Group wants access to high-cost medicine for rare disease – 02/08/2023 – Health

An initiative formed by patients, family members, academics and civil entities seeks access to a set of high-cost cystic fibrosis drugs.

Produced by Vertex, currently the sole patent holder, the set of drugs marketed by the names Trikafta (elexacaftor/tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), Symdeko (tezacaftor/ivacaftor) and Kalydeco (ivacaftor) are capable of modulating the cellular action that leads to the accumulation of mucus in the lungs and digestive system. The expectation is to increase the life of people with the condition by up to 27 years.

Cystic fibrosis is a rare genetic disease caused by a mutation in the CFTR gene that prevents proper fluid exchange between cells, causing mucus to build up in the lungs and other organs. If not treated, it worsens the quality of life during childhood and adolescence and can also lead to early death.

The problem is the cost of treatment, estimated at around US$ 327,000 per patient per year (or more than R$ 1.6 million converted directly, without the discounts agreed between the government and the pharmaceutical company), in the case of Trikafta, and US$ 300,000 (or around R$ 1.5 million), in the case of Kalydeco, leading patients and their families to look for ways to expand access.

Launched this Tuesday (7), the campaign aims, among other actions, to obtain a compulsory license to produce CFTR modulators, facilitate the production of generics and offer greater access in the diagnosis and treatment of patients in four countries: South Africa, India , Ukraine and Brazil.

Called Vertex Save Us, the group is made up of patients with cystic fibrosis, family members and activists in conjunction with the NGO Just Treatment, headquartered in the United Kingdom, which seeks to raise awareness of the offer of the medicine that can save thousands of lives and put pressure on the pharmaceutical and governments to form agreements.

Last Monday (6), Abram (Brazilian Association for Assistance to Mucoviscidosis – Cystic Fibrosis) sent the Minister of Health, Nísia Trindade, a letter requesting that the government file a request for a compulsory license for the treatment of cystic fibrosis and with the creation of a national stockpile to end “the suffering and anguish of all people affected by this fatal disease”.

If the compulsory license is approved, Vertex would patent the medicine, which could then be produced by other pharmaceutical companies, paying a royalty amount to the company.

The patent breach process occurred twice in the world in the face of global emergencies: in the HIV/AIDS epidemic, in the late 1980s and early 1990s, and in the Covid-19 pandemic for the manufacture of vaccines.

In Brazil, it is estimated that about 6,600 people have cystic fibrosis. The diagnosis is usually given in early childhood – about half of patients diagnosed with cystic fibrosis do not survive the first 18 years of life.

Because it is a chronic and progressive disease, the clinical picture of patients tends to worsen over the years, and the average lifespan is 38 years in the United Kingdom and 51 years in the United States. In many low-income countries, patients die before age 18.

As Vertex justifies the treatment prices due to the agreements signed with some rich countries for reimbursement by the government of up to 80% of the value, the company practices the so-called “global market integrity” to maintain the same value of the medicine in countries like India and Brazil.

CFTR modulators, such as Trikafta, have been approved by Anvisa, but have not yet been approved by Conitec for incorporation into the SUS. The other drugs Orkambi and Symdeko were rejected by the commission because of their high cost. The price for purchase by the government of Trikafta was established at R$ 888,174.43, according to data from CMED (Chamber for Regulation of the Medicines Market).

Questioned by the report, Vertex replied, via press office, that the price of Trikafta “reflects the clinical value and benefits it brings to patients, caregivers and health systems” and that “it took more than 20 years of research [….] to make our treatments a reality”. He also said that the reimbursed prices are not defined unilaterally, but agreed with the authorities of each country.

In the case of Brazil, they are regulated and defined according to CMED criteria, thus existing a formal and wide access route since 2021. According to the pharmaceutical company, the incorporation request made to Conitec, in January of this year, is followed by an agency plenary, public hearing and another final plenary to define its recommendation, and the whole process can take up to 270 days.

The company also said that one of its goals is “to provide the drugs to the greatest number of patients with cystic fibrosis” in the world and that it has formal reimbursement agreements in more than 40 countries outside the US. “We continue to work actively to expand access, including in low-income countries, recognizing the complexities and challenges of access in these markets.”