or
corporations are a people without soul?
or
people cannot discern safe and useful and inexpensive entities like BCG as taught by Dr. Denise L Fausmtan
see faustmanlab.org and pubmed.org ristori + bcg or faustmanlab.org
I have anectdotal experience that BCG works for plaque psoriasis. I also have Type 1 diabetes.
I would be glad to be shot with BCG again and again as needed and have the record made on You Tube.
I will contribute some of the money I will save and some for the suffering I may avoid to worthy people for whom science and art have not yet provided such a safe, simple and effective alternative.
I also urge those with cancer of various sorts to consider the utility of treating their disease with metormin and aspirin. You will easily find reasons to do so by searching pubmed.org. Because metformin and aspirin are
inexpensive you will not see people testing same.
Life is priceless and cheap.
Politics and Policy
Costly Vertex Drug Is Denied, and Medicaid Patients Sue
Kalydeco, a $300,000-a-Year Cystic Fibrosis Treatment, Sparks Legal Battle in Arkansas and Shows Dilemma States Face
July 16, 2014 10:30 p.m. ET
Chloe Jones, a 14-year-old plaintiff in the Arkansas suit,
wears an inflatable vest three times a day to help clear mucus from her
lungs.
Karen E. Segrave for The Wall Street Journal
LITTLE ROCK, Ark.—
Vertex Pharmaceuticals Inc.
VRTX -0.72%
's $300,000-a-year cystic-fibrosis drug has sparked a legal battle
here, where the state's Medicaid program is restricting access to the
expensive therapy.
In a lawsuit filed in
Arkansas federal court last month, three people suffering from the
fatal lung disease allege Medicaid officials have for two years denied
them access to Kalydeco because of its cost. The plaintiffs allege state
officials have violated their civil rights under federal law governing
Medicaid, the government-run insurance plan for the poor.
The
patients all meet the eligibility criteria established by the Food and
Drug Administration when it approved Kalydeco in 2012, including the
presence of a rare genetic mutation it is designed to correct. But
Arkansas officials have said the patients must prove their disease has
failed to benefit from older, less-expensive therapies, a policy their
doctors say contradicts treatment guidelines.
Arkansas
officials declined to comment on specific allegations but said they are
mainly restricting access because existing data don't support the
drug's use as a first option. Cost also appears to be a factor: Emails
obtained by the patients' attorneys show officials discussing Kalydeco's
cost, and their worries about the expense of future cystic fibrosis
drugs.
The legal flap is the latest
example of the pressure expensive new drugs are putting on cash-strapped
government insurance programs. State prison systems and some Medicaid
programs in recent months have limited use of another expensive new
drug—
Gilead Sciences Inc.
GILD -1.15%
's hepatitis C pill, Sovaldi, which has a wholesale price of
$84,000 for a full course of treatment—to all but the sickest patients.
The American Society of Clinical Oncology, meanwhile, recently said it
would begin publishing a cost-benefit guide to cancer drugs.
And more of these types of expensive, niche drugs are on the way. A recent report by insurer
UnitedHealth Group Inc.
UNH +2.87%
estimated specialty-drug spending in the U.S. could more than
quadruple to about $400 billion in 2020, up from $87 billion in 2012.
That
creates a dilemma for state Medicaid agencies with limited budgets. New
high-cost treatments like Kalydeco and Gilead's Sovaldi are likely just
the "tip of the iceberg" for high-price therapies, said
Matt Salo,
executive director of the National Association of Medicaid
Directors, a professional association.
"We
have this public health mentality that all people have to be cured no
matter what the cost, and also let the innovators charge whatever they
want," said Mr. Salo. "Those are fine theories independently, but when
you combine them together in a finite budget environment, it's not
sustainable."
A state spokeswoman said
of Arkansas's Kalydeco policy: "Cost alone was not the determining
factor, but how we will pay for it is something we must consider in
advance as we are a state agency with limited funds."
Doctors,
patient advocates and Vertex said Arkansas is the only state they know
of that has denied Kalydeco to patients who meet the FDA criteria, and
that the state's policy appears to be unique.
Medicaid
benefits cover doctor visits, prescription drugs and other medical
services for more than 70 million low-income people. Unlike Medicare,
the health program for the elderly that is fully funded by the federal
government, Medicaid programs are administered by the states and, on
average, received an estimated 57% of their Medicaid budgets from
federal funds, according to the National Association of State Budget
Officers.
Providing the three people
with Kalydeco, which has an annual wholesale cost of $311,000, would
have a small impact on Arkansas Medicaid's prescription drug spending,
which totaled $351.28 million in 2013 and represented 7.5% of the
state's total Medicaid spending. But the prospect of paying for
similarly priced drugs for cystic fibrosis and other diseases in the
future appears to have been a factor in the state's policy.
Chloe Jones,
a 14-year-old plaintiff in the Arkansas suit, spends several
hours each day undergoing treatment and taking medications. One step
requires her to spend 45 minutes, three times each day in an inflatable
vest that puts pressure on the chest and loosens mucus in the lungs.
William Golden,
medical director of Arkansas Medicaid, said that since Chloe's
lung function was normal at the time her doctors prescribed Kalydeco,
the state couldn't justify approving Kalydeco.
The
state Medicaid agency denied her doctor's request for Kalydeco in June
2012, stating Chloe hadn't met the requirement of taking older
medications for 12 months, according to a letter Medicaid sent her
doctor. The state denied Chloe subsequent appeals in July and September
of 2012, and in August 2013, according to attorneys at Sufian &
Passamano LLP, a Houston law firm representing Chloe.
"They
just don't want to pay for it," Chloe said during an interview in
Little Rock, where she travels for treatment from her home in Walnut
Ridge, Ark. "I feel like they don't care about what's wrong with me,
that I'm not as important as everybody else."
Chloe's
physician,
Dennis Schellhase,
says Chloe has now been taking all of the medications required by
the state for close to a year and the hospital will soon submit another
application for the drug with the state Medicaid program.
(Arkansas
has approved the drug for two patients, Dr. Golden said, but one left
Medicaid and the other hasn't filled the prescription.)
Roughly
27% of Arkansas's population lives in poverty or near poverty, the
second highest rate in the U.S. after Mississippi, according to the most
recent U.S. Census data. Federal funds accounted for more than
two-thirds (70%) of Arkansas's $4.79 billion Medicaid budget in 2013,
according to a state report.
In
exchange for having their drugs covered by Medicaid programs, drug
makers are required to provide rebates that are split between the states
and Washington. Last year, Arkansas received rebates totaling $142.97
million, or about 41% of its total drug spending, of which it kept 22%,
with the rest accruing to the federal government.
Some
expensive new drugs, including Kalydeco, are more effective than older
therapies because they target specific genes that help cause a disease,
doctors say. Companies say the high price tags reflect the costs and
effort of developing the drugs, as well as their benefit to patients.
Rare disease drugs are priced higher to compensate for the lack of a
large patient population, industry officials say.
Kalydeco
is approved in the U.S. for patients aged six years or older with one
of several genetic mutations carried by about 1,100 people in North
America. Globally, about 2,150 people are eligible for the drug, or
about 3% of the 70,000 patients world-wide who have cystic fibrosis.
Patients take the pill twice a day for life.
The
mutations cause the disease by inhibiting the work of a protein that is
responsible for transporting water and salt through the lungs and other
organs. The disease causes thick mucus to build up in the lungs, which
can eventually lead to respiratory failure. There are more than 1,800
genetic mutations that can cause cystic fibrosis.
Kalydeco
is designed to restore function of the protein, which many doctors
expect will halt or substantially diminish lung damage and extend
patients' lifespan, though long-term studies haven't yet been completed
to prove that, said
Robert Giusti,
a cystic-fibrosis specialist at NYU Langone Medical Center in New
York.
"It would be the standard of care
to offer Kalydeco to all patients who are within that mutation panel,"
Dr. Giusti said. Older drugs alleviate symptoms of the disease, mainly
by clearing mucus from the lungs, but don't treat its underlying cause.
Chloe
and the other Arkansas plaintiffs,
Elizabeth West
and
Catherine Kiger,
both 21 years old, also sought financial assistance from Vertex,
which says it provides Kalydeco free to patients without insurance or
whose insurance doesn't cover the drug. The company informed her
hospital nurses that Chloe would have to exhaust the Medicaid coverage
appeals process before Vertex would provide the drug, according to her
lawyers and her doctor. But the company later said the assistance
program specifically excluded Medicaid patients, her doctor said.
"It's been like a yo-yo, and it's devastating every time," said Chloe's mother,
Amie Ledman.
(Kalydeco would be covered by the
Medicare drug program if patients lived into their 60s, but most cystic
fibrosis patients die by the age of 40.)
Vertex
Chief Commercial Officer
Stuart Arbuckle
said the company has never provided free Kalydeco to Medicaid
patients. If Vertex gives Kalydeco free to the Arkansas Medicaid
patients, it could lead other states to establish similar eligibility
criteria, Mr. Arbuckle said. The patient-assistance program, he said,
"isn't there to subsidize Medicaid, which is there to provide medical
care to poor families."
State Medicaid
programs are required to pay for most FDA-approved drugs, regardless of
their price, unless there are equivalent therapies available. For drugs
like Kalydeco that have no equivalent, states can require physicians to
prove the drugs are being used in a medically accepted way.
Amie Ledman, left, prepares medication for her daughter,
Chloe Jones, 14, in a hotel room in downtown Little Rock, Ark.
Karen E. Segrave for The Wall Street Journal
One of the main studies Vertex
conducted to gain marketing approval for Kalydeco tested the drug
against placebo. Patients in one arm of the study took Kalydeco in
addition to standard cystic fibrosis therapies; the other group took
placebo in addition to standard therapies. Patients taking Kalydeco had
an average lung function improvement of 10.1% after about 11 months,
compared with a decline of 0.4% in patients taking placebo, according to
data published in the New England Journal of Medicine in 2011.
Dr.
Golden said the study doesn't prove Kalydeco is more effective because
patients weren't taking hypertonic saline, a salt water mist that is one
of several treatments patients take to help clear mucus from their
lungs. The treatment is recommended by the Cystic Fibrosis Foundation
and is estimated to have a moderate net benefit. The Foundation, a
nonprofit advocacy organization also responsible for issuing treatment
guidelines and accrediting medical facilities, also funds pharmaceutical
research—including to Vertex—and receives royalty payments from Vertex
on sales of Kalydeco.
In a series of
2012 emails obtained by Chloe's attorneys and reviewed by The Wall
Street Journal, Arkansas Medicaid officials discussed Kalydeco's cost.
In an email to colleagues discussing a review board's deliberations
about Kalydeco, a pharmacist named
Pamela Ford
wrote, "the consensus of the physicians on the board was that
none of the prescribers would have a clue that this will cost AR
Medicaid $303,408 per patient per year."
She
also noted in a separate email that Vertex was working on a new product
that would combine Kalydeco with another drug, which could treat the
majority of cystic fibrosis patients. The new therapy would be "likely
even more expensive" and a "budget-breaker!" Ms. Ford wrote. "So we will
be very strict in these reviews knowing it will come back even worse
the next go-round."
Vertex is developing other therapies for cystic fibrosis that it hopes will eventually treat all genetic mutations.
An Arkansas state spokeswoman said Ms. Ford was unavailable to comment due to department policy.
Cystic
fibrosis experts say Arkansas's criteria conflict with standard
treatment approaches for patients with the genetic mutation. In 2012,
Chloe's lung function was relatively stable at about 90% of what would
be expected in a healthy person, but cystic fibrosis is thought to
damage the lungs even when patients don't have symptoms, said Dr.
Schellhase, Chloe's physician. Since 2013, Chloe has been hospitalized
four times for roughly two weeks each stay after her condition worsened,
and her lung function sank as low as 71%, Dr. Schellhase said. Arkansas
Children's Hospital declined to specify the costs of Chloe's
hospitalizations but said the average annual cost of hospital stays is
about $109,000 for a child with cystic fibrosis.
If
Chloe had been given Kalydeco in 2012, "we probably would've avoided
most of the hospitalizations, if not all of them," said Dr. Schellhase,
who doesn't receive any money from Vertex.
Brian O'Sullivan,
a cystic-fibrosis specialist at University of Massachusetts
Medical School, said the consensus among doctors is that all patients
with the genetic defect should receive Kalydeco. Dr. O'Sullivan and
other doctors criticized Vertex's pricing of the drug in 2012, saying
the company was "leveraging pain and suffering into huge financial
gain." But he called Arkansas's policy "unconscionable."
Write to Joseph Walker at joseph.walker@wsj.com
Ristori
G, Romano S, Cannoni S, Visconti A, Tinelli E, Mendozzi L, Cecconi P,
Lanzillo R, Quarantelli M, Buttinelli C, Gasperini C, Frontoni M,
Coarelli G, Caputo D, Bresciamorra V, Vanacore N, Pozzilli C, Salvetti
M.
Neurology. 2014 Jan 7;82(1):41-8. doi: 10.1212/01.wnl.0000438216.93319.ab. Epub 2013 Dec 4.
- PMID:
- 24306002
- [PubMed - indexed for MEDLINE]
SUFIAN & PASSAMANO, L.L.P.
712 Main Street, Suite 2130
Houston, Texas 77002
Telephone: 713-224-1166
Facsimile: 713-224-1161
Perhaps the US needs to study more about cholera or simply have more cases of cholera?
712 Main Street, Suite 2130
Houston, Texas 77002
Telephone: 713-224-1166
Facsimile: 713-224-1161
Perhaps the US needs to study more about cholera or simply have more cases of cholera?
Braz J Med Biol Res. 2014 Feb;47(3):179-91. doi: 10.1590/1414-431X20133063. Epub 2014 Mar 3.
From Escherichia coli heat-stable enterotoxin to mammalian endogenous guanylin hormones.
Abstract
The isolation of heat-stable enterotoxin (STa) from Escherichia coli and cholera
toxin from Vibrio cholerae has increased our knowledge of specific
mechanisms of action that could be used as pharmacological tools to
understand the guanylyl cyclase-C and the adenylyl cyclase enzymatic
systems. These discoveries have also been instrumental in increasing our
understanding of the basic mechanisms that control the electrolyte and
water balance in the gut, kidney, and urinary tracts under normal
conditions and in disease. Herein, we review the evolution of genes of
the guanylin family and STa genes from bacteria to fish and mammals. We
also describe new developments and perspectives regarding these novel
bacterial compounds and peptide hormones that act in electrolyte and
water balance. The available data point toward new therapeutic
perspectives for pathological features such as functional
gastrointestinal disorders associated with constipation, colorectal
cancer, cystic fibrosis,
asthma, hypertension, gastrointestinal barrier function damage
associated with
enteropathy, enteric infection, malnutrition, satiety, food preferences, obesity, metabolic syndrome, and effects on behavior and brain disorders such as attention deficit, hyperactivity disorder, and schizophrenia.
enteropathy, enteric infection, malnutrition, satiety, food preferences, obesity, metabolic syndrome, and effects on behavior and brain disorders such as attention deficit, hyperactivity disorder, and schizophrenia.
 |
|
|
|
|
|
|
||||
|
|
| |||||
No comments:
Post a Comment