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Not all amyloidosis patients at a major hospital system could get their hands on commercial tafamidis (Vyndaqel or Vyndamax), and when they did, it was often with financial assistance, researchers reported.

From May to November 2019, immediately after FDA approval, 50 people were prescribed the transthyretin (TTR) protein stabilizer tafamidis, with 86% successfully obtaining the drug, according to a group led by Ahmad Masri, MD, MS, of Oregon Health and Science University in Portland.

These patients largely had prescription insurance, which paid for most of the 30-day cost of the drug (averaging $23,485), Masri's group showed in a paper published online in.

FDA approved tafamidis in May 2019 for the treatment of the rare disease TTR amyloid cardiomyopathy based on reduced all-cause mortality and cardiovascular hospitalizations in the ATTR-ACT trial. Listed at $225,000 per year, tafamidis became the "most expensive cardiac medication in history," Masri's group noted.

People in their study eligible for financial assistance, either from a foundation or the manufacturer, ended up paying nothing for their tafamidis. Those not on financial assistance had 30-day out-of-pocket costs totaling a median of $250 (or $1,683 on average).

"Our initial experience prescribing tafamidis demonstrates that the current system depends heavily on copayment assistance programs," the investigators said. They noted that 30% of patients qualified for help from a foundation and another 26% received financial assistance from the manufacturer.

Of the seven individuals who did not obtain the drug, three could not afford out-of-pocket costs, two stopped trying to get the drug, one died before receiving it, and one decided to enroll in a research study.

One problem is that funds from nonprofit foundations may not last forever. "We have experienced periods during which programs have closed temporarily, causing tremendous uncertainty for patients," according to the authors.

Additionally, financial assistance programs exclude many people who cannot afford tafamidis despite crossing a financial threshold, Masri and colleagues said.

"In our experience, some of the patients who were initially denied manufacturer assistance had success with an additional level of appeal. In these cases, patients directly appealed to the manufacturer with more granular explanations of unaccounted financial obligations and hardship that may not be captured on an income tax report."

Their center benefited from having an integrated multidisciplinary amyloidosis program, a specialty pharmacy, and a dedicated pharmacist who spent an average of 1 hour per patient to ensure they could afford tafamidis.

Thus, Masri's team cautioned that their early experience with commercial tafamidis might not be applicable to other centers.

The 50 people in the present study averaged 76 years of age and happened to be all men.

Insurance type was Medicare Part D for 76%, private insurance for 12%, Veterans Affairs insurance for 4%, and other insurance for 6%. All tafamidis prescriptions required prior authorization from the insurer. Only 2% of people did not have prescription insurance.

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