The National Organization for Rare Disorders (NORD) says it’s
“disappointed and dismayed” after the House of Representatives voted 227-205
last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform
package.
A similar package before the Senate Finance Committee does
not repeal the credit entirely, but cuts incentives nearly in half by lowering
its value from 50 percent of qualified clinical testing expenses to 27.5
percent.
A statement signed by 91 patient organizations under the
NORD umbrella noted that without the Orphan Drug Tax Credit, new therapies for
rare disease patients could tumble by more than a third.
“We will not stand idly by as Congress deliberates on
diminishing the hope of the 95 percent of individuals with a rare disease still
waiting for their very first treatment,” the petition, released Nov. 20, reads.
“Any proposal that stands in their way to finally obtaining a safe and
effective therapy is unacceptable.”
Dozens of groups ranging from the Alport Syndrome Foundation
and the ALS Association to the Consortium of Multiple Sclerosis Centers, Parent
Project Muscular Dystrophy and the Friedreich’s Ataxia Research Alliance
endorsed the statement.
NORD, a nonprofit organization formed in 1983 — the same
year President Reagan signed the Orphan Drug Act into law — says the tax
incentives this law offers to U.S. pharmaceutical companies are crucial in
spurring the development of new therapies.
The New York Times reported Nov. 8 that the tax credit —
whose elimination could save the government an estimated $54 billion over the
next decade — “has come under scrutiny because critics say that some major
drugmakers have exploited it by obtaining the orphan designation for
billion-dollar blockbuster drugs like Humira for treating rheumatoid arthritis
and Crestor, the cholesterol drug, that were already on the market.”
Paul Melmeyer, director of federal policy at NORD, discusses
2018 legislative priorities at the recent summit.
But a study commissioned by NORD and released Oct. 17 during
the organization’s Rare Diseases & Orphan Products Breakthrough Summit in
Washington, D.C., found that approved drugs for orphan indications accounted
for only $36 billion, or 7.9 percent, of the $460 billion Americans spent on
pharmaceuticals in 2016.
According to the 32-page report, “Orphan Drugs in the United
States: Providing Context for Use and Cost,” the median annual cost for an
orphan drug in 2016 came to $32,880, but the 10 therapies used most by patients
averaged $14,909.
Speaking at the summit, Paul Melmeyer, NORD’s director of
federal policy, said the tax credits enshrined in the 1983 act have led to the
approval of 451 orphan drugs for 590 rare disease indications since its
passage.
“There is still a ton of misinformation out there on what
the Orphan Drug Act does and does not do. If the orphan drug tax credit were
repealed, we’d see one-third fewer drugs going forward, so we’re trying to do
our best to emphasize to Congress how important this tax credit is to our rare
disease community,” he said. “We believe that’ll be one of the most important
roles we’ll be playing over the course of the next 12 months.”
State legislation also of concern
While the Orphan Drug Act has certainly grabbed headlines,
it’s not the only legislative issue on NORD’s radar screen.
“NORD was incredibly active this summer fighting harmful
changes to the Affordable Care Act,” said Tim Boyd, the organization’s director
of state policy. “The changes that were being proposed were very harmful. This
will continue to be a priority for us.”
In fact, various proposals at the state level concern the
rare disease community as well, especially when they reportedly conflict with
guidelines set by the U.S. Food and Drug Administration (FDA).
One example, say NORD officials, is the Massachusetts 1115
Waiver program. This program expands MassHealth — the state Medicaid program —
to low-income families, but rations medicines to children and the elderly.
“We have recently seen a disturbing trend where states are
ignoring what the FDA says about drug safety and effectiveness,” Boyd said.
“This has resulted in rising out-of-pocket costs for drugs, patients getting
substandard treatment, or no treatment at all.”
The 1115 Waiver could “potentially limit access to new and
innovative” FDA-approved therapies made possible through the 21st Century Cures
Act, which Congress approved in December 2016 with widespread bipartisan
support.
“Restricting MassHealth’s drug benefits to a closed
formulary would limit the ability of providers to make the best medical
decisions for the care of their patients, effectively taking the clinical care
decisions away from the doctor and patient, and giving it to the state,” stated
an Oct. 18 letter to Eric Hargan, acting secretary of the U.S. Department of
Health and Human Services. The letter was signed by NORD and more than 100
other advocacy groups.
Other “states to watch” when it comes to the 21st Century
Cures Act, said Boyd, include Minnesota, New Jersey and Oregon.
“We find that many decisions about utilization are made
without allowing patients to weigh in,” he said. “We believe states are making
decisions about who gets a drug and who doesn’t, without consulting experts in
the field about these diseases. This is really disturbing to us.”
Other priorities
Other priorities include increased federal funding for
newborn rare disease screening programs, and expanding insurance coverage for
medical foods.
To that end, Melmeyer said NORD supports the creation of an
Office of Patient Affairs, as well as passage of a Medical Nutrition Equity
Act.
“Currently, insurance coverage for medical nutrition is
awful. We need to cover it. It’s medically necessary,” he said. “We must also
improve access to pre-approval therapies. Right now, we have an expanded access
program which is working for a lot of people, but we think it could work for
even more people.”
Another hot-button issue is so-called “Right To Try”
legislation, which allows terminally ill patients to try therapies that have
passed the FDA’s Phase 1 clinical trial process but are not yet commercially
available. So far, 37 states have passed Right To Try laws; several others are
considering similar legislation.
Power of Rare
Copies of Victoria Jackson’s book, “The Power of Rare,” were
displayed at the NORD summit.
According to the Phoenix-based Goldwater Institute, more
than one million Americans die from terminal illnesses every year, yet fewer
than 3 percent gain access to investigational treatments through clinical trials.
“Compassionate use exceptions are only granted to about
1,000 patients a year,” says a fact sheet on the institute’s website. “Many
patients run out of time before they can qualify for the exemption … Right To
Try laws help patients gain immediate access to the medical treatments they
need before it’s too late.”
Said Melmeyer: “Our goal is to expand access to
investigational therapies for those who do not qualify. But it’s not the FDA
withholding access, it’s the companies.”
FDA Commissioner Scott Gottlieb weighed in on the issue at
the NORD summit.
“We are fully committed to the goals of getting patients
earlier access when they’ve exhausted other options,” said Gottlieb, a keynote
speaker at the summit. “But there are various obstacles. There just isn’t
product available in many cases because of manufacturing issues. Companies are
using batches developed just for the purpose of clinical trials that have to be
rationed, even in a pre-approval setting.”
Martha Rinker, NORD’s vice president of policy, said she
expects two or three hearings on this topic, but Right to Try “is probably not
going to see legislation on the federal level.”
Melmeyer advised the many patient advocates attending NORD’s
event to “get to know the individuals at the FDA” who can help organizations
navigate federal bureaucracy — and to keep pressure on Congress to do what’s
right for the estimated 30 million Americans with rare diseases.
“All the folks who called their congressmen, tweeted or sent
that email, made that video or Facebook post, they were so effective,” he said
of past efforts. “It was the perfect testament to the power of advocacy — and
why we need to be involved.”
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