PHARMACY

FDA panel backs Pfizer drug for rare genetic disease

BY Alaric DeArment

NEW YORK — An experimental drug made by Pfizer for a rare and fatal neurodegenerative disease got a favorable vote from a Food and Drug Administration expert panel.

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 13-4 that the drug tafamidis meglumine would provide certain clinical benefits to patients with transthyretin familial amyloid polyneuropathy, or TTR-FAP, a hereditary disease that affects about 8,000 people worldwide. The FDA is not required to follow the recommendations of FDA advisory committees, but usually does.

The drug has received orphan drug designation, which the FDA gives to drugs that treat conditions affecting fewer than 200,000 people in the United States. Currently, there is no FDA-approved treatment for TTR-FAP.

"The panel’s assessment represents a positive step forward in our goal to provide this much-needed medicine to patients suffering from this rare and fatal disease," Pfizer Specialty Care business unit medicines development group head Yvonne Greenstreet said. "Pfizer will continue to work with the FDA as the agency finalizes its review of our [new drug application] for tafamidis."


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Retail clinics, drug store medicine the future of health care? Sounds familiar

BY Antoinette Alexander

WHAT IT MEANS AND WHY IT’S IMPORTANT — At the start of the article titled "Retail Clinics and Drug store Medicine" published in the Journal of the American Medical Association, author Christine Cassell, a physician, acknowledges that retail-based clinics have been "criticized in some quarters" but states that, despite this, the clinics have experienced success by patient satisfaction and quality scores. The fact that this message is coming from a physician makes it especially important.

(THE NEWS: Physician-authored JAMA article highlights importance of retail clinics, drug store medicine. For the full story, click here.)

That criticism that Cassell is referring to largely stems from the community of primary care physicians, some of whom have felt threatened by the concept and have expressed concern about the collaboration of care. So it is important to note that a premiere medical journal for physicians ran an article written by a physician who believes that retail clinics and drug store medicine are the future of health care in America.

Hmmm … sounds familiar. That’s a point that Drug Store News and the convenient care industry have stressed repeatedly, but we understand that when it comes from a doctor you have to take it seriously. "Dr. Cassel observes many of the most important elements of retail-based convenient care that the Convenient Care Association and its members have been talking about — and successfully demonstrating — for years, and also offers welcome insights into how the retail-based convenient care clinic industry figures into the future of health care," said Tine Hansen-Turton, executive director of the CCA.

"We appreciate and agree with Dr. Cassel’s assessment of how convenient care clinics can offer both an invaluable point of care for patients as well as critical partnership to other healthcare providers," Hansen-Turton continued. "We welcome conversations with all healthcare stakeholders to work toward effective care coordination and collaboration among providers."

At the close of the article, Cassel wrote: "There are challenges, but this is happening already. The question is whether this phenomenon will grow and flourish in the ways described here or whether 20th-century attitudes about physician and hospital dominance in health care will prevent market-based solutions to the healthcare access and cost crisis. This model is a challenge to medical and hospital leadership, as well as to leaders in the retail health clinic industry, as they pursue the potential opportunities and benefits for the American people."

Let’s hope this charge to the rest of the medical community serves as a final wakeup call as to the importance of this model to REAL healthcare reform.

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Generic Drug User Fee Act passes in Senate

BY Allison Cerra

WASHINGTON — The generic drug industry has applauded the passage of legislation that will expedite the Food and Drug Administration’s review of generic drugs and biosimilars, as well as enhance the agency’s ability to inspect foreign manufacturing sites.

The Generic Drug User Fee Act, which passed in the Senate on Thursday, is designed to provide the FDA with additional resources and ensure all participants in the U.S. generic drug system — whether U.S.-based or foreign — comply with strict quality standards to ensure all Americans receive timely access to safe, effective and affordable generic drugs. The GDUFA calls for the generic drug industry to pay $299 million annually in user fees for the next five years, beginning Oct. 1.

The Generic Pharmaceutical Association, which applauded the legislation, said the user fee programs are part of GPhA’s multipronged effort to ensure patients have access to lifesaving generic medicines.

"The Senate’s vote [today] is another critical step in ensuring patients will have access to the lifesaving generic medications they require for years to come," GPhA president and CEO Ralph Neas said. "The Generic Drug User Fee Act will expedite the availability of low-cost, high-quality generic drugs to Americans and further safeguard the quality and accessibility of our nation’s drug supply. We urge the House to continue the remarkable bipartisan cooperation shown throughout this process and approve the legislation in a timely manner so that patients, the FDA, and generic manufacturers can begin to see its many benefits."


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