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FDA, HIV/AIDS, In the News

FDA panel recommends use of new cost-effective tool to curb AIDS epidemic

In a very promising step forward, a U.S. Food and Drug Administration panel has taken the unusual step of recommending approval of the first once-a-day pill to prevent HIV. The panel suggested the pill be prescribed for people at high-risk of infection, such as gay men with multiple sex partners. The hope is to reduce the number of new infections in the United States, which reached 56,000 last year (in a talk last month at Stanford, Anthony Fauci, MD, head of NIAID, called this number “embarrassing”).

The move comes on the heels of a Stanford study (subscription required) that found use of the pill, marketed as Truvada, could be cost-effective if targeted at these high-risk groups. The Stanford study, published last month, found that if the pill were prescribed to just 20 percent of high-risk gay men – those with five or more sexual partners a year – some 41,000 new infections could be prevented over 20 years at a cost of about $16.6 billion.

“Adopting (the pill) for men who have sex with men at high risk of acquiring HIV is an investment with good value that does not break the bank,” Jessie Juusola,  a PhD candidate and the study’s lead author said at the time.

A landmark trial published in 2010 found the pill could reduce an individual’s risk of infection by 44 percent when taken daily. Patients who religiously followed the daily regimen reduced their risk to an even greater extent – by 73 percent, the trial results showed.

Scientists caution, however, that patients still need to be monitored for side-effects from the pill, which can affect the kidneys. Moreover, they need to be counseled to continue to use condoms and other protective measures, as the pill isn’t foolproof.

Previously: Anthony Fauci: End of AIDS pandemic in sight, Preventing HIV with daily drug is costly but useful and Treat patients early to stop HIV spread, study finds

Clinical Trials, FDA, Health Policy, Stanford News

New ideas to help FDA discover drug risks

The U.S. Food and Drug Administration sometimes finds out that a drug it has approved for the market has dire and unanticipated side effects. A report released today by the Institute of Medicine suggests steps to help the agency catch such problems earlier in the process.

A key point is for the FDA to use more vigorously its authority to order additional studies after a drug is on the market. “Right now many studies are commissioned mainly when there’s a fire,” said Stanford Professor Steven Goodman, MD, PhD, a co-chairman of the Institute of Medicine (IOM) committee that produced the report, in an interview with me last week. “We think it’s better to initiate them when there’s just smoke.” Goodman is the medical school’s associate dean for clinical and translational research and a professor of medicine and of health research and policy.

The report comes as Congress is considering legislation reauthorizing the user fees that help to fund the FDA and is weighing ways to improve the agency’s drug-approval and tracking systems. Recent situations with the antidiabetes drug Avandia, the pain-reliever Vioxx and the cholesterol-reducing drug Crestor underscore the need to adopt new safety measures. The report proposes some factors that could indicate the need for a post-approval study. According to a release from the IOM:

[The report] identified some circumstances in which a product’s benefits or risks are particularly uncertain, including “first in class” drugs that have been approved based on surrogate endpoints used previously for other drug classes, and drugs for which several endpoints provide conflicting evidence about risk, such as an anti-hypertensive drug that lowers blood pressure but increases weight. In such cases, the committee recommended that FDA require safety research after approval or provide a public rationale for why it is not necessary. Early initiation of such studies could limit the harm done by drugs with risks that are later found to be unacceptable and avoid crises in which the agency is faced with few good options, the committee said.

Addiction, FDA, Health Policy, In the News

What’s being done about the way tobacco companies market and manufacture products

What's being done about the way tobacco companies market and manufacture products

A week ago at Med School 101, an annual Stanford event for area high-schoolers, I sat in on a session by tobacco expert Robert Jackler, MD. The Edward C. and Amy H. Sewall Professor in Otorhinolaryngology, Jackler serves as principal investigator of Stanford Research into the Impact of Tobacco Advertising. His session, “Get ‘em young and train ‘em right: How the tobacco industry targets teens,” detailed ways advertising transforms healthy teens into lifelong smokers.

Jackler took an historical view of the gimmicks employed in print and television ads since the mid-twentieth century. (The one I found most disturbingly catchy was this 1952 Old Gold commercial featuring tap-dancing cigarette cartons in what appears to be a mother-daughter duet.) Besides equating smoking with fun and obscuring the real dangers it poses, tobacco companies have worked around bans on flavored cigarettes by sweetening or spicing up various forms of smokeless tobacco, Jackler pointed out.

Fresh from my Med School 101 primer, I was interested to read today about the latest step by the Food and Drug Administration to fight the tobacco epidemic and “prevent misleading marketing about the risks associated with tobacco products.” An FDA release announced its two new guidance documents:

The first document provides guidance on how companies will comply with the requirement to report on the quantities of potentially harmful chemicals in tobacco products. The second document provides guidance to companies that seek to advertise or market a tobacco product as less harmful or associated with reducing the risk of tobacco-related disease.

“We are  forging new territory to ensure that tobacco companies provide accurate information and do not mislead American consumers,” said FDA Commissioner Margaret Hamburg, M.D. “We are committed to stopping such practices that may cause people to start or continue using tobacco products that could lead to preventable disease and death.”

An Associated Press article in USA Today provides some more background on the second guidance:

The FDA’s handling of modified-risk products has been highly anticipated by both the public health community and bigger tobacco companies, which are looking for new products to sell as they face declining cigarette demand due to tax increases, health concerns, smoking bans and social stigma.

Previously: How have U.S. tobacco regulations affected smokers?, Study shows anti-tobacco programs targeting adults also curb teen smoking and A conversation about the FDA’s new graphic health warnings for cigarettes
Photo by DLSimaging

Cancer, FDA, In the News, Patient Care, Pediatrics

An in-depth look at the even-deeper problem of drug supply

A shortage of chemotherapy drugs continues to plague patients in the U.S. The recent, widespread scarcity of medications continues to grow in severity, Healthland reports today in the first of a two-part series investigating the issue:

The shortfalls aren’t limited to cancer drugs either–antibiotics, anesthetics, vaccines and even medications to treat ADHD are getting scarcer. According to the Food and Drug Administration (FDA), demand outstripped the supply of 178 drugs in 2010. The University of Utah Drug Information Service, which works with the American Society of Health System Pharmacists (ASHP) to track shortfalls, says the number was actually closer to 211. Last year, the ASHP and Utah group say the number of drugs in short supply reached a record high of 267.

Stanford’s Michael Link, MD, the Lydia J. Lee Professor in Pediatric Cancer, provides this perspective in the article:

“There’s little question that it has never been like this, not just with cancer drugs, but with drug shortages in general,” says [Link], president of the American Society of Clinical Oncology. “We have had shortages before, but they have been intermittent, and never anything as extensive both in terms of the breadth of drugs affected and the depths of shortages and how long they lasted.”

Examining the economics of drug supply and the sometimes-competing interests in the supply chain, the Healthland piece offers a hard look at a complex problem in an urgent stage.

Previously: Childhood leukemia patient on methotrexate shortage and A look at the dramatic improvement in pediatric cancer survival rates
Photo by Phil and Pam

Applied Biotechnology, FDA, Stanford News

Medical technology entrepreneurs discuss challenges facing start-ups at Stanford event

Medical technology entrepreneurs discuss challenges facing start-ups at Stanford event

Earlier this week on the Stanford campus, medical technology entrepreneurs shared advice and commented on the challenges faced by young companies in the biotechnology, medical device development and healthcare fields. KQED reports:

As part of the university’s Entrepreneurship Week, StartX, Stanford’s student-run startup accelerator program, hosted a panel where former students and company founders now working in the medical technology sector offered insight into all angles of the startup struggle.

All seven speakers agreed that funding problems are the largest roadblock but not wholly detrimental to the creative process.

“Due to the volatility of the FDA, most investors won’t invest until the later stage” of development, said Lily Truong, chief executive of ClearEar. The company’s total ear-care tool safely and quickly removes severe earwax buildup. “This causes the problem that if investors want late-stage companies, how does a startup get to that stage without help in the beginning?”

Previously: FDA begins to revamp approval process for medical devices, Stanford physician-entrepreneur discusses need to change FDA approval process, Stanford Biodesign Program releases video series on the FDA system, Is the United States losing ground as a leader of medical innovation?

Cancer, FDA, Pediatrics

Childhood leukemia patient on methotrexate shortage

If, like me, you’ve been following news of the nation’s methotrexate shortage, you already know that severely curtailed supplies of this powerful chemotherapy drug are threatening the lives of children who need it for cancer treatment. (Lucile Packard Children’s Hospital’s Michael Link, MD, the president of the American Society of Clinical Oncology, has been quoted in several news stories explaining the problem.)

Today, CNN’s health blog The Chart covers this story in a powerful new way, through the words of a 12-year-old boy now being treated for acute lymphoblastic leukemia. Here’s part of what Owen McMasters, who was diagnosed in November, says about his experiences in cancer treatment:

I take many different kinds of chemotherapy drugs, at least once per week and sometimes many days per week. I have to get my blood drawn frequently. I have to get up early, go to the doctor’s office or the hospital, and spend many hours getting treatment. I have been in the hospital sometimes for up to 10 days in a row.

I had a severe infection from my immune system being wiped out.

I have to get radiation treatments in my brain. Sometimes the treatments don’t bother me too much; sometimes they make me tired, weak, nauseated and unable to eat for days. They make me cramp all over and swell.

While none of this is any fun, I do what I have to do and deal with it in my own way. I joke with the doctors. I entertain my parents. Sometimes I use bad words and get away with it because I have cancer. But I do everything I am told to do because I am going to live and be cured of cancer. The doctors say I have a very good chance to be cured if I take all of the treatments, which last for three and a half years.

The story goes on to explain that, at a recent check-up, Owen’s doctor said the next phase of his treatment is threatened by the methotrexate shortage. Owen needs high-dose methotrexate delivered through an IV in his spine.

Soon after receiving the bad news, Owen was visited by representatives from the Make-a-Wish foundation. His wish?

I wanted to make the shortage of methotrexate known by everyone who could do something about it. I wanted the drug companies to share my goal of curing my cancer and cancer in lots of other kids by making these drugs, even though they are cheap and might not make as much money as some others.

The FDA has said that methotrexate will be available for now. But this problem is not solved for good. A permanent solution to this problem must be found. I don’t care how the problem is solved, it just needs to be solved.

Here’s hoping this brave young man’s story makes a difference.

FDA, Technology

FDA accepting public comments on social media rules for drug companies

Two years ago, the U.S. Food and Drug Administration held hearings on regulating how drugs and medical devices under its oversight are promoted through social media and on the web. The agency recently released social-media marketing guidelines (.pdf) for pharmaceutical companies and is currently taking public comments on the proposal.

American Medical News reports:

The draft guidance encourages all responses to unsolicited requests for information, which could include requests from physicians for information on off-label uses for certain drugs, to be made in a nonpublic format.

The agency calls on companies to respond to public requests, such as those sent through Facebook or Twitter, by guiding the requestor to the appropriate personnel or department for private, one-on-one help. The agency said the private responses should be science-based and answer only the question or questions asked.

The guidance does not apply to information or requests received in response to a solicitation by the pharmaceutical or medical device company.

Comments are being accepted until March 26. They can be submitted to Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD, 20852.

Cancer, FDA, Public Health

Avastin Q&A from the Food and Drug Administration

Anyone interested in the trials and tribulations of bringing a new cancer therapy to market, and keeping it there, is likely already aware of the fate of Genentech’s Avastin. Last Friday, the FDA revoked its approval of the use of the drug to treat metastatic breast cancer, to the dismay of many patients and their advocates. The revocation was based on studies showing that the drug, which can have serious side effects, failed to help patients live longer.

FDA Commissioner Margaret Hamburg, MD, issued a 69-page document explaining her decision. Today David Kroll, author of the PLoS Blog Take as Directed, called my attention to the fact that the lengthy document includes a good Q&A about the issue to help patients and their physicians understand what the new ruling means to them. Topics include whether physicians can continue to prescribe the drug for metastatic breast cancer, how the FDA ensured that Avastin got a fair hearing, and whether it’s possible that Avastin could again be approved at some point in the future for use in breast cancer patients.

You can read the full Q&A on Kroll’s blog post, or find it in the opening pages of the FDA document

Cancer, FDA, In the News, Medicine and Society

An in-depth look at pharmaceutical shortages

Jessica Wapner at Work in Progress, which is part of the PLoS Blogs network, has just posted a fascinating look at some very disturbing drug shortages. She begins with a broad stroke of the problem:

Drug shortages are not new, but they are increasing. Though not confined to cancer, this disease is being hit most severely because many chemotherapy drugs don’t have equivalent substitutes. If you’re short of a drug for blood pressure, you can select another one, but this is often not the case for cancer (though sometimes it is). And, 132 of 178 shortages in 2010 were for sterile injectables, many of which were for cancer.

From there Wapner offers two examples of pharmaceuticals in short supply. She moves on to discuss many of the causes of the shortages, as well as some of the consequences thta extend beyond clinical care:

Drug shortages are also slowing down investigations of new drugs. Clinical trials depend on having a control arm, in which well-known treatments are given to a group of patients for the purpose of comparison. If those standard treatments are not reaching the clinic, then these studies get delayed, slowing down the already years-long process of bring effective new drugs to market.

In all, it’s a very well written, in-depth look at a troubling problem. Highly recommended.

FDA, Health Policy, Public Health, Research

How have U.S. tobacco regulations affected smokers?

In 2009, with the passage of the Family Smoking Prevention and Tobacco Control Act, the FDA was given the authority to regulate tobacco products. Now the FDA and National Institutes of Health are teaming up to study whether the ensuing regulations, such as new warnings and labels on cigarette packs, have affected smokers. From a release:

Investigators will follow more than 40,000 users of tobacco-product and those at  risk for tobacco use ages 12 and older. They will examine what makes  people susceptible to tobacco use; evaluate use patterns and resulting health  problems; study patterns of tobacco cessation and relapse in the era of tobacco  regulation; evaluate the effects of regulatory changes on risk perceptions and  other tobacco-related attitudes; and assess differences in attitudes, behaviors  and key health outcomes in racial-ethnic, gender, and age subgroups.

Study findings will help the FDA assess the impact of the Tobacco Control Act  and will inform the agency about how to best use its tobacco regulatory  authorities, such as making decisions about marketing of products, setting  product standards, and communicating the risks from tobacco use to protect the public health.

Previously: Study shows anti-tobacco programs targeting adults also curb teen smoking, A conversation about the FDA’s new graphic health warnings for cigarettes, Cigarette ads turn teens on to smoking, Massachusetts stores may be required to post graphic anti-smoking signs and Australia enacts world’s first ban on branded cigarette packaging
Via @Reuters_Health
Photo by Melanie Tata

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