When Geography, Cost, and Policy Become as Life-Limiting as a Disease

When Tom Sayiner was diagnosed with the fatal neurodegenerative disease ALS, he and his wife, Tamara, learned that tofersen had been approved in Europe as a therapy that could slow the progression of his genetic form of the disease. But the Sayiners, who live in Sweden, soon discovered they could not access the drug because a Swedish health technology assessment council determined there was insufficient data to demonstrate that the costly therapy provides a clear benefit. Instead, they are working to raise funds to purchase the drug in Germany, while Tom has also enrolled in a clinical trial in hopes of accessing a potentially beneficial therapy. The Sayiners discuss the gap between regulatory approval and real-world access in Sweden, the steps they have taken to explore cross-border care; and how geography, cost, and policy can be as life-limiting as the disease itself.