Protalix BioTherapeutics Approved in Israel

 

 

Protalix BioTherapeutics Receives Marketing Authorization for Elelyso™ for the Treatment of Gaucher Disease from the Israeli Ministry of Health

 

 

Congratulations again to our friends and colleagues at Protalix Biotherapeutics (Israel; all below) for another regulatory approval. Target Health is pleased to announce that it began collaborating with Protalix in 2004 and shepherded the entire program from pre-clinical to NDA. Dr. Glen Park (left), head of clinical and regulatory at Target Health, took the lead on the program together with Dr. Einat Almon (3rd from the left). And then there is our favorite scientist Yossi Yoseph (kneeling). Congratulations also must also go to Dr. Heschi Rotmensch, Managing Director of Cato Research Israel, and his team who managed all ex-North American Activities.

 

Protalix and friends at FDA in 2006
Left to right: Glen Park, Daniel Bartfeld, Einat Almon, Sharon Hashmueli, Ari Zimran, David Aviezer, Jules Mitchel and Yoseph Shaaltiel (kneeling), (Photo by Raul Chertkoff MD, Medical Director)

 

 

Protalix BioTherapeutics, Inc. announced this week that it has received marketing authorization from the Israeli Ministry of Health for Elelyso™ (taliglucerase alfa) for injection, an enzyme replacement therapy (ERT) for the long-term treatment of adults with Type 1 Gaucher disease. This is the second marketing approval of Elelyso, which was also approved by the FDA on May 1, 2012. Marketing applications have been filed in additional territories. Elelyso is marketed in the United States by the Company’s commercialization partner, Pfizer Inc.

 

Elelyso is the first plant cell-based biopharmaceutical approved for marketing by the Israeli Ministry of Health. It is also the first plant cell-expressed drug derived from ProCellEx® to achieve regulatory approval for marketing. ProCellEx is the Company’s proprietary plant cell-based protein expression system. Elelyso is a form of the human lysosomal enzyme, glucocerebrosidase, used to treat Gaucher disease.

 

“We are very excited to have our first drug product approved in our home country,” said Dr. David Aviezer (second from the right), President and Chief Executive Officer of Protalix BioTherapeutics. “In our development efforts, we enjoyed the cooperation of the leading Israeli medical and academic institutions, and we rely in part on support from research grants from the Israeli government. We are proud that our Gaucher disease treatment will be available for commercial sale in Israel.”

 

“The clinical studies of Elelyso to date, both the pivotal and the extension studies, demonstrate that Elelyso is an effective treatment for Gaucher disease,” said Professor Ari Zimran, M.D., (3rd from the right) Director of the Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel. “The results of the Company’s 24-month naive extension trial and switch over study support Elelyso as an important treatment alternative for Gaucher patients in Israel.”

 

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 at www.targethealth.com

Pellagra

 

The name “pellagra” comes from the Italian “pelle”, skin + “agra”, rough = rough skin, referring to the skin problems in pellagra.

 

 

Pellagra is a vitamin 1) ___ disease most commonly caused by a chronic lack of niacin (vitamin B3) in the diet. It can be caused by decreased intake of niacin or tryptophan, and possibly by excessive intake of leucine. It may also result from alterations in protein metabolism in disorders such as carcinoid syndrome. A deficiency of the amino acid lysine can lead to a deficiency of niacin, as well.

 

Pellagra is called the “disease of the four D’s” —

 

1.  Diarrhea;

2.  Dermatitis: A scaly rash on skin exposed to light or trauma;

3.  2) ___: Mental disorientation, delusions and depression; and

4. Death.

 

Other features are ulcerations within the mouth (glossitis), nausea, vomiting, seizures and balance disorder (ataxia).

 

Pellagra is now rare in developed countries which enjoy balanced diets and fortified foods, but it was once a huge 3) ___ health problem in the US. Three million Americans contracted pellagra and 100,000 died of it from 1906-40. Pellagra was especially a problem for the poor in the South whose meals usually consisted of the “three M’s”: meat (pork fatback); molasses; and meal (cornmeal). Today pellagra continues to be a problem in developing countries where there is significant 4) ___ or where niacin-deficient foods such as corn and rice are the primary sources of nutrition.

 

In 1937, Conrad Elvehjem, of Madison, Wisconsin, showed the vitamin niacin cured pellagra (manifested as black tongue in dogs). Later studies by Tom Spies, Marion Blankenhorn, and Clark Cooper established that niacin also cured pellagra in 5) ___, for which Time Magazine dubbed them its 1938 Men of the Year in comprehensive science. Research conducted between 1900 and 1950 found the number of cases of 6) ___ with pellagra was consistently double the number of cases of afflicted men. This is thought to be due to the inhibitory effect of estrogen on the conversion of the amino acid tryptophan to niacin, or to the differential and unequal access to quality foods within the household. Some researchers of the time gave a few explanations regarding the difference. As primary wage earners before 1950, men were given consideration and preference at the dinner table. They also had pocket money to buy food outside the household. Women gave quality protein foods to their children first. And, again before 1950, women also would eat after everyone else had a chance to eat

 

Pellagra can be common in people who obtain most of their food energy from maize (“corn” in American English), notably rural South America, where 7) __ is a staple food. If maize is not nixtamalized, it is a poor source of tryptophan, as well as niacin. Nixtamalization corrects the niacin deficiency, and is a common practice in Native American cultures that grow corn. Following the corn cycle, the symptoms usually appear during spring, increase in the summer due to greater sun exposure, and return the following spring. Indeed, 8) ___ was once endemic in the poorer states of the U.S. South, such as Mississippi and Alabama, as well as among the residents of jails and orphanages as studied by Dr. Joseph Goldberger.

 

Since 1900, safer and healthier foods have resulted from decreases in microbial contamination and increases in nutritional content. Identifying essential micronutrients and establishing food-fortification programs have almost eliminated major nutritional deficiency diseases such as rickets, goiter, and pellagra in the United States.

 

Untreated, the disease can kill within four or five years. Treatment is with nicotinamide, a chemical related to 9) ___. The frequency and amount of nicotinamide administered depends on the degree to which the condition has progressed.

 

ANSWERS: 1) deficiency; 2) Dementia; 3) public; 4) malnutrition; 5) humans; 6) women; 7) maize; 8) pellagra; 9) niacin

 

History of Pellagra (You Tube)

 

Joseph Goldberger MD (1874 – 1929)

 

 

Joseph Goldberger, M.D. (1874-1929) was a Hungarian Jewish physician and epidemiologist employed in the United States Public Health Service (PHS). He was an advocate for scientific and social recognition of the links between poverty and disease. He was nominated five times for the Nobel Prize for his work on the etiology of pellagra.

 

Goldberger was born in Giralt, Hungary (now in Slovakia). The youngest of six children, he emigrated to the U.S. with his parents in 1883, eventually settling in Manhattan’s Lower East Side. After completing his secondary education, Goldberger entered the City College of New York intending to pursue an engineering career. After a chance encounter in 1892, however, Goldberger became interested in medicine and transferred to the Bellevue Hospital Medical College (now the New York University School of Medicine), receiving his M.D. degree in 1895.

 

Goldberger joined the PHS in 1899, serving first post at the Port of New York, where he conducted health inspections of newly arrived immigrants. From 1902-1906, Goldberger held a number of epidemiology posts – in Mexico, Puerto Rico, Mississippi and Louisiana. He was involved the PHS’ efforts to combat yellow fever, typhus, dengue fever, and typhoid fever. He gave a particularly noted lecture in Boston on the effects of parasites in disease transmission. In 1909, Goldberger published his research on Schamberg disease, an acarine mite-based parasitic infection common among poor, inner-city populations. He also worked with John F. Anderson investigating the transmission of measles and typhus.

 

In 1914, Goldberger was asked by US Surgeon General, Rupert Blue to investigate pellagra, then an endemic disease in the Southern US. Goldberger’s theory that pellagra was associated with diet contradicted the commonly held medical opinion that pellagra was an infectious disease. After multiple restricted-diet experiments on prisoners over several years, Goldberger was able to demonstrate that individuals who consumed heavily corn-based diets (to the virtual exclusion of other foods) were at a greatly increased risk of contracting pellagra. Despite his careful experiments, Goldberger’s discovery proved socially and politically unacceptable, and he made little progress in gaining support for the treating of pellagra. With further research, however, in 1926 Goldberger was able to prove that a Vitamin B deficiency was the cause of pellagra.

 

Conrad Elvehjem discovered the specific mechanism-that pellagra is caused by a dietary lack of the B vitamin niacin along with reduced levels of the essential amino acid tryptophan-in 1937. The traditional food preparation method of corn (maize), nixtamalization, by native New World cultivators who had domesticated corn, required treatment of the grain with lime, an alkali. The lime treatment now has been shown to make niacin nutritionally available and reduce the chance of developing pellagra. When corn cultivation was adopted worldwide, this preparation method was not accepted because the benefit was not understood. The original cultivators, often heavily dependent on corn, did not suffer from pellagra; it became common only when corn became a staple that was eaten without the traditional treatment.

 

Pellagra was first described in Spain in 1735 by Gaspar Casal, who published a first clinical description in his posthumous Natural and Medical History of the Asturian Principality (1762). This led to the disease being known as “Asturian leprosy”, and it is recognized as the first modern pathological description of a syndrome. It was an endemic disease in northern Italy, where it was named pelle agra (pelle = skin; agra = sour) by Francesco Frapoli of Milan. Because pellagra outbreaks occurred in regions where maize was a dominant food crop, the belief for centuries was that the maize either carried a toxic substance or was a carrier of disease. Later, the lack of pellagra outbreaks in Mesoamerica, where maize is a major food crop, led researchers to investigate processing techniques in that region.

 

In the early 1900s, pellagra reached epidemic proportions in the American South. Pellagra deaths in South Carolina numbered 1,306 during the first ten months of 1915; 100,000 Southerners were affected in 1916. At this time, the scientific community held that pellagra was probably caused by a germ or some unknown toxin in corn. The Spartanburg Pellagra Hospital in Spartanburg, South Carolina, was the nation’s first facility dedicated to discovering the cause of pellagra. It was established in 1914 with a special congressional appropriation to the PHS and set up primarily for research. In 1915, Joseph Goldberger, assigned to study pellagra by the Surgeon General of the United States, showed pellagra was linked to diet by inducing the disease in “volunteer” prisoners, using the Spartanburg Pellagra Hospital as his clinic. By 1926, Goldberger established a balanced diet or a small amount of brewer’s yeast prevented pellagra. After inspecting Southern orphanages, mental hospitals and prisons, Goldberger made the pivotal observation that the malnourished inmates of those institutions often developed pellagra while the better-fed staff did not. Pellagra, he deduced, did not arise from germs, as was commonly believed, but rather from a nutritional deficiency. To prove this, Dr. Goldberger, his assistants and even his wife engaged in experiments called “filth parties.” They injected themselves with blood or ingested the scabs, feces and body fluids of patients. None developed pellagra. He also did decisive experiments with Mississippi prison inmates (who “volunteered” in return for a full pardon). Dr. Goldberger fed them a poor diet that he believed caused pellagra and within months, many developed the disease. He then added meat, fresh vegetables and milk to their diet and reversed all of the signs and symptoms of pellagra. Dr. Goldberger never identified the dietary principle that had this extraordinary effect. He died in 1929 (of kidney cancer).

 

Eight years later, the factor was found to be niacin. This discovery was made in 1937 at the University of Wisconsin. Niacin is abundant in red meat, fish, poultry, and green leafy vegetables. Niacin can prevent pellagra (and can cure it).

Clinical Trial of a Farnesyltransferase Inhibitor in Children with Hutchinson-Gilford Progeria Syndrome

 

Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare, fatal, segmental premature aging syndrome caused by a mutation in LMNA that produces the farnesylated aberrant lamin A protein, progerin. This multisystem disorder causes failure to thrive and accelerated atherosclerosis leading to early death. Farnesyltransferase inhibitors have ameliorated disease phenotypes in preclinical studies.

 

Leon Botha, a South African artist and DJ who became widely known through his association with the band Die Antwoord. He was 26 when he died one day after his birthday. Botha was one of the longest-living persons ever to have been diagnosed with this rare disease.

 

 

According to an article published online in the Proceedings of the National Academy of Sciences (24 September 2012), 25 patients with HGPS received the farnesyltransferase inhibitor lonafarnib for a minimum of 2 years. Primary outcome success was predefined as a 50% increase over pretherapy in estimated annual rate of weight gain, or change from pretherapy weight loss to statistically significant on-study weight gain. Results showed that 9 patients experienced a >50% increase, six experienced a >50% decrease, and 10 remained stable with respect to rate of weight gain. Secondary outcomes included decreases in arterial pulse wave velocity and carotid artery echodensity and increases in skeletal rigidity and sensorineural hearing within patient subgroups. All patients improved in one or more of these outcomes.

 

According to the authors, results from this clinical treatment trial for children with HGPS provide preliminary evidence that lonafarnib may improve vascular stiffness, bone structure, and audiological status.

ONCOLOGY

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Major Cancer Protein Amplifies Global Gene Expression

 

MYC (c-Myc) is a regulator protein that codes for a transcription factor. Myc aids in cell activation, a process in which cells mature and divide quickly. During an immune response, for example, white blood cells are activated to help fight infections. If activation isn’t properly regulated, then cells can start growing out of control and result in cancer. Researchers have known that abnormally high levels of Myc can lead to cancer, but until now, no one had been able to explain how it can lead to so many different cancers.

 

According to an article published online in Cell (28 September 2012), a discovery may have taken place to explain why Myc can provoke a variety of cancers. Like many proteins associated with cancer, Myc helps regulate cell growth, but unlike many other cell growth regulators, Myc does not turn genes on or off, but instead boosts the expression of genes that are already turned on.

 

The authors used a specially designed fluorescent protein that allowed them to track Myc in white blood cells in vitro. White blood cells were chosen, specifically B cells and T cells that fight infections, because they are frequently affected by abnormal Myc and can transform into lymphoma and myeloma cells. The authors exposed the B and T cells to foreign toxins to stimulate an immune response and activate the fluorescent Myc. Is this way it was possible to examine the cells at different time points and see which genes the Myc proteins seemed to affect.

 

The analysis revealed that Myc didn’t prefer any specific type of gene. Instead, Myc proteins were present at nearly every gene that was already expressed, or turned on. The study also noticed that the amount of Myc at each expressed gene correlated with how active that gene was prior to immune stimulation. The more active the gene, the more Myc gathered there. Myc appeared to amplify productivity relative to the initial expression levels where it gave a small boost to genes with low activity and a big boost to genes with high activity. The study validated the idea of Myc as a universal amplifier by developing a set of B cells that did not produce functional Myc. When they were stimulated, the total cellular amount of RNA – an indicator of how much protein is being made – did not rise. When normal B cells were activated, the total cellular RNA did rise. When the authors conducted the same analysis in embryonic stem cells, they got similar results.

Cardiovascular Disease Risk in Healthy Children and its Association with Body Mass Index

 

According to an article published in the British Medical Journal (2012;345:e4759), a study was performed to describe the association and its magnitude between body mass index (BMI) category, gender, and cardiovascular disease risk parameters in school aged children in highly developed countries.

 

The study was a systematic review and meta-analysis of published data. Data sources included Embase, PubMed, EBSCOHost’s cumulative index to nursing and allied health literature, and the Web of Science databases for papers published between January 2000 and December 2011.

 

The analysis database included healthy children aged 5 to 15 in highly developed countries enrolled in studies done after 1990 and using prospective or retrospective cohort, cross sectional, case-control, or randomized clinical trial designs in school, outpatient, or community settings. Included studies had to report an objective measure of weight and at least one prespecified risk parameter for cardiovascular disease.

 

The authors included 63 studies of 49,220 children. All studies reported a worsening of risk parameters for cardiovascular disease in overweight and obese participants. Compared with normal weight children, systolic blood pressure was higher by 4.54 mm Hg (n=12,169, eight studies) in overweight children, and by 7.49 mm Hg (n=8,074, 15 studies) in obese children. Similar associations were found between groups in diastolic and 24 h ambulatory systolic blood pressure. Obesity adversely affected concentrations of all blood lipids; total cholesterol and triglycerides were 0.15 mmol/L (n=5,072) and 0.26 mmol/L (n=5,138) higher in obese children, respectively. Fasting insulin and insulin resistance were significantly higher in obese participants but not in overweight participants. Obese children had a significant increase in left ventricular mass of 19.12 g (n=223), compared with normal weight children.

 

According to the authors, having a body mass index outside the normal range significantly worsens risk parameters for cardiovascular disease in school aged children. This effect, already substantial in overweight children, increases in obesity and could be larger than previously thought. The authors concluded that there is a need to establish whether acceptable parameter cut-off levels not considering weight are a valid measure of risk in modern children and whether methods used in their study and reporting should be standardized.

TARGET HEALTH excels in Regulatory Affairs. Each week we highlight new information in this challenging area.

 

 

FDA Releases Preliminary Data on Arsenic Levels in Rice and Rice Products

 

 

As part of an ongoing and proactive effort to monitor food safety and address contaminants in food, the FDA has released preliminary data on arsenic levels in certain rice and rice products. The data are part of a larger FDA data collection and analysis about arsenic levels in rice and is based on the first set of approximately 200 samples of rice and rice products collected in the U.S. marketplace.

 

The FDA is in the process of collecting and analyzing a total of approximately 1,200 samples to examine the issue thoroughly. This data collection will be completed by the end of 2012. Once the data collection is completed, FDA will analyze these results and determine whether or not to issue additional recommendations. Based on the currently available data and scientific literature the FDA does not have an adequate scientific basis to recommend changes by consumers regarding their consumption of rice and rice products.

 

There are two types of arsenic compounds found in water, food, air, and soil: organic and inorganic. Together, the two types are referred to as total arsenic. The new data show how much inorganic arsenic the FDA found in its initial samples, which include various brands of rice (non-Basmati), Basmati rice, brown rice, rice cereals (puffed, non-puffed, hot cereal, and infant cereals), rice cakes, and rice milk.

 

The FDA’s analysis of these initial samples found average levels of inorganic arsenic for the various rice and rice products of 3.5 to 6.7 micrograms of inorganic arsenic per serving. Serving sizes varied depending on the rice product (for example, one serving of non-Basmati rice was equal to one cup cooked). While the FDA data is consistent with results from Consumer Reports recently published, the initial data collection is a first step in the agency’s ongoing more thorough data analysis. There are many different types of rice and rice products that are grown in different areas and under different conditions. Further analysis is needed to assess how these variations may affect the results.

Vitamin B Benefits of Chickpeas

 

 

 

Half a cup of chickpeas supplies about half a milligram of this cancer-fighting B vitamin, which doesn’t sound like much. But you only need slightly north of 3.5 milligrams daily for healthy benefits. And in a recent study, people from many different walks of life seemed to benefit from ample B.  No one will get pellagra with chickpeas in their diet. It is one of the earliest cultivated legumes: 7,500-year-old remains have been found in the Middle East.

 

The name “chickpea” traces back through the French chiche to cicer, Latin for ‘chickpea’ (from which the Roman cognomen Cicero was taken). The Oxford English Dictionary lists a 1548 citation that reads, “Cicer may be named in English Cich, or ciche pease, after the Frenche tonge.” The dictionary cites “Chick-pea” in the mid-18th century; the original word in English taken directly from French was chich, found in print in English in 1388 and became obsolete in the 18th century.

 

Domesticated chickpeas have been found in the aceramic levels of Jericho along with Cayonu in Turkey and in Neolithic pottery at Hacilar, Turkey. They are found in the late Neolithic (about 3500 BCE) at Thessaly, Kastanas, Lerna and Dimini. In southern France Mesolithic layers in a cave at L’Abeurador, Aude have yielded wild chickpeas carbon dated to 6790+90 BCE.

 

By the Bronze Age, chickpeas were known in Italy and Greece. In classical Greece, they were called erebinthos and eaten as a staple, a dessert, or consumed raw when young. The Romans knew several varieties such as venus, ram, and punic chickpeas. They were both cooked down into a broth and roasted as a snack. The Roman gourmet Apicius gives several recipes for chickpeas. Carbonized chickpeas have been found at the Roman legion fort at Neuss (Novaesium), Germany in layers from the first century CE, along with rice.

 

Chickpeas are mentioned in Charlemagne’s Capitulare de villis (about 800 CE) as cicer italicum, as grown in each imperial demesne. Albertus Magnus mentions red, white and black varieties. Nicholas Culpeper noted “chick-pease or cicers” are less “windy” than peas and more nourishing. Ancient people also associated chickpeas with Venus because they were said to offer medical uses such as increasing sperm and milk, provoking menstruation and urine and helping to treat kidney stones. “White cicers” were thought to be especially strong and helpful.

 

 

Hearty Chickpea Salad with Artichoke, Tomato & Arugula

 

 

It took a while to get all of these ingredients right  I think the secret flavors are the turmeric, the garlic, cilantro and the extremely refreshing lemon (or lime) zest and juice. The citrus addition is like adding sunshine to your taste buds.  I purposely watched and waited for my husband (and best critic) to sample the first bite; and my patience was rewarded with an emphatic, “Mmmmm wow!”  Need to say more?

 

Ingredients

  • One 15-oz. can chickpeas, rinsed and drained (if you want to soak & cook your own, do it the day before you make this salad)
  • One frozen package Artichoke hearts (steam for 8-10 minutes, drain, & towel dry)
  • 1 stalk celery, finely chopped
  • 2 ripe Tomatoes, cut into small pieces
  • ½ to 1/3 cup thinly sliced black olives (choose your favorite)
  • 1 avocado, cut into cubes
  • 2 cloves garlic, juiced (throw the pulp away)
  • 1 Onion, chopped very fine
  • 1/4 cup finely chopped fresh parsley
  • 1/3 cup chopped cilantro
  • 1 teaspoon turmeric
  • Zest & Juice from ½ a lemon (or lime)
  • 2 Tablespoons Olive oil
  • 1 cup baby arugula, stuff them down into the cup (wash these leaves very well, dry with paper towels)
  • 1 cup grated soy Mozzarella or crumbled feta cheese, or  crumbled gorgonzola cheese
  • Several grinds Black pepper & (optional) salt

 

Directions

  1. In a small bowl make the dressing.  Add the olive oil, garlic juice, chopped onion, turmeric, lemon zest and lemon juice, several grinds of black pepper (salt if desired)
  2. In your salad bowl, toss together chickpeas, artichoke hearts, celery, tomatoes, olives, cilantro and parsley.
  3. Now add to the chickpea mixture the dressing and stir well.  Next stir in slowly the arugula, avocado, and the cheese you selected to use.  Taste to check the seasoning and add more grinds of pepper, or anything else, if needed.
  4. Serve with warm pita bread and/or a grainy bread and a nice chilled Sauvignon Blanc or Pinot Grigio, or a crisp Chardonnay or even a dry rose

 

Cheers!

Targeting the Debates

 

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

 

As we enter the final stretch of what seems an endless presidential campaign the upcoming series of debates looms large as an unprecedented opportunity to see the candidates together, and if properly moderated, have a substantive discussion on issues of major importance.

 

Despite the recent and tragic intrusion of foreign policy as a major campaign issue, health care remains prominently on the agenda. As with other critical issues in this election, health care policy offers an array of clear choices for Americans. However, unlike many other matters where vagueness predominates and obfuscation is the rule, due to the Affordable Care Act (ACA) the Democratic Party’s ‘all-in’ health care positioning has been articulated with unusual precision. The Affordable Care Act (ACA) is the law; it has survived, with modifications, Supreme Court scrutiny and is in the process of implementation. One party is committed to its success, the other to its repeal. Politics doesn’t get much clearer than this.

 

The Democratic Party positions are embedded in the Act itself, and we’ve had an extensive public discussion of the new protections, benefits, potential restrictions, and costs, that are included in the Legislation. Virtually all acknowledge that the changes in the health care system embraced within the ACA are profound; as a consequence its repeal will be consequential. There are, therefore, important questions for the debate moderator to pose to the Republican nominee. The sample questions recommended below are not intended to make partisan points; indeed, Governor Romney should relish the opportunity, given a national podium, to reassure Americans that his proposals will retain key protections afforded them by the ACA and sustain long established programs upon which they and their families have come to rely.

 

Some sample questions –

 

First, as a policy goal the Republican platform articulates a goal of moving Medicare and Medicaid from defined benefit to defined contribution models. We’re currently in the midst of a similar transition in retirement planning, from older (and similarly apparently financially unsustainable) defined benefit pensions towards defined contribution (401k type) plans. Unfortunately many people, for an array of reasons, have not navigated this transition successfully, and we’re now seeing Americans forced to postpone retirement or confront an unexpectedly diminished lifestyle. Please explain how a Republican plan to similarly transition health programs to a defined contribution model will incorporate lessons from the retirement experience to avoid similar pitfalls.

 

Second, insurability and access concerns by, and for, citizens with pre-existing conditions have been drivers of health care reform efforts. Fear of coverage loss impacts virtually every family and has increased with recent job losses affecting especially vulnerable older Americans. The ACA addresses and for many solves this problem. The Republican Platform states that “Individuals with pre-existing conditions who maintain continuous coverage should be protected from discrimination,” and commits to assisting patients with pre-existing conditions “through reinsurance and risk adjustment.” These aspirations lack the certainty of the now formal guarantees in the ACA that coverage will be available and affordable. Please explain how these goals will translate into practical policies assuring that vulnerable Americans have affordable coverage beginning on “Day #1” following the proposed repeal of the ACA.

 

There are undoubtedly many additional questions to be asked. However, the fundamental challenge is clear: having resolved to repeal the ACA, the opposition needs to tell us, in equivalent detail, how they will replace it.