New Breakthrough Drug for Cystic Fibrosis Approved
The FDA has announced the approval of Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy to treat the most common form of cystic fibrosis. This new drug has the cystic fibrosis (CF) community excited because it is expected to treat 90% of the CF population. For comparison, another recently developed drug to treat CF, Kalydeco, can only treat about 6% of the CF population because it targets a different genetic marker.
Cystic fibrosis is a rare genetic disease that produces thick mucus in the lungs, digestive tract, and other parts of the body. This thick mucus causes difficulty breathing, digesting food and ultimately leads to many infections and secondary diseases such as diabetes. Trikafta treats the most common genetic mutation that causes CF and helps the defective gene protein work more effectively.
In the U.S., it is expected that Trikafta will help nearly 27,000 people with CF and it will have a price tag of around $26,000 per month. Due to the rarity of this disease and the genetic component, we would expect to see Trikafta on most PBM’s pharmacogenomic prior authorization program and specialty drug lists.
Peanut Allergy Drug Inches Closer to FDA Approval
Aimmune Therapeutics' peanut allergy treatment, Palforzia, has passed the FDA's advisory committee review. The committee voted 7 to 2 in favor of the drug's effectiveness and 8 to 1 on safety but concerns about the drug’s effectiveness remain.
In clinical testing, this oral immunotherapy outperformed placebo but users also relied on epinephrine injections, like EpiPen, twice as often as those taking placebo. Also, due to the way the study was designed, it remains unclear if this drug will actually reduce the incidence and severity of allergic reactions caused by peanut exposure.
Palforzia is an oral capsule consisting of “pharmaceutical-grade” peanut powder that needs to be taken daily along with food. While that seems simple enough, the committee discussed the need for a Risk Evaluation and Mitigation Strategies (REMS) plan. Since Palforzia can actually trigger an allergic reaction, a patient willing to take this medication would need to attest to carry epinephrine everywhere they go and agree to limit dose escalation to only certified facilities. The proposed labeling would also likely include warnings about avoiding hot showers or exercise in the hours after dosing to reduce the risk of a reaction.
Aimmune Therapeutics' has not announced the price for Palforzia yet, but it has been suggested that the list price could possibly be as high as $20,000. The first 6 months of therapy are expected to cost between $5,000 and $10,000 followed by $400 per month after that.
OIG Report Shows Value of Rebates for Medicare Part D
Congress asked the Office of Inspector General to examine the value of drug manufacturer rebates for Medicare Part D. The OIG reviewed 1,510 brand-name drugs that had rebates every year from 2011 – 2015 and there were some interesting findings.
Total Part D drug spend (before rebates were applied) increased from $55 billion to $65 billion over the 5-year period. Once rebates were applied, the drug spend only increased from $46 billion to $48 billion. So, net of rebates, Part D drug costs only increased $2 billion which is much better than the $10 billion increase they would have seen if rebates were eliminated.
Another part of the analysis revealed that rebates did not always increase at the same rate as drug prices. In fact, for 99% of these drugs, when the drug prices and rebates increased over time, the increase in absolute dollars for its rebate was less than the increase in absolute dollars for its reimbursement.
While the future of pharmaceutical rebates remains uncertain, Washington is busy trying to figure out how to lower the price of drugs and this report shows that there is value in having rebates to offset the increases in drug prices. And while rebates considerably reduced the growth of Part D spending, they did not prevent increased overall spending for brand-name drugs since Medicare still spent $2 billion more for brand-name drugs with rebates in 2015 than in 2011.