The inter-ministerial commission on Medicine Prices (CIPM) has proposed the total or partial financing of eight medicines, including tafamidis, a very expensive drug whose price was criticized by the academics who helped develop it a few years ago.
Tafamidis, marketed by Pfizer under the brand name Vyndaqel, is used for the treatment of native or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy.
Amyloidosis is a heterogeneous group of rare diseases, which are defined as: multisystemic, multiorganic, endemic, degenerative, in some cases hereditary and, in others, spontaneous.
It arises as a consequence of the deposit of amyloid protein in the extracellular spaces of different organs, which leads to their progressive dysfunction. When it accumulates in the heart it causes restrictive cardiomyopathy, heart failure, and ultimately death.
Although heart or liver transplantation is considered a treatment option in some patients, it is often hampered by their advanced age, which is around 60 years. It is estimated that in Spain there are a total of 400 patients.
Tafamidis, which slows disease, helps patients live longer and keeps them safe from hospitalization, is the first drug approved by agencies in the US, at the beginning of 2019, and Europe, three years ago, for the cardiac form of amyloidosis.
The big problem is the price. 651 dollars a day or 225,000 dollars a year, as established by Pfizer in November 2019 in the US, for a treatment that is for life. The pharmaceutical company considered it an appropriate price in the sense that it is a medicine required by a small number of patients.
An estimated 100,000 to 150,000 people in the United States suffer from transthyrtin cardiac amyloidosis, but only 4% to 5% are diagnosed, as it is often confused with other conditions such as common heart failure.
Matthew Maurer, a professor at Columbia University, was the lead author of the trial that won tafamidis approval, but he openly disagreed with Pfizer's pricing policy. "I told them - to Pfizer - that I thought $25,000 a year might have been a reasonable cost," he said, arguing that the drug can be taken by many more people than predicted by the laboratory, and for years or decades (one pill daily, a dose of 61 mg).
Some specialists maintain that the disease is not as rare as it seems, but that it is underdetected due to the difficulties of diagnosis and the lack of treatments up to now.
In Spain it is estimated that there are a total of 400 affected. In September 2021, the Drug Prices Commission decided that tafamidis would not be financed by public health, "taking into account criteria for rationalizing public spending and the budgetary impact of the SNS" and the high budgetary impact compared to other drugs for insufficiency cardiac.
Pfizer set a price of 11,000 euros for each pack of 30 Vyndaquel tablets, bringing the monthly cost of the treatment to around 11,200 euros.
In this sense, the Visible Amyloidosis (AV) association qualifies as "historic" the financing of tafamidis, "the medicine that improves the lives of patients". The organization has collected 120,000 signatures, has held a traveling exhibition and has maintained numerous contacts with authorities in search of this objective. “We cannot sit idly by now and think that everything has been achieved. We are not going to let our guard down since this disease is still unknown and it is necessary to continue investigating”, affirms its president, José Manuel Pérez.
Along the same lines, the Spanish Association of Amyloidosis (Amilo), created in 2018 with the aim of offering care to those affected and their families and contributing to research, applauds the decision of the Price Committee.
"It is a step that we have been demanding for a long time because patients and relatives of this disease, classified as rare, were a bit abandoned. For this reason, the sensitivity of those who have given their approval to finance this medicine is appreciated," said the president de Amilo, Mari Carmen Nadal, has already claimed the approval of another key drug for those affected, daratumumab.
The positive financing proposals of the CIPM become resolution proposals by the General Directorate of Portfolio and Pharmacy, which have to be accepted by the pharmaceutical companies.
At its last meeting, the CIPM proposed the total or partial financing of eight new medicines, four of them orphan drugs for rare diseases.
