Winning Spirit Newsletter Summer 2010
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Research Updates
Hemophilia and Healthcare Reform: What You Need to Know
Healthcare reform will occur in stages By David Linney

While national healthcare reform
passed in March, implementing it
will occur in stages. The legislation is
detailed and has many provisions. It is
important for you to understand what
the legislation does and does not do.
The legislation does not create a
national health insurance program.
Rather, it fills the gaps in our current
system, helping to provide insurance
options for roughly 32 million of the
46 million Americans who have no
insurance.
Some of the legislation will go into effect
within six months of the March 2010
enactment, but a large portion will not
be implemented until 2014 or later.
Changes in 2010
Effective in June, 90 days after the
legislation’s enactment:
• Temporary national high-risk pools will
offer insurance for individuals with
pre-existing conditions who have been
uninsured for at least six months.
These risk pools will be operational
until January 1, 2014, when options
through the insurance exchanges
become available.
Effective in September, six months after
the legislation’s enactment:
• Pre-existing condition exclusions for
children will be eliminated in all
health plans.
• Young adults up to age 26 may be
covered as dependents under all
health plans.
• Lifetime limits will be prohibited for
all individual and group plans.
• Prior to 2014, annual limits on
coverage will be restricted as
determined by the US Department of
Health and Human Services.
• Rescissions—or health policy
cancellations usually due to higher use
of benefits and pre-existing
conditions—will be prohibited, except
in cases of fraud.
Changes in 2011
Effective January 1, 2011, insurance
companies will be required to
pay rebates to enrollees if the
company does not spend a minimum
percentage of enrollees’ premium
dollars on healthcare, as opposed to
administrative expenses. For large
group plans the minimum is 85%; for
individual and small group plans the
minimum is 80%.
Changes Beginning in 2014
• Pre-existing condition exclusions for
adults will be eliminated for all health
plans.
• Waiting periods for new policy
coverage to become effective will be
limited to 90 days for all plans.
• Annual limits on new individual and
group plans and existing group plans
will be prohibited.
• Medicaid eligibility will be extended
to all uninsured low-income
individuals younger than age 65,
including adults without dependent
children, who have incomes up to
133% of the federal poverty level
(FPL). Current poverty guidelines are
$14,404 for an individual and $29,326
for a family of four.
• Most citizens and legal residents will
be required to have health insurance
or pay a tax penalty.
• Employers with 50 or more employees
that do not offer insurance may face
a tax penalty if any worker receives
federal subsidies to purchase health
insurance.
• Individuals and small employers will
be eligible to purchase insurance
through state-based American Health
Benefit Exchanges. Individuals with
incomes between 133% and 400% of
the FPL (or $14,404 to $43,320 for
individuals and $29,326 to $88,200
for a family of four) will be eligible
for premium and cost-sharing credits,
also known as subsidies.
Previous article taken from HemAware.org, an online publication of the National
Hemophilia Foundation, 4/10.
Gene Therapy for Hemophilia B Enters into New Phase of
Development
By Molly Polen
A cooperative effort by researchers
from the U.S. and the United Kingdom
(UK) to develop a gene therapy for
hemophilia B has resulted in a new
phase of clinical development.
Netherlands-based Amsterdam Molecular
Therapeutics (AMT), a company that
specializes in gene therapy for different
diseases and disorders, announced in
March that the first patient has been
dosed in a Phase I/II exploratory clinical
trial with a gene therapy product for
hemophilia B. The study includes the
use of AMT’s proprietary gene therapy
technology. The principal investigator
of the trial is Arthur W. Nienhuis, MD, a
faculty member at St. Jude Children’s
Research Hospital in Memphis, TN.
The gene therapy in the trial uses
adeno-associated viruses (AAV), small
viruses that do not cause disease and
produce mild immune responses, as
vectors (delivery vehicles) to introduce a
functioning factor IX (FIX) gene into the
liver cells of subjects with hemophilia
B. The goal of the therapy is to trigger
long-term FIX protein production through
a single administration of the therapy.
This could reduce or eliminate bleeding
episodes in hemophilia B patients. Earlier
pre-clinical studies of FIX gene therapy
were promising, demonstrating the
potential for long-term FIX production.
The seminal work on this therapy was
started more than a decade ago by
St. Jude researchers Andrew Davidoff,
MD, and Amit Nathwani, MD, PhD, now
with the University College London
(UCL). The current trial involves the
collaboration of St. Jude, UCL and a
number of other institutions in the UK
and the U.S.
The purpose of the current trial, which
has been approved by both the UK
Medicines and Healthcare products
Regulatory Agency and the U.S. Food
and Drug Administration, is to measure
the safety and efficacy of different
doses of the therapy. Upon a successful
trial, AMT intends to follow up with
additional clinical studies.
Source: Medical News Today, March 11, 2010
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