Optimal cancer treatment, which can offer cures or extend a
patient’s life by months, years, or even decades, could be
derailed by a lack of supply of critical therapies. Drug shortages,
which first became widespread in the mid-2000s, are becoming an
increasingly serious problem in health care and in oncology
specifically. As of the time of this writing, the U.S. Food and Drug
Administration (FDA) reported 232 drugs in short supply in 2011,
breaking the previous year’s record of 211 drugs in shortage.
The agents in scarce supply tend to be older, generic, injectable drugs
that are the backbone of standard chemotherapy (either alone or in
combination) for common adult and pediatric cancers.
When a drug is unavailable, patients are faced with the disheartening
options of switching to an alternative therapy (which may be more
expensive, more toxic, and/or less effective), receiving treatment at a
different site where the drug is available (which may be much further
from the patient’s home), or—if no alternative
therapy is available—delaying treatment altogether until a
supply of the necessary drug can be found (which may adversely affect
survival).
Cascading
problems for practices, researchers
Oncology care providers also face a significant cost from drug
shortages, and not simply because the next-best therapy may be more
expensive. Practices may have to turn away patients because they do not
have adequate supplies of a treatment. They must also dedicate
significant staff time to tracking down supplies of drugs in shortage
and developing systems for managing or rationing the small stock that
they have, often not knowing when—or if—new
supplies will come in.
In addition to the immediate impact of drug shortages on patients
currently undergoing active treatment, clinical research is also
threatened by the dearth of several agents. More than 300 NCI-funded
clinical trials depend on a drug in short supply, reported Howard K.
Koh, MD, MPH, an Assistant Secretary of Health in the U.S. Department
of Health and Human Services (HHS), in September 2011.
Below, common questions about drug shortages are addressed. ASCO
members are encouraged to request more information or share experiences
and perspectives on the issue by sending an email to
publicpolicy@asco.org or by leaving a comment in the “
DrugShortages: How Is Your Practice Managing?” forum.
What
is causing these drug shortages?
There is no single root cause that makes a drug vulnerable to supply
problems, but rather several underlying causes:
- Drug discontinuation.
Pharmaceutical companies are not required to continue producing a drug,
even if it is needed by many patients. Some companies have chosen to
discontinue the production of less-profitable older generic drugs in
favor of newer more-profitable agents. With production of generic
agents concentrated among a small number of companies, an interruption
in manufacturing at a single location can lead to a serious shortage.
(Read Dr. Peter Yu’s blog on the topic.)
- Manufacturing problems. A
scarcity of raw materials, FDA compliance issues, and production or
shipping delays can lead a manufacturer to slow or temporarily stop its
production of a particular drug.
- Quality issues. The majority
of drugs in shortage are sterile injectables, which are manufactured
with a complex multistep process to ensure their safety and quality.
This yields more opportunity for manufacturing errors or contamination
that render a batch unsafe or unusable. Because of their complexity,
sterile injectables are more expensive to produce, which may lead a
company to focus on manufacturing more-profitable oral drugs.
- Regulatory requirements.
Maintaining or obtaining active approved applications from the FDA to
manufacture older generic drugs may lead companies to shut down
production of particular agents rather than try to fulfill what many
perceive as expensive and time-consuming regulatory requirements.
- Free-market limitations. The
2003 Medicare Modernization Act (MMA) led to greater transparency in
drug pricing and changed the way that most intravenous drugs are
reimbursed by Medicare. Reimbursement is now based on the
drug’s average selling price (ASP) plus 6%, which may make it
difficult for companies to raise the price of a drug even if there is
increased demand—leaving companies little incentive to
increase their production of low-margin drugs.
How
has ASCO been addressing drug shortages?
ASCO has been carefully monitoring drug shortages and has been
particularly active on the issue over the past year.
In January 2011, ASCO, along with the American Society of
Anesthesiologists (ASA), the American Society of Health-System
Pharmacists (ASHP), and the Institute for Safe Medication Practices
(ISMP), issued preliminary recommendations to help ease the
drug-shortage crisis. The recommendations stemmed from the November
2010 Drug Shortages Summit jointly convened by the organizations.
In February 2011, ASCO supported the introduction of the Preserving
Access to Life-Saving Medications Act in the Senate (S. 296, introduced
by Senators Amy Klobuchar [D-Minn.] and Robert Casey [D-Pa.], and
subsequently H.R. 2245, introduced by Representatives Tom Rooney
[R-Fla.] and Diana DeGette [D-Colo.] in June 2011). Although there are
some small differences between the Senate and House versions of the
bill, if passed, this act would require a manufacturer to formally
notify the FDA six months before discontinuing or interrupting the
production of any drug.
In July 2011, representatives from the oncology community, including
ASCO President Michael P. Link, MD, a pharmacist, and a cancer patient
affected by the shortages, discussed the issue at a special Capitol
Hill briefing.
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| | Top: Richard Schilsky, MD, Chair of ASCO’s Government Relations Committee, informs Rep. John Carney on issues impacting the oncology community. Bottom: W. Charles Penley, MD, Chair-Elect of ASCO’s Government Relations Committee, testifies before Congress on the nation’s drug-shortage crisis. Photos by Amanda Narod.
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In September 2011, the ASCO Government Relations Committee sponsored a
reception for Congressional members and staffers to discuss drug
shortages and other legislative issues relevant to cancer care. In
attendance were Representatives John Carney (D-Del.), Brett Guthrie
(R-Ky.), Ron Kind (D-Wis.), and John Barrow (D-Ga.), as well as key
staff from 22 Congressional offices. Later that month, ASCO
representatives expressed the urgent need for policymakers to engage in
serious efforts to solve and prevent cancer drug shortages at a public
workshop held by the FDA.
Also in September, W. Charles Penley, MD, of Tennessee Oncology and
Chair-Elect of ASCO’s Government Relations Committee,
testified at a hearing of the House Energy and Commerce Subcommittee on
Health. He spoke on ASCO’s behalf and also represented his
practice, which has experienced ongoing problems with drugs
in short supply for the past year. Dr. Penley’s
complete testimony is available at ascoaction.asco.org.
“The hearing was scheduled because many government agencies
have been getting reports of drug shortages,” said Dr.
Penley. “We were glad to provide testimony on behalf of our
members and patients because it brought the issue to a much higher
level of
public attention.”
In October 2011, ASCO supported an amendment to the fiscal year 2012
Senate Agriculture Appropriations bill that would give the FDA $10
million to address drug shortages. The amendment, filed by Senator
Robert Casey (D-Pa.) gives the Office of the Commissioner of the FDA
increased funding to “remedy the current drug-shortage
crisis and to prevent future shortages.”
On October 31, President Barack Obama issued an executive order,
supported by ASCO, directing the FDA to curb drug shortages by
broadening its reporting of potential shortages, expediting regulatory
reviews, and working with the Department of Justice to investigate
illegal price gouging or stockpiling. President Obama also offered
support for the bipartisan House and Senate legislation that would
require drug manufacturers to give the FDA prior notice of a shortage.
What
solutions have been proposed?
Numerous solutions have been suggested to address the drug-shortage
crisis; a true resolution of the issue will likely require several
elements in combination. Some of the proposed solutions include:
- offering financial
incentives for manufacturers to continue producing or increase
production of generic sterile injectables, such as tax breaks,
stipends, or market exclusivity;
- amending the 2003 MMA to
increase reimbursement and eliminate de facto price controls on generic
drugs;
- forming a nonprofit
organization that will import and manufacture drugs to maintain a
steady supply;
- creating a national
stockpile of cancer medicines, similar to the existing stockpile of
antibiotics at the CDC;
- decreasing administrative
and regulatory barriers to encourage manufacturers to enter or remain
in the generic market; and
- empowering the FDA with
increased authority to manage shortages.
What
drugs are currently in short supply?
The FDA and the ASHP maintain current lists of drugs in short supply on
their respective websites:
In addition to chemotherapeutic agents, drugs in short supply include
surgical anesthetics, emergency medications, and intravenous
electrolytes.
My
practice/institution is experiencing a drug shortage. What can I do?
To report a shortage situation or supply issue to the FDA, send an
email to
drugshortages@fda.hhs.gov. You can send a report to ASHP using
an online form at
ashp.org/drugshortages/report/. Using the ASHP form
generates a simultaneous email to ASHP, the FDA, and the University of
Utah Drug Information Service, which tracks shortages for ASHP.
You’re also encouraged to keep ASCO apprised of any
drug-shortage situations in your community by contacting
publicpolicy@asco.org. Share your
experience with drug shortages, recommend solutions, and confer with
colleagues in the “
Drug Shortages: How is Your PracticeManaging?” forum. You can find guidance
on how to contact your Congressional representative about legislation
related to oncology drug shortages by visiting ASCO’s ACT
Network (
capwiz.com/asco).
Dr. Penley recommends that ASCO members work to raise awareness about
drug shortages at the local level. “If a drug shortage arises
in your community and it’s having a direct, personal impact
on patients, speak with your local media outlets to continue to keep
the level of interest high. We have to keep this issue in the forefront
for policymakers,” he said.
What
is the drug shortage “gray market”?
In the midst of the drug-shortage crisis, there have been reports that
some secondary distributors are significantly marking up the prices of
agents in short supply—up to 20, 50, or even 80 times the
typical contract price of the drug. This price-gouging parallel market
is being referred to as the “gray market.”
Representative Elijah E. Cummings (D-Md.), ranking member of the House
Committee on Oversight and Government Reform, is leading an
investigation into five pharmaceutical distributors reported to be
engaged in gray-market drug sales.
“Price gouging for drugs that treat cancer in children is
simply unconscionable,” Mr. Cummings said in an October 2011
press release. “We want to know where these companies are
getting these drugs, and how much they are making in
profits.”
If your practice or institution is approached by a drug distributor
selling scarce drugs at an exorbitant mark-up, you can send a report to
the Congressional “Gray Market” Tipline at
forms.house.gov/oversight_majority/webforms/grey_market.html. Find more information on the Congressional
gray market investigation at
democrats.oversight.house.gov.
What
are ASCO leaders and members saying about drug shortages?
ASCO President Michael P. Link, MD, has been the Society’s
voice in speaking with numerous media outlets about the serious issue
of drug shortages:
“Something has to be done soon in order to try to alleviate
this problem. Right now we’re already seeing patient care
suffering.”
—“
Authorities Perplexed by Drug Shortage Spike,”
Reuters,
October 2011
“These are drugs that have actually resulted in cures and are
life-saving. I’m a pediatric oncologist...and some of these
drugs are really the whole therapy. We can now cure almost 80% of
children with cancer and without these drugs, our hands are tied. This
is really a crisis for us.”
—“
On Point with Tom Ashbrook,” National
Public Radio (NPR), October 2011
“[Leukemia is] the type of cancer that doesn’t have
time to wait [for a renewed supply of cytarabine].”
—“
Coming Up Short,”
The Economist,
September 2011
“These shortages are just killing us. These drugs save lives,
and it’s unconscionable that medicines that cost a couple of
bucks a vial are unavailable.”
—“
U.S. Scrambling to Ease Shortage of Vital Medicine,”
New York Times,
August 2011
“You can imagine the conversation and I’m sure
they’re going on all over—doctors have to tell
their patients or their patients’ parents that we
can’t give them the proven drug because we don’t
have it.”
—“
U.S. Cancer Drugs Shortage Has Doctors Scrambling,”
Reuters,
June 2011
Robert J. Mayer, MD, of the Dana-Farber Cancer Institute and an ASCO
Past President, was quoted along with Dr. Link in the June 2011
Reuters
article. On the issue of drug substitutions and alternative therapies,
he offered the analogy, “One could say that substituting
Pepsi for Coca-Cola doesn’t make a difference. Maybe it does
and maybe it doesn’t. But more often it might be substituting
7-UP for Coca-Cola, and that might make a difference.”
Ezekiel J. Emanuel, MD, PhD, of the University of Pennsylvania,
contributed
an op-ed to the New York Times
in August 2011, in which he wrote: “You don’t have
to be a cynical capitalist to see that the long-term solution is to
make the production of generic cancer drugs more profitable. Most of
Europe, where brand-name drugs are cheaper than in the United States,
while generics are slightly more expensive, has no shortage of these
cancer drugs....Scare-mongering about death panels and health care
reform has diverted attention from real issues in our health care
system. Shortages in curative cancer treatments are completely
unacceptable.”
Where
can I find more information on oncology drug shortages?
Visit the following websites to find more information on drug
shortages:
LIVESTRONG Blog Considers, “What If?”
Craig R. Nichols, MD, of Virginia Mason Medical Center, Seattle, an ASCO member and a LIVESTRONG Board of Directors member, contributed a guest blog post on livestrong.org, considering how his most famous patient—seven-time Tour de France winner, philanthropist, and testicular cancer survivor Lance Armstrong—would have fared if he had been diagnosed during the ongoing drug shortages.
Dr. Nichols wrote: “I’m often asked whether we gave Lance some sort of mythical special treatment or molecular wizardry reserved for celebrities and politicians. The answer is no. His recovery was due to tried-and-true treatments and medications like cisplatin, a drug developed in the mid-1960s. Today, cisplatin remains the cornerstone of cure for testicular cancer and is highly effective against many other malignancies. And this life-saving drug is on the Food and Drug Administration’s drug shortage list. . . . Were Lance diagnosed today, the odds would be stacked against him.”
Read Dr. Nichols’ complete post, “
If Diagnosed Today, How Would America’s Most Famous Cancer Survivor Fare?".