[Ip-health] AstraZeneca Pushes to Protect Crestor From Generic Competition

PERFECT, Chase cperfect at coalitionplus.org
Mon Jul 11 07:20:56 PDT 2016

Thanks, Thiru! A quick technical question for the audience: does the Orphan
Drug Act consider pediatric and adult populations as entirely
distinct--i.e. for the purposes of calculating the number of Americans with
a given condition at a given time? For example, if 175k adult Americans had
a given condition and 50k in the pediatric population had the condition,
would that indication still qualify for orphan status? Just want to clarify
that this loophole does indeed exist; that is, a loophole to the criterion
that the condition in question must affect less than 200,000 Americans at
the time of application.

With regards to defining a pediatric population--a matter that also has
obvious relevance for obtaining a general exclusivity extension via
performance of pediatric trials--I found the following excerpt on the FDA
site and thought I would pass it along:

"A sponsor must address all of the pediatric age groups in which the drug
is appropriate for use.* FDA considers pediatric patients to be those aged
birth up to the 16th birthday. *Age categories are set as: neonates (0-1
month), infants (1 month to 2 years); children (2 years to 12 years);
adolescents (12 years to 16 years). Sponsors must address the entire age
range but need not necessarily use these specific age categories. If a
sponsor bases its studies on characteristics other than ages, such as
physiological development, the sponsor should support its categories with
scientific, developmental, compliance, or ethical reasons."

On Mon, Jul 11, 2016 at 11:12 AM, Thiru Balasubramaniam <thiru at keionline.org
> wrote:

> http://www.nytimes.com/2016/06/28/business/astrazeneca-pushes-to-protect-crestor-from-generic-competition.html?_r=0&mtrref=www.facebook.com
> AstraZeneca Pushes to Protect Crestor From Generic Competition
> JUNE 27, 2016
> No more than a few hundred American children have a rare disease
> characterized by ultrahigh levels of bad cholesterol.
> Yet to the giant drug maker AstraZeneca, this small group could be worth
> billions of dollars.
> The company is making a bold attempt to fend off impending generic
> competition to its best-selling drug, the anti-cholesterol pill Crestor, by
> getting it approved to treat the rare disease. In an unusual legal
> argument, the company says Crestor is entitled to seven years of additional
> market exclusivity under the Orphan Drug Act, a three-decade-old law that
> encourages pharmaceutical companies to develop treatments for rare
> diseases.
> Critics say AstraZeneca is trying to abuse the law, since the overwhelming
> use of Crestor is for treating adults with high cholesterol, not children
> with the rare disease.
> “This represents a deviation from the intent of the Orphan Drug Act,” said
> Dr. Martin A. Makary, a professor of health policy at Johns Hopkins Medical
> School. “It is now being used to dominate the market with retrofitted
> indications.”
> Crestor, a cholesterol-lowering statin, was prescribed 20.3 million times
> in the United States last year, the second-most of any brand-name drug
> after Synthroid, a thyroid medicine, according to the prescription tracker
> IMS Health. The drug, also known as rosuvastatin calcium, is scheduled to
> lose patent protection on July 8, potentially exposing it to an onslaught
> of generic competition.
> The introduction of generics would weigh heavily on AstraZeneca’s bottom
> line. Crestor is the company’s best-selling drug, accounting for $5 billion
> of its $23.6 billion in product sales last year. About $2.8 billion in
> sales were in the United States, where the retail price is about $260 a
> month, according to GoodRx.com.
> The chain of events leading up to the current controversy began in late
> May, when AstraZeneca won approval of Crestor to treat children with the
> rare disease, homozygousfamilial hypercholesterolemia, or HoFH. That
> approval gave Crestor market exclusivity for seven years, though it applies
> only to that disease.
> One implication is that detailed prescribing information about using the
> drug for the rare disease cannot be included on the label of generic
> versions for seven years. However, the Food and Drug Administration can in
> theory allow generics on the market for other uses, such as treating high
> cholesterol in adults.
> Yet AstraZeneca immediately petitioned the F.D.A., arguing that if the
> correct dose for children with HoFH could not be on the generic label, then
> it would be illegal and dangerous to approve any generic versions for any
> use at all. That is because doctors might still prescribe the generic for
> children with HoFH and choose the wrong dose, posing “substantial safety
> and efficacy risks.”
> Michele Meixell, a spokeswoman for AstraZeneca, said the company pursued
> the new use of Crestor as “part of our standard practice to address unmet
> needs.” She said the Orphan Drug Act “provides exclusivity as an incentive
> for pharmaceutical companies to develop products to help address the needs
> of an underserved patient population.”
> It is far from certain that AstraZeneca’s argument will succeed, and it is
> not known when the F.D.A. will decide. One response might be for the agency
> to approve generic versions of Crestor on schedule, on July 8.
> Spectrum Pharmaceuticals lost a lawsuit this month after trying in a
> similar way to protect its cancer drug Fusilev from generic competition.
> Otsuka Pharmaceutical recently tried a similar tactic to shield its
> big-selling antipsychotic, Abilify, from generic competition by getting it
> approved for a rare disease. The F.D.A. approved generics in April and its
> decision was upheld in court.
> Perhaps the most famous case was when Bristol-Myers Squibb tried for three
> extra years of protection from generics for Glucophage, its big-selling
> drug for adult-onsetdiabetes, by getting it approved to treat the
> relatively few children with that disease. Outraged members of Congress,
> calling the move an abuse of the system, amended the law in 2002 to
> eliminate what came to be called the Glucophage loophole.
> But Otsuka, in its recent court case, argued that lawmakers at that time
> did not explicitly mention orphan exclusivity as a loophole to be closed.
> The United States District judge hearing the case in Maryland, George J.
> Hazel, dismissed that argument.
> “It would defy logic,” he wrote, “to believe that in enacting this measure
> to prevent a three-year exclusivity from becoming a ‘fundamental abuse of
> the system’ that harmed consumers, Congress nonetheless intended to permit
> the seven-year exclusivity Otsuka seeks here.”
> In its petition to the F.D.A., AstraZeneca argues that the F.D.A. was
> legally incorrect in the Otsuka case.
> Kurt R. Karst, a director of the Washington law firm Hyman, Phelps &
> McNamara and principal author of the FDA Law Blog said, “Given the Otsuka
> precedent, it’s going to be an uphill battle” for AstraZeneca. He predicted
> that the company would sue the F.D.A. if its request were denied.
> While AstraZeneca has been studying use of Crestor for pediatric conditions
> for some years, it began a clinical trial for children 6 to 17 years old
> with HoFH only in 2014. About 300 to 1,000 people in the United States have
> the disease, which involves two deleterious mutations, one from each
> parent, said Katherine Wilemon, president of the FH Foundation, an advocacy
> group. She said some people with the disease suffer heart attacks when they
> are only 5 years old.
> Michelle Watts of Hagerstown, Md., said that she and her husband, Jason,
> both have high cholesterol, but, “We had no idea that we could both carry a
> gene and there’s the possibility of us giving it to our children.”
> Their 7-year-old daughter, Avery, has HoFH and already has some narrowing
> of the arteries. She takes a generic statin and Zetia, another
> anti-cholesterol pill. Every two weeks, the family drives three hours each
> way for a treatment similar to dialysis, in which Avery’s blood is run
> through a machine that cleanses it of cholesterol.
> Mr. and Ms. Watts, who both use Crestor to treat their own high
> cholesterol, declined to comment on AstraZeneca’s move, saying they did not
> know enough about it.
> AstraZeneca’s trial involved only 14 children and tested a single dose of
> Crestor, according to the company’s petition. After six weeks of treatment,
> those who took Crestor had a bigger decline in LDL, or bad cholesterol,
> than those receiving a placebo. This result was expected, in that statins
> like Crestor are already a cornerstone of treatment for such children.
> Dr. Sidney M. Wolfe, founder and senior adviser of the Public Citizen
> health research group, said that if AstraZeneca was interested in that rare
> disease, “They could have done it a long time ago, not right before
> midnight when it is about to turn into a pumpkin.”
> One generic version of Crestor, sold by Allergan, reached the market in May
> under a patent litigation settlement with AstraZeneca. But drug prices do
> not typically plummet until there multiple generics are on the market.
> The issue of using orphan diseases to give drugs extended protection from
> generic competition is playing out on a bigger stage as well. A broad
> pharmaceutical policy bill passed by the House of Representatives last
> year, called the 21st Century Cures Act, would give six extra months of
> protection from generic competition if an already marketed drug gets
> approved for an orphan disease.
> Max G. Bronstein, a senior director at the EveryLife Foundation for Rare
> Diseases, an advocacy group, said the measure was backed by dozens of
> patient groups because “repurposing” existing drugs is a quicker and less
> costly way to get medicines approved for rare diseases than is developing
> totally new drugs.
> But Public Citizen predicted that the provision could add up to $12 billion
> to the nation’s drug bill over 10 years because “cheaper generic versions
> of drugs will be inaccessible to patients with much more common, nonorphan
> diseases for an additional six months.”
> The measure has not been passed by the Senate. Advocates hope it will be
> added as an amendment to the Senate counterpart of the broader legislation.
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Chase Perfect
Access to Medicines Policy Officer
HIV/HCV Drug Affordability Project
Coalition Plus (Paris, France)

Skype ID: cperfect1984
Telephone: +1 919 801 3115

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