By Matthew Perrone, AP Health Writer
Thursday, January 26, 2012
WASHINGTON
(AP)—The pharmaceutical industry won approval to market a record number
of new drugs for rare diseases last year, as a combination of
scientific innovation and business opportunity spurred new treatments
for diseases long-ignored by drug companies.
Drug
companies are increasingly taking advantage of the commercial benefits
of developing so-called orphan drugs, which include extra patent
protections, higher pricing and a streamlined review process by FDA.
Among the innovative treatments approved in the past year were the first
new drug for lupus in 50 years and the first new drug for Hodgkin's
lymphoma in 30 years.
But
the focus on specialty drugs has put pressure on the U.S. government to
ramp up its own spending on vaccines, antibiotics and drugs for more
widespread health threats, which are less profitable for companies.
Since
2006, government spending on research for familiar diseases like staph
infections, smallpox and botulism has increased more than 660 percent,
from $54 million to $415 million last year
"Many
of these are everyday, general diseases that we thought we had
conquered decades ago, but we've seen some of them pop up again," said
Dr. Robin Robinson, director of the Biomedical Advanced Research and
Development Authority, which is tasked with acquiring vaccines, drugs
and other necessities for public health emergencies.
Since
2005, BARDA has awarded $3.5 billion to outside companies to encourage
research and production of antibiotics, flu vaccines and other products
that are seen as less profitable than specialty drugs.
"We
have pushed the envelope more toward diminishing the risk for companies
so that they'll be more interested in getting involved with us and
developing things like vaccines and antivirals," said Dr. Anthony Fauci,
infectious diseases chief at the National Institutes of Health, which
funds research into bird flu, tuberculosis and other potential
pandemics. The government's role in developing new therapies goes beyond
awarding contracts and includes offering assistance in designing trials
and recruiting test subjects.
The
need for such assistance stems in part from a new focus among
pharmaceutical companies on drugs for rare diseases or unusual strains
of common diseases.
Eleven
of the 30 new drugs approved last year, or 37%, were for rare medical
conditions, the highest percentage on record since the FDA began
offering incentives to develop such therapies, known as orphan drugs,
about 30 years ago. Additionally, nearly half of the 30 drugs were
cleared under FDA's "fast track" program reserved for drugs that fill an
unmet medical need.
"The
companies are saying 'this is actually a viable model.' Whereas back in
the nineties they were skeptical, now they seem convinced," said Mark
Schoenebaum, an analyst with International Strategy & Investment.
Analysts
credit scientific advances and looming patent expirations with the
spate of innovative products. Drugs worth a mammoth $255 billion in
global annual sales are set to go off patent before 2016, according to
EvaluatePharma Ltd., a London research firm.
The
pharmaceutical industry reached its peak of profitability in the 1990s
with heavily marketed drugs for common afflictions, like AstraZeneca
PLC's Nexium pill for heart burn and Pfizer Inc.'s Lipitor for high
cholesterol. In the last decade drugmakers managed to extend the patents
on those drugs by tweaking their formulations, resulting in so-called
'follow-on' drugs. But with most of those products on the cusp of losing
patent protection, drugmakers have finally been forced to innovate,
often turning to hard-to-treat diseases for which there are few existing
therapies.
The
FDA grants companies seven years of exclusive, competition-free
marketing for each newly approved orphan drug, as well as tax breaks on
the costs of developing the drugs. Orphan drugs also typically command
much higher prices than other drugs. Last year French drugmaker Sanofi
paid $20 billion to acquire specialty drugmaker Genzyme, whose products
range from $100,000 to $300,000 for one year's supply.
One
side effect of the focus on developing drugs for rare diseases is
increased investment by the government to spur research into more common
public health threats with the potential to cause mass outbreaks of
illness. One such threat comes from so-called superbugs, or bacteria
that have grown resistant to antibiotic drugs.
Robinson
says government support is needed to spur antibiotic development
because of how sparingly the products are used in medical practice.
After decades of routine use, many first-generation antibiotics like
penicillin are no longer effective against common bacterial strains,
such as the staphylococcus aureaus, which causes staph infections.
Physicians are encouraged to use newer antibiotics only in critical
situations so that superbugs have less chance to build a resistance to
them. As a result, drugmakers do not see a large commercial market for
new antibiotics. Now the federal government is providing an incentive.
BARDA
has awarded a series of contracts to encourage development of new
antibiotics that can be stockpiled for use in a natural outbreak or
during a bioterrorism attack.
- The agency has allocated up to $64 million to Achaogen, a San Francisco
startup, for development of a new antibiotic against tularemia, a
bacterium that can cause pneumonia and urinary tract infections. Public
health officials are especially focused on Tularemia because it could
also be used in a potential bioterrorism attack. Robinson says the
contract is an example a new strategy of encouraging companies to
produce therapies with dual uses: as federal preparatory measures and as
commercial medical products.
- Achaogen
has received $155 million in research contracts and has several
antibiotics in early and mid-stage, though none are currently available
for sale.
- Under a $38.5 million contract awarded in September, BARDA will help
GlaxoSmithKline PLC test an experimental antibiotic against both
bioterrorism agents and infections like hospital-acquired pneumonia.
The
U.S. government has used a similar pump priming strategy to encourage
investment in flu vaccines. The Department of Health and Human Services
wants to be able to provide enough vaccine for the entire U.S.
population within six months of a flu pandemic. To meet that goal the
government has tried to boost vaccine production by encouraging more
Americans to get the standard flu vaccine each year. The government's
hope is that by making the shots routine for more Americans, companies
will invest in larger vaccine facilities that can ramp up production in
the event of a pandemic.
Last
month Swiss drugmaker Novartis AG opened the first U.S. vaccine
facility equipped with cell culture technology, a faster method for
producing vaccines than the traditional technique using chicken eggs.
The U.S. government provided half of the $1 billion investment for the
facility, as part of its preparations for a potential flu pandemic.
SOURCE: The Associated Press
