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 The Dutch hospital has been offering it to patients for more than a
decade at 16,000 euros ($18,000) for one course of treatments. Drug
firm Novartis, which in 2018 acquired rights to sell it in Europe,
is asking more than five times that for its proprietary version,
Lutathera.
Thomasa is part of a protest against high drug prices launched by an
unlikely group of rebels: Dutch pharmacies.
Three - Erasmus, Amsterdam's University Medical Center (UMC) and the
Transvaal Pharmacy in The Hague - have vowed to bypass drug company
products and make treatments for a handful of rare diseases
themselves, exercising their right to "compound" medicines.
The Dutch market is small, with only hundreds of patients for the
diseases in question. But the dispute is part of a growing global
backlash against high drug prices, from the United States and Canada
to Japan, and campaigners said it was being closely watched by
health experts elsewhere.
"People with rare diseases are dependent on medicines that are so
expensive that they can't afford them, when they could be offered
for a much lower price," said UMC pharmacist Marleen Kemper.
"The pharmaceutical industry plays an important role in developing
good products, but we think it's not fair if these firms make big
money off these patients."
Compounding is the ancient practice of preparing medicines for
individual patients. Pharmacists are trained to compound, though
nowadays most medicines are made by industrial producers.
Drug companies have raised concerns about the safety of compounded
medicines that have not been approved by European regulators. But
the specialized compounding pharmacies, which have on-site
laboratories, have been backed by the Dutch government as part of
efforts to tame rapidly rising medicine costs.
UMC received a 5-million-euro grant last month to expand its
compounding program. It plans to use the money to set up a center to
swap know-how with pharmacies at home and abroad.
The drug industry, including Swiss giant Novartis and Italian-based
Leadiant, argue Dutch national supervision of pharmacy preparations
is not comparable to the European standards that apply to
pharmaceutical companies.
A spokesman for Leadiant said when Dutch patients suffering from
rare diseases were exposed to medicines that had not been subject to
the stringent testing required of authorized drugs, it could have
"significant repercussions" for their health.
Novartis CEO Vas Narasimhan told Reuters the price his company asks
for Lutathera is justified. He said it was impossible to compare the
treatment made at Erasmus with Lutathera, which received approval
from EU watchdogs after proving it worked in its exact formulation.
The potential perils of compounding were shown in 2012 in the United
States, where the market is more developed. A meningitis outbreak
that killed more than 60 people was traced to contamination in
steroid injections made at the New England Compounding Center, which
was then overseen by Massachusetts state regulators.
GLOBAL BACKLASH
The worldwide push against high drug prices has seen the Trump
administration in the United States declare bringing down
prescription prices a top priority.
In Canada, industry lobbying groups have offered concessions as they
seek to avert a government crackdown. And in Japan, regulators are
considering a cost-effectiveness test as a means of capping prices.
The Dutch pharmacies, whose production is on a small scale,
acknowledge they may face legal challenges from the drug industry.
However, such a case could set a precedent for other European
countries.
The Netherlands has an influential voice in Europe as the seat of
the European Medicines Agency, which scrutinizes and approves drugs
for all EU countries. The EMA has moved to Amsterdam as a result of
the Brexit vote.
Pharmacies retain the right under European and American law to
prepare medicines for individual patients. Common examples including
making lower dosage versions for children or liquid versions for
people with difficulty swallowing.
However, in the United States, for-profit compounding pharmacies
have tested the limits of what they are allowed to do in recent
years, including mixing medicines in large quantities. In some
cases, that has prompted legal conflicts with drugmakers.
Official scrutiny of the practice increased after the 2012 deaths in
Massachusetts, which led to U.S. lawmakers requiring bulk
compounding facilities to register with the Food and Drug
Administration (FDA) and meet quality and labeling standards - still
not as stringent as full FDA drug approval.
Els Torreele, who oversees the Doctors Without Borders Access
Campaign, applauded the Dutch pharmacies' move. She said the
developments in the Netherlands were being followed by medical
experts and politicians in other countries, which could follow a
similar path.
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Torreele said big pharmaceutical companies sometimes lost sight of
the fact that rules granting them exclusive manufacturing rights for
new drugs were intended to foster innovation, not guarantee large
profits.
"If (drug companies) can play a role in providing medicines at an
affordable price, fine, but when they become a barrier, then there's
a problem," she said.
Dutch Medical Care Minister Bruno Bruins attended the opening of a
new compounding laboratory at the Transvaal pharmacy in January. His
government has vowed to curb drug price increases, a pledge welcomed
by the public.
Costs for new medicines in the Netherlands rose 9 percent in 2018,
according to a report by the country's Heath Authority, continuing a
multi-year trend.
"We need drugmakers like Novartis for medicines now and the
medicines to come," Bruins said. "We really can't do without them.
And yet we want them for a reasonable price."
NOVARTIS CEO TROUBLED
The first two drugs targeted by Dutch pharmacies are Novartis's
Lutathera and a drug called CDCA, registered in Europe by Leadiant.
High prices are not unusual for new rare disease drugs, as companies
must recoup development costs from a relatively small group of
patients.
Novartis charges 92,000 euros for a one-off course of four Lutathera
injections. But while the drug is innovative, its development costs
were relatively small and cannot justify the price tag, said Erasmus
MC Chief Executive Ernst Kuipers.
Main development costs amounted to about 40 million euros, including
15 million euros in public funding, according to Erasmus - where
most of the testing took place - and a review published in the Dutch
Journal of Medicine.
Novartis bought the rights to Lutathera with a $3.9-billion-euro
takeover of French company AAA last year.
The drug had just been approved by the EMA as a treatment for
certain cases of endocrine cancers, which affect one in 27,000
people. Although Lutathera is not a cure, it often gives patients
several extra years of healthy life.
Novartis boss Narasimhan said he was worried by developments in the
Netherlands.
"The Netherlands' characterization that the local medicine that is
made in the hospitals is the same as what we've done from a
regulatory, full-development standpoint, particularly given (the
Dutch) will house the European Medicines Agency, I think is
troubling," he told Reuters in an interview.
GENETIC DISEASE
In the case of CDCA, a decades-old gallstone medicine, the drug used
to cost 30,000-40,000 euros for a year's worth of pills. Since the
1990s it has been used only as an off-label treatment for a rare
genetic disease known as CTX.
That changed when Leadiant formally registered the drug as a
treatment for CTX with the EMA in 2017, and began asking 163,000
euros.
Amsterdam's UMC, which treats all 60 known CTX patients in the
Netherlands, compounded CDCA itself from April-August 2018. Then the
Dutch Health Inspectorate found an impurity in its formulation after
Leadiant requested a lab inspection.
The inspectorate "rightly protected patient safety", Leadiant told
Reuters.
UMC says it intends to resume production of CDCA once it can
guarantee it meets all required standards.
There are only 200 people in Europe known to have CTX, and only
several hundred cases have been recorded worldwide. But scientists
say it goes undiagnosed in most cases, and the true number of
sufferers in the United States alone could be 10,000.
With early and lifelong treatment with CDCA, the effects of CTX,
which include mental deficiencies and eventually death, are
preventable.
Leadiant defends its pricing, saying it bore significant costs by
undertaking a human study demonstrating the drug's effectiveness,
which had never been done before.
Battle lines are drawn.
Bruins told parliament last month that he planned to "confront"
drugmakers and demand transparency on development costs.
"I can and will be having talks with a lot of individual companies,"
he said. "And I'll say, of course I want to keep your innovative
power, but temper your prices."
(Reporting by Toby Sterling, John Miller and Elyse Tanouye; Editing
by Pravin Char)
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