Tag: "FDA"

Colorado Patients Win the “Right to Try” New Medicines Before the FDA Approves Them

Earlier this month, Colorado governor John Hickenlooper signed the nation’s first “right to try” law. The law allows a patient suffering from a disease, for which no medicine has been approved by the FDA, to try an experimental new medicine before the FDA approves it. The law allows, but does not force, drug-makers to provide their experimental drugs to patients. Other states, such as Louisiana and Missouri, are set to follow.

These patients are in dire straits. They suffer from diseases for which there is no other cure, and have short life expectancies. Most of us cannot imagine being in their position: They are willing to take far greater risks than most would accept, in their search for a cure.

Although the Food and Drug Administration (FDA) has an exemption for “compassionate use”, that exemption requires jumping through too many bureaucratic hoops to be useful. So, scholars at the Goldwater Institute developed the idea of state “right to try” laws that would enable residents to use experimental new drugs without FDA approval.

Hits and Misses

Physician and Nurse Pushing GurneyEmergency room visits increase in wake of ObamaCare.

Nanotech chip detects signs of cancer in blood protein markers.

318,000 federal workers owe $3.3 billion in back taxes.

1 in 4 Americans now consults Google before going to doctor.

California counties sue drug-makers for marketing painkillers in accordance with FDA regulation.

Some New Medicines are Approved More Quickly than Others

Joe DiMasi of the Tufts Center for the Study of Drug Development, and colleagues, have reviewed the time it takes for the FDA to review different types of new drugs.

seniors-and-prescriptionsFDA’s Neurology division, which approves drugs for Alzheimer’s disease, multiple sclerosis, Parkinson’s disease, and stroke, takes three times as long to approve drugs as the Oncology division. These differences cannot be explained by differences workload, the type and complexity of the drugs reviewed, or the safety of the drugs approved.

If the FDA could cut the performance gap between the divisions in half, the authors estimate that the cost of developing a new drug would decrease by $46 million — a savings that adds up to approximately $874 million per year.

Full report available from the Manhattan Institute.

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)