious virus is spread by coughing and sneezing via close personal contact or direct contact with secretions. Risk factors for measles virus infection include: immunodeficiency caused by HIV or AIDS, leukemia, alkylating agents, or corticosteroid therapy, regardless of immunization status; travel to areas where measles is endemic or contact with travelers to endemic areas; and the loss of passive, inherited antibodies before the age of routine immunization.

 

Alternatively, laboratory diagnosis of measles can be done with confirmation of positive measles IgM antibodies or isolation of measles virus RNA from respiratory specimens. For people unable to undergo phlebotomy, 8) ___ can be collected for salivary measles-specific IgA testing. Positive contact with other patients known to have measles adds strong epidemiological evidence to the diagnosis. The contact with any infected person in any way, including semen, saliva, or mucus, can cause infection. In developed countries, children are immunized against measles at 12 months, generally as part of a three-part MMR vaccine (measles, mumps, and rubella). The vaccination is generally not given earlier than this because sufficient anti-measles immunoglobulins (antibodies), acquired via the placenta from the mother during pregnancy may persist to prevent the vaccine viruses from being effective. A second dose is usually given to children between the ages of four and five, to increase rates of immunity. Vaccination rates, until recently, have been high enough to make measles relatively uncommon. Adverse reactions to vaccination are rare, with fever and pain at the injection site being the most common. Life-threatening adverse reactions occur in less than one per million vaccinations (<0.0001%). In developing countries where measles is highly endemic, WHO doctors recommend two doses of vaccine be given at six and nine months of age. The vaccine should be given whether the child is HIV-infected or not. The vaccine is less effective in HIV-infected infants than in the general population, but early treatment with anti-retroviral drugs can increase its effectiveness.

 

There is no specific 9) ___ for measles. Most patients with uncomplicated measles will recover with rest and supportive treatment. It is, however, important to seek medical advice if the patient becomes sicker, as they may be developing complications. Some patients will develop pneumonia as a sequel to the measles. Other complications include ear infections, bronchitis (either viral bronchitis or secondary bacterial bronchitis), and brain inflammation. Brain inflammation from measles has a mortality rate of 15%. While there is no specific treatment for brain inflammation from measles, antibiotics are required for bacterial pneumonia, sinusitis, and bronchitis that can follow measles. The use of vitamin A in treatment has been investigated. A systematic review of trials into its use found no significant reduction in overall mortality, but it did reduce mortality in children aged under two years. A specific drug treatment for measles ERDRP-0519 has shown promising results in animal studies, but has not yet been tested in humans. The majority of patients survive measles, though in some cases, complications may occur, which may include bronchitis, and – in about 1 in 100,000 cases – panencephalitis, which is usually fatal. Acute measles encephalitis is another serious risk of measles virus infection. It typically occurs two days to one week after the breakout of the measles exanthem and begins with very high fever, severe headache, convulsions and altered mentation. A patient may become comatose, and death or brain injury may occur.

 

10) ___ is extremely infectious and its continued circulation in a community depends on the generation of susceptible hosts by birth of children. In communities which generate insufficient new hosts the disease will die out. This concept was first recognized in measles by Bartlett in 1957, who referred to the minimum number supporting measles as the critical community size (CCS). Analysis of outbreaks in island communities suggested that the CCS for measles is about 250,000. In 2011, the WHO estimated that there were about 158,000 deaths caused by measles. This is down from 630,000 deaths in 1990. In developed countries, death occurs in 1 to 2 cases out of every 1,000 (0.1% – 0.2%). In populations with high levels of malnutrition and a lack of adequate healthcare, mortality can be as high as 10%. In cases with complications, the rate may rise to 20-30%. Increased immunization has led to an estimated 78% drop in measles deaths among UN member states. This reduction made up 25% of the decline in mortality in children under five during this period.

 

ANSWERS: 1) virus; 2) sneezes; 3) vaccine; 4) five; 5) ears; 6) A; 7) spots; 8) saliva; 9) treatment; 10) Measles

 

Measles

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6th century Aztec drawing of someone with measles Wikipedia.com

 

The Antonine Plague, 165-180 AD, also known as the Plague of Galen, who described it, was probably smallpox or measles. The epidemic may have claimed the life of Roman emperor Lucius Verus. Total deaths have been estimated at five million. Estimates of the timing of evolution of measles seem to suggest this plague could have been something other than measles.

 

The first scientific description of measles and its distinction from smallpox and chickenpox is credited to the Persian physician Rhazes (860-932), who published The Book of Smallpox and Measles. Given what is now known about the evolution of measles, this account is remarkably timely, as recent work that examined the mutation rate of the virus indicates the measles virus emerged from rinderpest (Cattle Plague) as a zoonotic disease between 1100 and 1200 CE, a period that may have been preceded by limited outbreaks involving a virus not yet fully acclimated to humans. This agrees with the observation that measles requires a susceptible population of >500,000 to sustain an epidemic, a situation that occurred in historic times following the growth of medieval European cities.

 

Measles is an endemic disease, meaning that it has been continually present in a community, and many people develop resistance. In populations that have not been exposed to measles, exposure to a new disease can be devastating. In 1529, a measles outbreak in Cuba killed two-thirds of the natives who had previously survived smallpox. Two years later, the disease killed half the population of Honduras, and had ravaged Mexico, Central America, and the Inca civilization. A Scottish physician, Francis Home, demonstrated in 1757 that measles was caused by an infectious agent present in the blood of patients.

 

Historically, measles was prevalent throughout the world, as it is highly contagious. According to the National Immunization Program, 90% of people were infected with measles by age 15. Before the vaccine was introduced in 1963, there were an estimated 3-4 million cases in the U.S. each year. Between roughly 1855 to 2005 measles has been estimated to have killed about 200 million people worldwide. Measles killed 20% of Hawaii’s population in the 1850s. In 1875, measles killed over 40,000 Fijians, approximately one-third of the population. In the 19th century, the disease decimated the Andamanese population who live off the coast of India. In 1954, the virus causing the disease was isolated from an 13-year-old boy from the United States, David Edmonston, and adapted and propagated on chick embryo tissue culture. To date, 21 strains of the measles virus have been identified. While at Merck, Maurice Hilleman developed the first successful vaccine. Licensed vaccines to prevent the disease became available in 1963. An improved measles vaccine became available in 1968.

 

 

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Thomas C. Peebles MD (1921-2010), Isolated Measles Virus

 

Thomas Chalmers Peebles (1921- 2010) was an American physician who made multiple discoveries in the field of medicine, including being the first to isolate the measles virus. Peebles also did research that led to the development of fluoridated vitamins and did research that showed that tetanus vaccine could be given once every 10 years, rather than annually as had been the widespread practice. Peebles was born on June 5, 1921, in Newton, Massachusetts, and graduated from Harvard University in 1942 with a major in French language. He enlisted in the United States Navy and served as a bomber pilot in the Pacific Theater of Operations, earning the Distinguished Flying Cross. He had been recruited by KLM as a pilot after completing his military service but chose to apply to Harvard Medical School, which rejected him because he had received a D in college biology. He attended Boston University for a year, taking the pre-med courses he had not taken as an undergraduate. He was finally accepted by Harvard Medical School, but spent an intervening year teaching at an elementary school in South Carolina. He worked at a laundry to help pay for those medical school costs not covered by the G.I. Bill. After completing medical school he did an internship and residency in pediatrics at Massachusetts General Hospital, where he later became a Chief Resident of Pediatrics. He was also a Fellow at Children’s Hospital Boston, where he worked with Dr. John Franklin Enders. In the early 50s, Dr. Enders turned his attention to measles, which nearly every child then got by age 15 and which killed around 450 people a year in the United States. Dr. Enders asked Dr. Peebles, just two years out of medical school, to help. When word came of a measles outbreak at a nearby school, Dr. Peebles hastened to take blood samples and throat swabs from children there. It was from a throat swab of an 11-year-old student named David Edmonston that Dr. Peebles was able to grow a virus in a soup of human tissue.

 

But Dr. Peebles was the only one in the laboratory, including Dr. Enders, who recognized the potential significance of the giant cellular glob formed by smaller cells that had broken down and coalesced. Dr. Enders, believing that his associate had not found anything of significance, took Dr. Peebles off measles research. But Dr. Peebles continued his work on his own and was eventually successful in cultivating generations of the virus. He inoculated monkeys, and they came down with measles. Peebles and Enders would lead others in developing a measles vaccine, which was grown from that first tissue sample from David Edmonston, that has almost eradicated the disease in most developed countries. Dr. Peebles’s identification of the virus was the necessary first step.  The vaccine for measles has led to the near-complete elimination of the disease in the United States and other developed countries. Peebles died at age 89 on July 8, 2010, in his home in Port Charlotte, Florida. He was survived by his wife, the former Annie Diffley, as well as by a daughter, two sons and five grandchildren.

 

 

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Maurice Hilleman’s measles vaccine is estimated to prevent 1 million deaths every year.

 

Maurice Ralph Hilleman (1919 – 2005) was an American microbiologist who specialized in vaccinology and developed over 36 vaccines, an unparalleled degree of productivity. Of the 14 vaccines routinely recommended in current vaccine schedules, he developed eight: those for measles, mumps, hepatitis A,hepatitis B, chickenpox, meningitis,pneumonia and Haemophilus influenzae bacteria. He also played a role in the discovery of the cold-producing adenoviruses, the hepatitis viruses, and the cancer-causing virus SV40. He is credited with saving more lives than any other medical scientist of the 20th century. Robert Gallo described him as “the most successful vaccinologist in history.” Hilleman was born on a farm near the high plains town of Miles City, Montana. His parents were Anna and Gustav Hillemann, and he was their eighth child. His twin sister died when he was born, and his mother died two days later. He was raised in the nearby household of his uncle, Robert Hilleman, and worked in his youth on the family farm. He credited much of his success to his work with chickens as a boy; since the 1930s fertile chicken eggs have often been used to grow viruses for vaccines. His family belonged to the Missouri Synod Lutheran Church. When he was in the eighth grade, he discovered Charles Darwin, and was caught reading The Origin of Species in church. Later in life, he rejected religion. Due to lack of money, he almost failed to attend college. His eldest brother interceded, and Hilleman graduated first in his class from Montana State University with family help and scholarships. He won a fellowship to the University of Chicago and received his doctoral degree in microbiology in 1941. His doctoral thesis was on chlamydia infections, which were then thought to be caused by a virus. Hilleman showed that these infections were, in fact, caused by a species of bacterium, Chlamydia trachomatis, that grows only inside of cells. After joining E.R. Squibb & Sons (now Bristol-Myers Squibb), Hilleman developed a vaccine against Japanese B encephalitis, a disease that threatened American troops in the Pacific Ocean theater of World War II. As chief of the Department of Respiratory Diseases at Army Medical Center (now the Walter Reed Army Institute of Research) from 1948 to 1958, Hilleman discovered the genetic changes that occur when the influenza virus mutates, known as shift and drift. That helped him to recognize that a 1957 outbreak of influenza in Hong Kong could become a huge pandemic. Working on a hunch, after nine 14-hour days he and a colleague found that it was a new strain of flu that could kill millions. Forty million doses of vaccines were prepared and distributed. Although 69,000 Americans died, the pandemic could have resulted in many more deaths in the United States. Hilleman was awarded the Distinguished Service Medal from the American military for his work.

 

In 1957, Hilleman joined Merck & Co. (Whitehouse Station, New Jersey), as head of its new virus and cell biology research department in West Point, Pennsylvania. It was while with Merck that Hilleman developed most of the forty experimental and licensed animal and human vaccines with which he is credited, working both at the laboratory bench as well as providing scientific leadership. In 1963, his daughter Jeryl Lynn came down with the mumps. He cultivated material from her, and used it as the basis of a mumps vaccine. The Jeryl Lynn strain of the mumps vaccine is still used today. The strain is currently used in the trivalent (measles, mumps and rubella) MMR vaccine that he also developed, the first vaccine ever approved incorporating multiple live virus strains. He and his group invented a vaccine for Hepatitis B by treating blood serum with pepsin, urea and formaldehyde. This was licensed in 1981, but withdrawn in 1986 in the United States when it was replaced by a vaccine that was produced in yeast. This vaccine is still in use today. By 2003 150 countries were using it and the incidence of the disease in the United States in young people had decreased by 95%. Hilleman considered his work on this vaccine to be his single greatest achievement. Hilleman was one of the early vaccine pioneers to warn about the possibility that simian viruses might contaminate vaccines. The best-known of these viruses became SV40, a viral contaminant of the polio vaccine, whose discovery led to the recall of Salk’s vaccine in 1961 and its replacement with Albert Sabin’s oral vaccine. The contamination actually occurred in both vaccines at very low levels, but because the oral vaccine was ingested rather than injected, it did not result in infections or any harm. Hilleman served on numerous national and international advisory boards and committees, academic, governmental and private, including the National Institutes of Health’s Office of AIDS Research Program Evaluation and the Advisory Committee on Immunization Practices of the National Immunization Program. In his later life, Hilleman was an adviser to the World Health Organization. He retired as senior vice president of the Merck Research Labs in 1984 at the mandatory retirement age of 65. He then directed the newly created Merck Institute for Vaccinology where he worked for the next twenty years. At the time of his death on April 11, 2005, at the age of 85, Hilleman was Adjunct Professor of Pediatrics at the University of Pennsylvania in Philadelphia. Hilleman was a forceful man who was at the same time modest in his claims. None of his vaccines or discoveries are named after him. He ran his laboratory like a military unit, and he was the one in command. He terminated every subordinate who did not measure up to his standards. For a time, he kept a row of “shrunken heads” (actually fakes made by one of his children) in his office as trophies that represented each of his fired employees. He used profanity and tirades freely to drive his arguments home, and once, famously, refused to attend a mandatory “charm school” course intended to make Merck middle managers more civil. His men were fiercely loyal to him.

 

Hilleman was an elected member of the U.S. National Academy of Science, the Institute of Medicine, the American Academy of Arts and Sciences, and the American Philosophical Society. In 1988 President Ronald Reagan presented him with the National Medal of Science, the nation’s highest scientific honor. He received the Prince Mahidol Award from the King of Thailand for the advancement of public health, as well as a special lifetime achievement award from the World Health Organization, the Mary Woodard Lasker Award for Public Service and the Sabin Gold Medal and Lifetime Achievement Awards. In March 2005 the University of Pennsylvania School of Medicine’s Department of Pediatrics and The Children’s Hospital of Philadelphia, in collaboration with The Merck Company Foundation, announced the creation of The Maurice R. Hilleman Chair in Vaccinology. Robert Gallo, co-discoverer of the virus that causes AIDS, once said “If I had to name a person who has done more for the benefit of human health, with less recognition than anyone else, it would be Maurice Hilleman. Maurice should be recognized as the most successful vaccinologist in history.” After Hilleman’s death Ralph Nader wrote, “Yet almost no one knew about him, saw him on television, or read about him in newspapers or magazines. His anonymity, in comparison with Madonna, Michael Jackson, Jose Canseco, or an assortment of grade B actors, tells something about our society’s and media’s concepts of celebrity; much less of the heroic.” In 2007, Paul Offit published a biography of Hilleman, entitled Vaccinated: One Man’s Quest to Defeat the World’s Deadliest Diseases. On October 15, 2008, Merck named its Durham, North Carolina vaccine manufacturing facility in memory of Hilleman.

 

VIROLOGY

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Ebola Vaccine Trial Opens in Liberia

 

A large clinical trial to assess the safety and efficacy of two experimental vaccines to prevent Ebola virus infection is now open to volunteers in Liberia. The trial is being led by a recently formed Liberia-U.S. clinical research partnership and is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health. The Partnership for Research on Ebola Vaccines in Liberia or PREVAIL, a Phase 2/3 study, is designed to enroll approximately 27,000 healthy men and women aged 18 years and older. One vaccine candidate, cAd3-EBOZ, uses a chimpanzee-derived cold virus to deliver Ebola virus genetic material from the Zaire strain of virus causing the outbreak in Liberia. Published interim results from a Phase 1 trial of this vaccine, which was co-developed by NIAID scientists and GlaxoSmithKline, provided necessary safety information and showed that it prompted immune responses to the outer coat of Ebola virus. The other candidate, VSV-ZEBOV, employs vesicular stomatitis virus, an animal virus that primarily affects cattle, to carry an Ebola virus gene segment. The VSV-ZEBOV vaccine was developed by the Public Health Agency of Canada and licensed to NewLink Genetics Corporation through its wholly owned subsidiary BioProtection Systems Corporation. Phase 1 trial results of this vaccine also provided safety information and showed that it prompted immune responses to the outer coat of Ebola virus. These results have not yet been published but were made available to the regulatory bodies reviewing the study.

 

In addition to healthy adults in the general population, the trial will seek volunteers from groups at particular risk of Ebola infection, including health care workers, communities with ongoing transmission, contact tracers and members of burial teams. Social mobilization and community engagement activities began in Montserrado County, where the Liberian capital Monrovia is located, before the trial started and will continue in order to successfully recruit thousands of participants.

 

Participants will be assigned at random to one of three equal-sized groups. Volunteers in one group will receive a placebo (saline) injection, while the others will receive a single injection of either the cAd3-EBOZ vaccine or the VSV-ZEBOV vaccine. In addition to including a placebo group, the trial will be double-blinded. All participants will be advised on how to minimize the risk of becoming infected with Ebola virus and will be contacted by study staff about one week after the injection and then monthly for the duration of the study, which is currently expected to last about twelve months.

 

Given the current decline in the number of new Ebola cases in Liberia, study investigators anticipate the need for flexibility in the conduct and design of the trial to address the changing nature of the outbreak.

 

DIABETES

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Risk of All-Cause Mortality and Vascular Events in Women vs. Men with Type 1 Diabetes

 

Studies have suggested gender differences in the mortality rate associated with type 1 diabetes. As a result, a study published online in The Lancet Diabetes & Endocrinology (5 February 2015), performed a meta-analysis to provide reliable estimates of any gender differences in the effect of type 1 diabetes on risk of all-cause mortality and cause-specific outcomes.

 

The authors systematically searched PubMed for studies published between Jan 1, 1966, and Nov 26, 2014 and selected studies that reported gender-specific estimates of the standardized mortality ratio (SMR) or hazard ratios associated with type 1 diabetes, either for all-cause mortality or cause-specific outcomes. They then used random effects meta-analyses with inverse variance weighting to obtain gender-specific SMRs and their pooled ratio (women to men) for all-cause mortality, for mortality from cardiovascular disease, renal disease, cancer, the combined outcome of accident and suicide, and from incident coronary heart disease and stroke associated with type 1 diabetes.

 

Data from 26 studies including 214,114 individuals and 15,273 events were included. The pooled women-to-men ratio of the SMR for all-cause mortality was 1.37, for incident stroke 1.37, for fatal renal disease 1.44, and for fatal cardiovascular diseases 1.86. For incident coronary heart disease the gender difference was more extreme; the pooled women-to-men ratio of the SMR was 2.54. No evidence suggested a gender difference for mortality associated with type 1 diabetes from cancer, or accident and suicide.

 

According to the authors, women with type 1 diabetes have a roughly 40% greater excess risk of all-cause mortality, and twice the excess risk of fatal and nonfatal vascular events, compared with men with type 1 diabetes.

 

A Year of Significant Progress in Public Health

 

Source: FDA Blog

 

Drug Approvals: This past calendar year, FDA approved 51 novel drugs and biologics (41 by CDER and 10 by CBER), the most in almost 20 years. Among CDER’s 2014 approvals are treatments for cancer, hepatitis C and type-2 diabetes, as well as the most new drugs for “orphan“ diseases since Congress enacted the Orphan Drug Act over 30 years ago. Seventeen of these new approvals are “first in class“ therapies, which represent new approaches in the treatment of disease. In addition, CBER approved many important biological products in 2014, including a number of groundbreaking vaccines for meningitis B, the flu, and certain types of Human Papillomavirus, the latter of which is expected to prevent approximately 90 percent of the cervical, vulvar, vaginal and anal cancers caused by HPV.

 

According to FDA, these developments are a testament not just to our expanding understanding of human biology, the biology of disease and the molecular mechanisms that drive the disease process, but also to FDA’s innovative approaches to help expedite development and review of medical products that target unmet medical needs, while adhering to the established standards for safety and efficacy. These include enhanced guidance to shape the research and development agenda, early input on clinical study needs and design, expedited review programs, targeted regulatory advice and other tools and incentives that spur investment and innovation in new medical products to address unmet medical needs.

 

Opioids: This past year FDA took several actions to address the abuse of opioid drugs. First, FDA approved abuse deterrent labeling for three opioid products that are designed to deter prescription drug abuse. These drugs used different technologies to combat the abuse problem in different ways, such as making the product resistant to crushing or dissolving or using “aversive technology“ to discourage users from taking more than the approved dosage of the drug. To help encourage the development of more drugs in abuse-deterrent forms, FDA is also working to provide additional advice to manufacturers. Although abuse-deterrent opioid drugs are not a silver bullet to prevent opioid abuse, FDA believes that their work in this area will give physicians effective new treatment options with less risk of abuse. FDA also worked to improve the treatment of patients who overdose on opioids. FDA approved a new dosage form of naloxone, with an auto-injector to enable a caregiver to administer the drug in the emergency treatment of opioid overdose (as it rapidly reverses the effects of an overdose).

 

Antibiotic Resistance: FDA’s efforts, which are a critical part of the recently unveiled National Strategy on Combating Antibiotic Resistant Bacteria, offer a multi-pronged approach that recognizes that to effectively address this challenge means simultaneously addressing the many different causes for increasing antibiotic resistance. One important response has been efforts to expand the pipeline of new medical products, including therapeutics to treat and cure infection, diagnostics to aid in the identification of the cause of infection and of resistant infections, and vaccines to help prevent infection with bacteria in the first place. These efforts are already having an impact. In 2014, FDA approved four novel systemic antibiotics. In contrast, only five new antibiotics had been approved in the previous ten year period. In addition to working on the human medical product side, FDA also developed and, over the next two years will be implementing, an important complementary strategy to eliminate the use of medically important antibiotics for growth promotion in food-producing animals. This strategy, once fully implemented, also will bring the remaining uses of such drugs to treat, control or prevent disease in these animals under the oversight of veterinarians. All 26 animal health companies who produce those drugs have committed to participate, and 31 products already have been withdrawn from the market.

 

Pharmacy Compounding: FDA continues to respond effectively to the 2012 outbreak of fungal meningitis that was linked to contaminated compounded drugs. This included conducting more than 90 inspections of compounding facilities across the nation in the past year. As a result, numerous firms that engaged in poor sterile practices stopped making sterile drugs, and many firms recalled drugs that have been made under substandard conditions. FDA continues to implement the compounding provisions of the Drug Quality and Security Act (DQSA), and to develop and implement policies to address compounding by state-licensed pharmacies and the new category of registered outsourcing facilities.

 

Target Health congratulates FDA for these accomplishments and their willingness to make a difference.

 

Vaccination Machination

 

Mark L. Horn, MD, MPH, Chief Medical Officer, Target Health Inc.

 

Germany is battling a measles outbreak that is 10 times worse than the one in the U.S.

 

The United States experienced a record number of measles cases during 2014, with 644 cases from 27 states reported to CDC’s National Center for Immunization and Respiratory Diseases (NCIRD).

 

There is a growing public appreciation, fueled by media reports featuring interviews with experts from public health and medicine of the dangers caused by failure to vaccinate children for measles. What appears to be a benign request has become a political football. Does the following quote from New Jersey Governor, Chris Christie, really support the virtue of balance in the suddenly urgent vaccine discussions?

 

“I also understand that parents need to have some measure of choice in things as well. So that’s the balance that the government has to decide.“

 

Realistically though, how can one balance the certainty of placing individuals at risk for undoubtedly serious and potentially lethal diseases – measles most prominently in the current instance, with the so-called “right“ to make a decision unsupported by mainstream scientific evidence?

 

The vaccination conundrum has become especially difficult because of the frightening nature of one (erroneously) presumed complication, development of an autism spectrum disorder. These disorders are justly feared; they are of unknown cause, unpredictable in onset, have devastating impact, no cure, and affect children. Now discredited research implicated vaccination as a potential cause and, despite refutation of the work by the scientific community, fear of vaccination persists among the public.

 

Many State Governments, presumably wishing to be responsive to the desires of the public, have resisted mandatory vaccination and allowed objecting parents to “opt-out“ for various reasons. In many cases refusals can be based upon ?philosophical’ objections alone, essentially providing a limitless loophole.

 

The results of this experiment are now available, and the situation has evolved as expected.

 

The percentage of vaccinated children has declined, dropping below the level required to ensure population immunity thereby allowing the emergence of presumptively eliminated infectious diseases, measles among them. Given the epidemiology and severity of measles it is a certainty that some will become seriously ill, and a small number may well die from this entirely avoidable illness.

 

So, what duty do we owe parents who make a personal “philosophic“ choice at variance with accepted scientific evidence, a choice placing their own and other children–and adults–at risk? Reciprocally, what duty do they have to the rest of us since their choice imposes a risk without our consent?

 

“Top of mind“ solutions all seem burdened with complexity and cost.

 

For example, while it might seem reasonable to simply segregate unvaccinated children attending public schools, these children potentially create special risks for each other, as they are all unvaccinated. Bundling them together might create enhanced risk for youngsters who are not responsible for their status. In addition, unless the parents assume the cost of creating and maintaining this restricted educational environment, society would be, in effect, subsidizing their ?philosophical’ decision.

 

Perhaps, in States willing to leave the vaccination decision to parents, creating, managing, (and supporting) alternative educational arrangements should likewise be a parental responsibility. For those “voluntarily“ unvaccinated, families would assume responsibility for alternative (but acceptable, e.g. approved home schooling, private schools) educational arrangements. It is true that this will incur costs for these families, but with rights come responsibilities. Exercising the “right“ not to vaccinate confers the “responsibility“ to protect the public from the consequences of this decision.

 

A different set of issues exist for non-educational (non-mandatory) public and private activities. Private vendors (Disneyland, remember, is the epicenter of the current measles outbreak) could require proof of vaccination for attendance; public recreational facilities could do likewise.

 

Clearly solutions to this dilemma are complex, costly and potentially highly intrusive; however, individuals opting out of vaccinations are imposing costs and risks upon society. If the demand for this particular freedom of choice is to be fairly met, those exercising it must assume proper responsibility for the burdens imposed.

 

Alternatively, we could simply agree to make vaccination mandatory.

 

Delicious Fresh Fruit Dessert with Cointreau

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A wonderfully refreshing end to any meal! – ©Joyce Hays, Target Health Inc.

 

Ingredients

 

1 Blood orange

1 Cara Cara orange

1 Clementine orange

1 Pummelo orange

1 Satsumo orange

1 Sumo orange

1 Tangelo

1 Mandarin

1/2 pink grapefruit

4 to 5 whole Kumquats, rinsed and dried

Red seedless grapes, rinsed and dried

Fresh blueberries, rinsed and dried

Bottle of Cointreau to your taste

 

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Some of the ingredients ©Joyce Hays, Target Health Inc.

 

Directions

 

Depending on what’s available in your fresh fruit counter, choose 3 or 4 of the above types of citrus fruit now available from California. Try to have 3 different colors of orange. Since they’re all wonderful flavors, the variety of color makes a beautiful presentation.

 

Peel your 3 or 4 citrus types and then pull each segment of the fruit apart and put into a serving bowl.

 

Add the kumquats, grapes and blueberries.

 

Then pour Cointreau over the whole bowl of fruit and stir. Press the fruit lightly as you stir, to release some of the luscious juices. Let the fruit sit for at least an hour, before you serve it.

 

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Clementine oranges

 

In the middle of the freezing winter, we’re so lucky to have a plethora of fresh fruit

 

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Blood orange

 

Is Better Always the Enemy of Good? NO

 

The story starts with trying out a new orange that I hadn’t heard of – Sumo a Japanese creation. They were so delicious, low in calories and healthy, that I ate one each afternoon, as a snack to carry me over until dinner time. I ordered a case of 9, see below.

 

 

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Case of Sumo Oranges ©Joyce Hays, Target Health Inc.

 

The discovery of these Sumo oranges was so successful, that I began looking up other interesting citrus treats and came up with quite a few that’re accessible in my neighborhood. When I saw how colorful many were, I decided to combine them for a new dessert. See below the first of these desserts.

 

 

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Plain fresh citrus with nothing added, except fresh blueberries and red seedless grapes ©Joyce Hays, Target Health Inc.

 

In the middle of the freezing winter, we’re so lucky to have a plethora of fresh fruit. The above dessert was a fresh and welcome end to our dinner, one night.

 

But could this get better? Is better really the enemy of good?

 

The next night, I used the left-over fruit from the night before and added some Cointreau. A second wonderful dessert that we really loved. We agreed that this was so good, it was the kind of dessert we could have for many more nights. Especially when the fruit juices mixed with the Cointreau, overnight –  it was especially good.

 

How could it get any better? Who knew.

 

 

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Plain blueberry cake, just out of the oven ©Joyce Hays, Target Health Inc.

 

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From one Sumo orange to this finest of desserts, the story ends here. ©Joyce Hays, Target Health Inc. (BTW, baking aromas from the kitchen, filled our entire home)

 

Just when it seemed like things couldn’t get much better, they did! To the plain blueberry cake, warm and just out of the oven, I added the fresh fruit soaked in Cointreau on top of the cake, dripping with the delectable juices. I can’t tell you how delicious this was! This could be served in dessert heaven!

 

I suppose you could gild the lily with vanilla ice cream or whipped cream or Cool whip, but we didn’t. This is the end of the rainbow and we are satisfied and happy.

 

 

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New Zealand’s fine Te Koko Sauvignon Blanc ©Joyce Hays, Target Health Inc.

 

 

From Our Table to Yours!

 

Bon Appetit!