Mon. Jan 24th, 2022

Reporting for this story was supported by a grant from the National Press Foundation.

Panama City, Panama – Maria has lost track of how many times she has seen a health care worker in the past three years. But despite countless medical examinations, she still has no clear answers about what makes her sick.

“I have been ill for so long. I want to know why, ”said Maria, who asked that her surname not be used to avoid possible repercussions at her Panama City workplace. “One doctor said maybe I had some rare condition, but he did not say which one.”

Maria, now in her early 20s, said she had been living for several years with chronic fatigue, muscle aches and other symptoms – and that it was gradually getting worse.

“I like watching the birds,” she told Al Jazeera from a bench in the city center as songbirds hovered in the shade of a nearby tree. “They help take my mind off everything.”

Print for answers

If Mary in fact with a rare disease, she can wait longer to come, as a proper diagnosis can often take several years in Europe and longer in Panama. Even then, if Maria is diagnosed with one of the 5 percent of rare diseases that have an approved therapy, she may not have access to it if Panama does not buy it for the public health system.

But a movement has mobilized in the Central American nation to change that.

Patient and parent-led Panamanian associations are joining forces to lead a concrete action to ensure early diagnosis, medical care and access to treatment for people living with rare diseases, and they have officially formed an umbrella group – the Panama Rare Disease Network Federation, RedER – in November to make their voices heard.

“We are stronger when we are united,” said Alaisa Arauz, director of the Panamanian Hemophilia Foundation, one of the 10 organizational members of RedER, which also incorporates individuals with diseases so rare that there are not enough patients in the country. for a group.

Rare diseases are only rare when considered individually. Worldwide, an estimated 300 million people live with one or more of more than 7,000 known rare diseases. The most rare diseases are genetic of origin is very life-threatening, and 95 percent has no approved therapy.

Hemophilia has an approved therapy in Panama, but it took 14 years of uninterrupted, patient-led advocacy in the country in the 1990s and early 2000s to pressure the government to obtain enough Factor VIII concentrates to meet patients’ needs . Factor VIII is the missing coagulation factor in hemophilia A patients’ blood.

“It was a great achievement,” Arauz told Al Jazeera. “It was years of struggle. With every government that took office, we were there. “

Arauz has two sons diagnosed with hemophilia, and she is living with Von Willebrand disease, another genetic bleeding disorder. But that was decades before she was diagnosed.

“It was [bleeding episodes] my whole life, ”said Arauz, who also acts as the delegate for patients with rare diseases in an intersectoral, government-created committee tasked with ensuring access to treatment for such patients. “Imagine if I had an early diagnosis. I would not have suffered for so long. “

Rare Diseases Act

Formally known as the Intersectoral Committee for the Prevention, Diagnosis, Comprehensive Attention and Research for the Treatment of Rare, Unusual and Orphan Diseases, the committee was born out of a 2014 Panamanian law specifically dealing with rare diseases – the only such legislation in Central America.

In Panama, as in the European Union, rare diseases are defined as those affecting less than one in 2,000 people.

The law aims to ensure social protection, diagnosis and treatment access for people with rare, uncommon and orphaned diseases. But although the legislation has been on the books for more than seven years, it has yet to be fully implemented.

“It’s a very good law, a very complete law, but as we called it, a ‘decorative’ law,” Arauz said.

For example, the law instructs the Ministry of Health to draw up a national program for the purchase of medicines for the treatment of rare diseases, “to enable immediate and equitable access for all patients”. But that key aspect has not yet been fulfilled.

One of the committee’s responsibilities is to conclude collaboration agreements with government and private sector bodies to fund treatments for rare disease patients. But the committee was only set up in 2019 and has made little progress since then. Along with Arauz, it includes six representatives from various government entities, as well as a pharmaceutical industry delegate.

The obstacle, according to Arauz and other leaders of rare disease patient associations, is that the Ministry of Health, chaired by the committee, has switched its representative several times and those representatives also have other duties in the ministry of health and are not only dedicated to rare diseases. .

The Ministry of Health was not available for comment prior to the time of publication, and the department has made very few public statements on the issue of rare diseases in the past two years.

In a statement as of October 2020, the ministry has noted some progress, including the identification of more rare diseases in Panama and a committee tasked with developing disease-specific management and treatment guides.

‘A full life’

But patient advocates say the longer it takes to implement the law, the longer people go without care.

sign outside a Panama City hospital saying you are not honkingWorldwide, an estimated 300 million people live with one or more of more than 7,000 known rare diseases [File: Jeffrey Greenberg/Universal Images Group via Getty Images]

“The law is not being followed and patients are not being treated,” said Enma Pinzon, president of the Panama Rheumatoid Arthritis Foundation and a longtime patient movement leader who is also involved with the National Federation of Critical, Chronic and Patient Patients’ Associations. Degenerative Diseases.

Pinzon told Al Jazeera that when rare diseases do get public attention in Panama, it is through charity and aid campaigns to improve the living conditions of people diagnosed with diseases for which there is no treatment.

This is important, but the focus ultimately overshadows the fact that there are some rare diseases with approved therapies and patients needing them, she said.

The focus of RedER will be advocating for forcing the government to implement the law and other measures, Pizon added, emphasizing that the goal is to ensure that everyone who needs medical care can get it.

“I have a chronic illness,” she said. “Thirty years ago, I was lucky enough to receive medication. I was lucky enough to receive an early diagnosis and here are the results. I have had a full life and that is what I am looking for for everyone with diseases in Panama. ”

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