Tag: "FDA"

Can States Overturn the FDA’s Limits on Compassionate Use?

At National Review Online, Jillian Kay Melchior, reports on a cancer patient who has run out of therapies. There are medicines under development that might help, but drug-makers won’t let her have them under the FDA’s “compassionate use” doctrine.

ClVariety of Medicine in Pill Bottlesinical trials accept only “typical” patients — Mikaela’s rare form of kidney cancer has ruled her out, but for others in her situation, complications as common as diabetes or high blood pressure could also be reason for ineligibility. And the Food and Drug Administration’s approval process for “compassionate use” of an experimental drug outside of clinical trials is extremely arduous.

First, a pharmaceutical company has to be willing to provide the experimental drug — a high-risk proposition, given that an atypical result in an atypical patient can prompt the FDA to delay approval or require significant and expensive additional testing…And approval from a pharmaceutical company is just the first step. Next, patients and doctors must fill out an exhaustive paperwork disclosure to submit to the FDA, which is estimated to take at least 100 hours to complete. The FDA then begins a review, which is supposed to take no longer than a month — but if the agency’s reviewers have any additional questions or need more information, that 30-day clock is reset. Finally, the hospital’s institutional review board has to give approval — and if a patient is being treated at a small hospital that lacks such an administrative panel, the case has to be reviewed by a bigger facility, often one unfamiliar with the patient.

Headlines I Wish I Hadn’t Seen

Blue Cross: 15% to 20% of enrollees aren’t paying premiums.

EPA tested deadly pollutants on humans.

Long-term antibiotics apparently work for lyme disease patients; but many doctors are afraid to prescribe them.

Are Foreign Drugs Safer Than You Think?

There are no reported examples of Americans dying by taking real, but F.D.A.-unapproved, medication bought online from a foreign pharmacy that requires valid prescriptions. This is after tens of millions of prescriptions have been filled online and internationally over the past 15 or so years, since online pharmacies were created. (NYT)

I Bet You Didn’t Know the FDA Regulates Sunscreens

…American beachgoers will have to make do with sunscreens that dermatologists and cancer-research groups say are less effective and have changed little over the past decade.

That’s because applications for the newer sunscreen ingredients have languished for years in the bureaucracy of the Food and Drug Administration, which must approve the products before they reach consumers.

 …The agency has not expanded its list of approved sunscreen ingredients since 1999. (Washington Post)

HT: Alex Tabarrok.

So Why Don’t We Speed Up Approvals And Save More Lives?

This is from an NBER Working Paper by Frank Lichtenberg:

prescription-bottleWe investigate the effect of the vintage (year of FDA approval) of the prescription drugs used by an individual on his or her survival and medical expenditure. When we only control for age, sex, and interview year, we estimate that a one-year increase in drug vintage increases life expectancy by 0.52%. Controlling for other variables including activity limitations, race, education, family income as a percent of the poverty line, insurance coverage, Census region, BMI, smoking and over 100 medical conditions has virtually no effect on the estimate of the effect of drug vintage on life expectancy.

New FDA Rule on Generic Drugs Could Hike Costs $4 Billion per Year

seniors-and-prescriptionsA proposed FDA rule on labeling generic drugs could increase generic drug-makers’ exposure to product-liability lawsuits. This could add $4 billion a year to the cost of generic drugs, according to Scott Gottlieb, MD, and colleagues.

  • A new regulation proposed by the Food and Drug Administration will compel generic drug makers to update their drug labels to reflect purported “new” safety issues.
  • The regulation will result in increased drug prices and leave generic drug firms vulnerable to “failure to warn” tort suits, but produce no public health benefit.
  • It would be far more efficient and effective for the FDA to either review and update generic drug labels itself or adopt a more rational and logical oversight process, known as Prior Approval Supplements. Either approach would allow updating of drug labels without additional tort liability.

Why Do Terminally Ill Patients Have to Ask for Permission to Take a Live-Saving Drug?

Idiopathic pulmonary fibrosis is the scarring of lung tissue over time with no known cause, cure or approved therapy. Patients survive on average for only two to five years after diagnosis, and about 40,000 die of fibrosis-induced respiratory failure every year — roughly the same number as from breast cancer.

A drug called pirfenidone has been shown to delay the decline in lung function and even prolong life. But the FDA refused to approve pirfenidone when it was last reviewed in 2010 because the regulators claimed the evidence was insufficient, and now a new clinical trial shows how careless that decision really was. (WSJ)

Digital Medicine

Today, all the physiological data monitored in a hospital intensive-care unit — including ECG, blood pressure, pulse, oxygenation, sugar level, breathing rate and body temperature — can be recorded and analyzed continuously in real time on a smartphone. A small piece of hardware, either the size of a cellphone, or one integrated with a cellphone, held against your body, functions as an ultrasound device. It can deliver information instantly to you or anyone you designate, and the information rivals that collected in a physician’s office or hospital setting. It can do so when you are experiencing specific symptoms — no appointment necessary — and at virtually no additional cost.

Smartphones-and-EMRThanks to more than 20 Silicon Valley startups and advances in microfluidic technology, smartphones will soon be able to function as a mobile, real-time resource for rapidly obtaining all the studies done currently in a medical laboratory, including chemistries, blood values and microbiological studies. A device worn on the wrist, called Visi, has been approved by the FDA for hospital use that can measure your heart’s electrical activity, respiratory rate, blood oxygen and blood pressure (without a cuff), and transmit the data wirelessly. Many other such devices are coming out that could be used by patients in their own homes. (WSJ)

FDA Regulations Kill

Paul Howard and Yevgeniy Feyman in Health Affairs:

Meningitis is a terrible disease that can kill its victims in a single day. About 4,100 new cases are diagnosed annually in the U.S., with a mortality rate of more than 10 percent. Even with treatment, survivors are often left with serious side effects that can include brain damage and limb loss…

Swiss drug manufacturer Novartis has developed a vaccine — Bexsero — that specifically targets this strain of meningitis; the drug has already been approved for use by the European Medicines Agency (EMA), the European Union’s equivalent of the Food and Drug Administration (FDA). And within about nine months the FDA allowed Princeton University to offer the vaccine on campus to its students.

But the drug is not available elsewhere in the U.S. And it won’t be until Novartis completes a costly phase III trial. The authors ask:

The natural question to ask is why the FDA shouldn’t just approve a product already approved for the European Union, Canada, and Australia, while monitoring the vaccine (as all vaccines are already monitored — Bexsero is being actively monitored in the E.U.) through postmarket surveillance records? (Indeed, the more than 5,000 patients vaccinated at Princeton are close to the total number of patients tested in the EMA required trials.)

The FDA is Hazardous to Your Health

Genzyme-Sanofi-LemtradaAlemtuzumab is used today as an intravenous treatment for a form of leukemia. But 20 years of research centered at Cambridge University also has shown that the action of this drug — depleting immune cells that become misdirected and attack one’s own body — is effective in treating multiple sclerosis.

Under the brand name Lemtrada (a product of Sanofi and its U.S. subsidiary Genzyme), the drug has been approved in recent months for treating MS in 30 countries, including Canada, Australia and all members of the European Union. But on Dec. 27, Food and Drug Administration reviewers at the division level (subject to a final decision by top officials) rejected an application to use the drug here to combat MS. (WSJ)