An innovative means for transporting organs may soon debut in the United States. It has the potential to bring organ transportation out of the “ice age.” TransMedics, a company headquartered in Andover, Massachusetts, has developed the Organ Care System, also known as the “heart in a box.” of the system can keep human organs “alive, beating, and breathing” during transport. Rather than transporting disconnected organs via ice cooler, the Organ Care System plugs hearts, lungs, livers, and other organs into a system that keeps blood, air, and other fluids circulating while the organ is maintained at body temperature. Organs – transported alive – have been successfully transplanted up to twenty-four hours after removal, which is roughly six times the current average life span for an organ outside the body. Waleed Hassanein, founder and president of TransMedics, has asserted that as long as an organ remains alive and “perfused in [the Organ Care System] . . . there really is no time limitation [for successful transplantation.]” Even more astonishing is the device’s ability to revive organs from recently deceased patients. Thus far, seventeen people have successfully received hearts from patients whose hearts had stopped beating for thirty minutes or more.
Although the Organ Care System has been in use for many years in Europe and Australia, the FDA has yet to approve its use in the United States. However, this may soon change: TransMedics is scheduled to introduce its system to the FDA on November 18, 2015. If FDA approves the system’s use, the “ice age” of transplant organ transportation may come to and end.
As previously reported, in August of this year, Turing Pharmaceuticals purchased the rights to Daraprim, a drug used to treat a variety of conditions, including toxoplasmosis, certain cancers, and malaria. Although the primary agent in Daraprim, pyrimethamine, has been available since 1953 and Daraprim was previously priced at approximately $13.50 per tablet, Turing Pharmaceuticals raised the price of the drug to $750.00 per tablet, sparking outrage across the county. However, in a refreshing turn of events, a recent announcement by generic drug manufacturer Imprimis could lead to a correction in Turing Pharmaceuticals’ pricing for Daraprim.
In an announcement covered by The San Diego Union-Tribune, seen here, generic drug manufacturer Imprimis introduced a competitor drug to Turing’s Daraprim, priced at $1.00 per capsule—a huge benefit for patients who would otherwise be subject to Daraprim’s high cost of $750.00 per capsule. Having previously cornered the market for the primary agent in Daraprim, pyrimethamine, Turing could seemingly set the price of the drug without fear of competition; however, with Imprimis’ announcement, patients have a more reasonably priced alternative for treatment. Imprimis additionally announced its intention to manufacture other generic drugs and sell them at more reasonable prices.
Imprimis, in designing the formulation of the generic drugs, uses specific FDA approved ingredients, compounded in FDA inspected compounding operations. However, because the combined ingredients (or “formulation”) is not FDA-approved, a specific and individualized prescription is required to administer the drug to patients. This method of operation saves Imprimis time and money by circumventing the lengthy process of seeking FDA approval of the drug’s formulation. Not surprisingly, this practice has received some criticism by providers who desire to prescribe FDA approved formulations.
In early March 2015, the Food and Drug Administration (“FDA”) approved Zarxio, the first “biosimilar” drug, to be introduced into the U.S. market. Biosimilars are expected to offer cheaper versions of existing biologics. They are nearly identical to the originals, and are used to treat the same conditions. Biosimilar drugs are expected to create a more competitive market by providing people with comparable, less expensive prescription medication.
Zarxio, the first of what is likely to be many biosimilar drugs to receive FDA approval, will compete with Neupogen, a drug prescribed to chemotherapy patients. Zarxio promises to be cheaper than Neupogen and similar enough to serve all five of Neupogen’s current uses. However, questions remain as to how effective biosimilars will be in lowering the cost of drugs. In fact, the makers of Zarxio have not announced how much cheaper the drug will be. Secondly, there is no telling whether clinicians will embrace biosimilars.
Amgen, the makers of Neupogen, asked a California federal judge for an injunction blocking the launch of Zarxio. They allege that the makers of Zarxio failed to follow the rules in seeking regulatory approval. A court hearing is set for March 13, and Novartis, the maker of Zarxio, has agreed not to sell Zarxio until the hearing or April 10, whichever is earlier.
GlaxoSmithKline created a vaccine to protect against four strains of the flu virus. Prior to its advent, vaccines protected against only three strains. More comprehensive viral protection is important because providers never know which strain will predominate in a given year. The vaccine is called Fluarix Quadrilivalent (Fluarix). Recently, the FDA approved Fluarix for shipment to health care providers, and the largest order for the vaccine came from the U.S. Centers for Disease Control and Prevention (CDC). The CDC ordered 4 million doses for initial distribution to local and state health care providers.
A substantial portion of the budget for the Food and Drug Administration (FDA) is comprised of user fees, collected from the industries that the agency regulates. The FDA spends the money collected from the user fees for specific purposes, such as the expedited review and approval of drugs or medical devices. At present, the user fees are not exempt from the sequester, which went into effect in January of this year. Rep. Anna Eshoo, (D-Calif.), is a lead sponsor of a bipartisan House bill, the FDA Safety over Sequestration (SOS) Act, which would exempt the user fees from sequestration. She stated that “private dollars” should not be sacrificed in the name of policy. Proponents of the Act maintain that applying the sequester to the user fees would stifle medical innovation and patient access to “timely therapies.” The SOS Act has found support amongst prominent medical device groups as well.
In a 5-4 ruling the Supreme Court held that the FDA is the primary authority on pharmaceuticals, and overturned the verdict and award for the plaintiff by the New Hampshire state court in Bartlett v. Mutual Pharmaceutical Company (Mutual Pharma). The Justices reasoned that generic drugs are exempt from legal liability for mislabeling, side effects and all other adverse reactions. Eighty percent of all drugs consumed in the United States are generic.
Originally, the Court of Appeals for the First Circuit upheld the jury verdict in favor of the plaintiff from the Federal District Court for the District of New Hampshire. The jury determined that Mutual Pharma was liable for mislabeling the generic anti-inflammatory drug, Sulindac, after it was deemed responsible for Ms. Bartlett’s suffering a “flesh-eating side effect.” That risk not included on the warning label. In overruling the lower court, the Supreme Court extended a 2011 ruling, which held that only the original inventors and manufacturers may be held liable for fraud or adverse side effects.