Lucila Pellettieri, GPJ Argentina
Cecilia Capitani sits in her dining room. Capitani, a journalist, was diagnosed with multiple sclerosis in 2004.
BUENOS AIRES, ARGENTINA — When Cecilia Capitani was diagnosed with multiple sclerosis in 2004, she was working as a journalist in Buenos Aires and planning to start a family with her husband of five years. At the time, the only available treatment for her was a combination of painful daily immunosuppressant injections and corticoids, which left her body so sore that she had to start taking 20 pills a day, including several painkillers and stomach-protecting medicines, to counter side effects. “A time came when her arms were hurting, her legs, everything,” recalls Marcelo Mora, Capitani’s husband. “I couldn’t hug her, and I couldn’t touch her because everything hurt.”
As the side effects became unbearable, Capitani decided to suspend her therapy. For nearly 10 years she kept her symptoms at bay, thanks to a mix of homeopathic treatments and changes in her daily habits, but in 2017 she began experiencing involuntary tremors, a sign that her illness was progressing.
Her doctor told her that there were now more modern medicines that could treat the illness without affecting her quality of life. He prescribed her natalizumab, a medication that could stop the disease from developing further by preventing the loss of mobility and relapses. Capitani immediately thought about how much the new therapy would cost.
“Is it affordable?” she wondered.
Capitani had a good reason to worry. Medicines used to treat rare diseases tend to have exorbitant costs and access to coverage is riddled with bureaucratic obstacles. The monthly dose of natalizumab, the most common drug prescribed to stop the effects of multiple sclerosis, for example, has a published price of about 2 million Argentine pesos (7,815 United States dollars).
Mora spent weeks filing paperwork to have his wife’s private health care provider cover the cost of her treatment before their request was finally approved. “We would never be able to pay for it ourselves,” he says.
In Argentina, legislation guarantees medical treatment to every patient who, like Capitani, is living with an illness affecting 1 in every 2,000 people or fewer. Once the medicine is prescribed by a doctor, patients can access it for free, either through their private health care providers or the public health care system. A similar scheme is also available to people with more common chronic illnesses, such as heart disease. But while providers only pay up to 70% of prescription costs for medicines used for common chronic conditions — leaving it to patients to foot the rest of the bill — they’re supposed to cover the entire cost of expensive medications for rare diseases.
As providers often exploit loopholes to refuse or delay these high-cost treatments, patients can initiate legal action to obtain their prescriptions. They have a high chance of winning. Between 2017 and 2020, patients won 90% of lawsuits against providers who refused coverage.
In recent years, facing increasing pressure from patient organizations, the Argentine government also approved a series of measures aimed at expanding public coverage of expensive therapies. In 2019, the government created the National Commission for Patients with Spinal Muscular Atrophy, which facilitates patients’ access to high-cost medications. The number of treatments delivered through the commission went from 59 in 2021 to 99 in 2022. The government’s plan to expand coverage, combined with judicial action, has allowed a record number of people to access treatment for SMA.
Thanks to scientific and medical advances, there are increasingly specific treatments able to stop, pause or reverse illnesses, compared to previous treatments that would only relieve symptoms, explains Gisela Streitenberger, a cardiologist and geneticist who works with rare diseases. But as more therapies are approved to treat the 3.6 million people currently living with a rare disease in Argentina, experts, officials and patient associations worry about the sustainability of the country’s health care system.
Public spending on high-cost medicines soared from about 204 million pesos (805,430 dollars) in 2018 to about 4.5 billion pesos (17.7 million dollars) in 2022.
“The system is at risk,” says Gustavo Marin, a professor of pharmacology at the Universidad Nacional de La Plata, one of the top public universities in Argentina. “We need to figure out what a health system needs to guarantee for everybody.”
Argentina’s public health care system also covers therapy costs for those who have no insurance, and in some cases, when private health care providers cannot afford expensive medicines, the Ministry of Health must step in to cover the costs.
“Medicines that cost 1 or 2 million dollars checkmate developing countries like ours and make it difficult to incorporate these types of drugs,” explains Maximiliano Nitto, a lawyer who heads the Health, Social Action, Education and Culture Department at the Defender of the People of the Nation, an independent organization aimed at protecting human rights in Argentina.
There are currently 4,270 genetic therapies aimed at treating rare genetic diseases — which account for nearly 80% of all rare diseases — in the last stages of their respective trials. Their presence on the market is imminent, says Florencia Braga Menéndez, the project director of the Argentine Alliance of Patients, a civil association that supports those with rare, chronic or difficult-to-diagnose illnesses, but with an estimated cost of 1 million dollars per treatment, Argentina won’t be able to afford them. Braga, who has a son with Stargardt, an ophthalmological disease that affects 1 out of every 8,000 to 10,000 people, eagerly follows the approval of new medications.
“These genetic therapies correct illnesses that used to be inescapable curses,” she says. “We need the medicines, but I want to ensure that there’s enough for everyone.”
Braga believes that, for everyone to be able to access these therapies, the government should keep a registry of patients who need them, so that Argentina can plan joint purchases at the national level, as well as with other Latin American countries, and negotiate better prices with drug companies.
Natalia Messina, director of special and high-cost medicines at the Ministry of Health — the agency in charge of defining criteria for coverage of high-priced drugs — says they’re already working on it. In 2018, the ministry created the Registry of Patients with Rare Diseases, and in January announced an agreement with Novartis, a pharmaceutical company, to lower the price of a genetic therapy for SMA.
Thanks to these negotiations, Messina says, the government has been able to lower the price from 2 million dollars to 1.3 million dollars per treatment and pay for it under a scheme of shared risk between the company and the public health care system in which the state only pays if the medicine benefits patients as expected.
Marin believes that, while such a scheme can be helpful, the pharmaceutical companies’ lack of transparency about the real costs of these treatments could hamper negotiations. “It’s really very difficult to take this kind of shared risk when I don’t know what the real price is,” Marin says.
Messina admits that the adoption of new high-cost medicines will generate a complicated scenario. She says that there are several bills proposing the creation of funds to finance high-cost treatments, but while Congress considers the bills, they must continue working on this issue, which for many is a matter of life and death.
In Capitani’s case, the medicine worked; she hasn’t had a flare-up in six years, nor has she lost further mobility. But the ghost of the illness and what could happen if her health care provider decides to stop paying for treatment still loom. “If I suddenly become paralyzed,” she says, “I’ll die.”
Lucila Pellettieri is a Global Press Journal reporter based in Buenos Aires, Argentina.
TRANSLATION NOTE
Sarah DeVries, GPJ, translated this story from Spanish.
