Enar’s nephew, Behram,1 was just a newborn when he was diagnosed with spinal muscular atrophy (SMA) Type 1, a rare genetic condition where nerve cells in the spinal cord and brainstem do not work properly, resulting in progressive muscle atrophy and weakness. If left untreated, Behram was unlikely to live past his second birthday, and there was no available treatment for him in Kosovo, where they live.
“Behram’s muscles were failing, he was moving less and less. It was heartbreaking. The family felt we were on the edge of disaster,” said Enar. She sent a letter to Project HOPE, an international global health and humanitarian aid nongovernmental organization with a focus on children’s health. “I can’t watch my baby nephew die,” she wrote in the letter. “I can’t say ‘goodbye.’”
Project HOPE issued a request for support. Biogen responded, knowing any further delay in treatment for Behram could mean significant disabilities, and potentially death.
We worked across functions and levels to orchestrate the operations needed to provide treatment in a country where Biogen had previously had no presence. Adding to the complexity, SPINRAZA® (nusinersen) is not the kind of medication anyone can pick up over the counter. Treatment requires an intrathecal injection administered by healthcare professionals. We were tenacious in finding a way to quickly deliver SPINRAZA to a hospital in Kosovo, supplying the medicine that Behram urgently needed.
“The combination of varied healthcare systems and delayed or insufficient access to therapies in some countries can be a challenge for patients and caregivers. It is humbling to see the passion of physicians who fight for their patients to receive the best possible care,” said Carmen Walbert, Biogen’s Medical Head for Europe, Canada and Partner Markets.
Although more than 14,000 people globally have been treated with our medicine,2 Behram was the first person with SMA to receive SPINRAZA in Kosovo. Biogen has been providing access in countries around the world where our medicines can make a real difference. With these efforts, we aim to help tackle disparities in treatment access across age, gender, race, ethnicity and socioeconomic levels. We have made SMA treatment available in 69 countries worldwide, including 19 low- and middle-income countries. Our SMA humanitarian access project in India is reaching 200 infants, and we are delivering solutions to other people worldwide who would not have otherwise had access to treatment.
"Biogen’s approach since the first approvals of SPINRAZA has been consistent, underscoring our commitment to the SMA community. We are passionate about expanding access to our treatment for patients in need.”
- Adrian Harrington, SMA Value & Access Lead
“Biogen’s approach since the first approvals of SPINRAZA has been consistent, underscoring our commitment to the SMA community. We are passionate about expanding access to our treatment for patients in need,” said Adrian Harrington, SMA Value & Access Lead. “In addition, we work with local clinics to ensure that SMA care standards are optimized, so that patients can get the best outcomes from treatment with SPINRAZA.”
"There are many underserved and underrepresented groups around the world who are living with debilitating diseases and insufficient access to treatment. As part of our credo of caring deeply, we aim to help address the unmet medical needs of these patients and improve their standard of care," said Maha Radhakrishnan, Biogen’s Chief Medical Officer.
We are working with our global partners to access disparate and hard-to-reach patient populations who have felt left behind. Engaging directly with local medical experts and centers of excellence in SMA helps us to evolve our thinking and approach to topics such as treatment objectives, patient profiles and measures to assess treatment effectiveness. Communicating with patient advocacy groups also helps us understand patient needs and to account for potential concerns of patients and their caregivers.
Biogen is committed to helping to improve outcomes for patients, like Behram, in the disease areas we treat. His treatment is lifelong, so his care team is taking things at his pace – and he is getting stronger every day. Enar said, “In the future, I hope to see him going to school, being more independent. He now has a shot at living a more normal, happier life.”
1Names have been changed for privacy and protection.
2Based on commercial patients, Early Access Program patients and clinical trial participants through December 31, 2022.
Note: People must meet specific criteria to receive access to Biogen’s therapies through compassionate use based on humanitarian grounds or through our humanitarian aid program. Biogen’s increasing global operational footprint facilitates reaching more patients and helps us better understand local needs. Learn more about our Access programs here.