Access all areas

– Alejandra Menéndez, HAE Argentina.

Fighting for access to and reimbursement for lifesaving modern HAE medication is in the DNA of HAEi, and as Alejandra tells us above, of the 100 member countries that make up this global community.

While rewarding, working to gain access to medication can be daunting. It can seem like there is little progress, but MOs are succeeding every day and in many ways. Global Perspectives spoke to Alejandra Menéndez from Asociación Argentina de Angioedema Hereditario (HAE Argentina), Fernanda and Raquel de Oliviera Martins from the Associação Brasileira de Portadores De AEH (Abranghe, HAE Brazil), and Mohand Cherif Negrouche of HAE Algeria, to hear about their current work and the status of treatment availability in their country.

To help us understand your situation, please tell us the status of reimbursement/approval of modern HAE medication in your country.

HAE Argentina: With the approval of Orladeyo by Argentina’s National Administration of Drugs, Foods, and Medical Devices (ANMAT) last November, our country now has 6 modern treatments available either to prevent or treat HAE attacks. These treatments are currently reimbursed by one of the three different major payers within our National Health System.

Argentina has a fragmented, federal health system, and while access to treatments is a right guaranteed by our National Constitution, it is complicated in the case of rare diseases. In theory, the right to health is essential, but still, you must fight for it. Nothing comes easy.

When we started our activities and the first HAE modern treatment was approved in Argentina, access was extremely restricted. According to a recent survey conducted by HAE Argentina, 94% of our patient population has access to at least one modern treatment, which is fully reimbursed. Disparities and difficulties still exist. There are inefficiencies, and the health system itself needs a federal commitment to all rare diseases.

Abranghe: The process of approval and availability of medicines in Brazil can be complex. It breaks down as:

Approval by ANVISA (National Health Surveillance Agency) based on the medicine’s safety and efficacy. The pharmaceutical industry requests/drives this process. Patients can take legal action to gain access, but this may take months.

Incorporation into the SUS (Single Health System) list. This approval means a medicine is available free of charge to all Brazilians. SUS approval implies the medicine is available in the hospital. Regarding HAE crisis (on-demand) medications and injectables, availability is for hospital care.

Finally, a clinical protocol of therapeutic guidelines (PCDT), can ensure that a medication is made available so that the patient has emergency or crisis medication at home.

In Brazil, Berinert (intravenous, for on-demand treatment) and Firazyr were approved and made available by the government following legal cases and judicial requests. In 2023, after public consultations, both were included on the SUS list. Berinert (subcutaneous) and Cinryze are approved but are only available via legal applications. Takhzyro is approved by the agency that regulates health plans (ANS) and is available via the private health system.

In April 2024, Orladeyo was approved by ANVISA and approved for incorporation into the SUS, which means it will soon be available to all Brazilians with HAE. This was a very important step because it will be the first modern oral medicine for prophylaxis available in Brazil. The availability of low-price oral medicine danazol made it hard to secure approval of a higher-cost medicine.

HAE Algeria: Our association was officially recognized in September 2020. The first modern medications were registered as follows: Cinryze in November 2022, Berinert in August 2023 and Takhzyro registered in February 2024. The medication is registered but not yet reimbursed. The final purchase/reimbursement approval of the registered medication is currently in progress at the Health Minister level.

What role has your Member Organization played in securing reimbursement/approval of HAE medication?

HAE Argentina: Our work to gain access to treatments began with the start of our organization. We looked at how other local rare disease organizations were dealing with the access issue. Then we engaged with regulatory authorities to raise awareness of HAE and highlight the risks of our condition and the unmet medical needs.

Most patients received treatments only after filing lawsuits against the Government or their individual health providers. HAE Argentina hired a law firm who specialize in health to guide patients through the judicial procedures.

Gradually, we unpacked a complicated scenario and, until recently, found ourselves in a stable position. As HAE treatment paradigms began to change, the demand for newer HAE medications increased. Thankfully, we are now equipped with many years of experience, placing us in a strong position to navigate the system.

Abranghe: Abranghe has always worked to secure access to modern medicines. Firazyr was approved by ANVISA in 2010, and Berinert in 2014. But, the SUS only approved them for wider availability at the end of 2023. Abranghe advocated for these approvals by participating in discussions with doctors and the government and encouraging patients to engage in relevant public consultations. For the approval of Orladeyo, we were part of the conversation and contributed demographic data about patients in Brazil, which is used to build the business case for drug approval. We have seen that the first modern medicine approval may be a longer and more difficult process, but those that follow can benefit from a speedier process.

HAE Algeria: We had a primary role. We raised awareness of HAE, and liaised with key stakeholders. This was especially important with the Ministry of Industry and Pharmaceutical Production (MIPP), which took place during the national HAE conference organized by our organization. [In Algeria, the MIPP is responsible for registration of local medicines as well as imported medicines]

Did any other individuals or organizations play a role?

HAE Argentina: HAE physicians and pharmaceutical companies have also been instrumental. The passing of laws regarding Rare Diseases also proved to be very useful, as well as our key partnership with Argentina National Federation of Rare Diseases (FADEPOF) which helped HAE gain more visibility, especially with regulatory authorities and health care providers. One specific collaboration was to address health regulator concerns about currently available international guidelines. HAE Argentina worked with a group of HAE expert physicians to create a guide to treatment in Argentina, which has proven to be one of our most valuable resources in the fight for access.

Abranghe: The Brazilian doctor group GEBRAEH actively partnered with ABRANGHE to fight for access. And of course, our patients, especially around public consultations.

HAE Algeria: Members of our organization’s scientific committee helped by giving their recommendations to the different health authorities.

What are the next steps to secure or maintain access to modern medication in your country?

HAE Argentina: We know that each new prospective HAE treatment poses a new challenge for our community. Securing medication access will continue to be a fundamental pillar of our work. We will steadfastly keep advocating, educating, and welcoming new treatment options. Given the several modern treatments approved, we firmly believe that each person with HAE has a right to an individualized, tailored treatment and that is what we will continue to fight for.

Abranghe: The advocacy for modern medicines does not stop, as science continues to evolve and produce more effective medicines with fewer side effects and different administration options. It is important that several medications are available so that the patient can access the one that best suits their needs.

HAE Algeria: We are organizing a second national conference on HAE. During this meeting we will advocate for HAE patients and try to convince the health ministry to speed up the process of reimbursement of registered medication for our patients.

How would you encourage other Member Organizations to advocate to gain access to modern HAE medication?

Abranghe: Access to modern medicines is essential for patients but it is long, continuous work. Engage with the pharmaceutical industries and cultivate a good relationship. You can share information but be ethical and respect data protection law and patients. Engage with doctors so that they are always aware of patients’ needs. And engage with government bodies so that they understand the effectiveness, safety and need of medicines for patients.

HAE Argentina: Please know that it can be done. There is always a way. Right now, it might seem unthinkable, but start with a robust patient group and focus on awareness, education, and building alliances. Never, ever quit. You will be discouraged at times, and that’s okay. A committed group of people can overcome adversity.

Modern treatments mark a huge, tremendous change in a patient’s life. Every person living with HAE should be entitled to much-needed medications. If well-treated and managed, people with HAE have no limits. I would say it is unprecedented for a rare condition such as HAE to have so many treatment options. It looks like the best is still yet to come. We are very much looking forward to it.

HAE Algeria: It can be really important to organize national conferences, and liaise with key stakeholders especially health authorities, media and doctors. It is also important to highlight the significant support we received from HAEi during the creation of our organization. This support was instrumental in our initial efforts, allowing us to network with doctors and pharmaceutical companies during the HAE Maghreb (the western and central North Africa region) conference organized by HAEi.