SMA: What is the future of the drug program?

Kos provided information to MPs, answering questions posed by a group of Civic Coalition MPs. Among other things, the MPs asked about guarantees of funding for the drug program for patients with spinal muscular atrophy (SMA), although in recent months representatives of the Health Ministry have repeatedly stressed that the stability of funding is not at risk - this applies both to patients diagnosed with SMA and those covered by the program. - On behalf of everyone, we ask that funding for the SMA treatment program be permanently included in the NHF budget," said Elzbieta Polak.

Deputy Minister Marek Kos assured from the parliamentary stand that SMA treatment will continue to be funded, while reminding that on September 30 the Medical Fund ended funding for gene therapy, and at the moment funding for the drug program is provided entirely by the National Health Fund. - In 2023, we spent PLN 444 million under the program, and by the end of October 2024 we have already spent PLN 475 million," he said.

More than a thousand people are currently being treated in the program. Several years ago, when the drug program was established (nusinersen was the first to be included), experts estimated that up to more than 50 children with SMA would be born each year. However, the mighty decline in the number of births means that this number is significantly lower, and at the moment it is assumed that 30-35 children will be included in the drug program each year, in whom the screening test will confirm the disease.

The drug program came under scrutiny this week by members of the parliamentary team working on SMA treatment. Experts pointed to two key challenges: first, increasing the vigilance of physicians, especially pediatricians and PCPs, in terms of identifying patients with local mutations in whom screening has not confirmed SMA, but are nevertheless sick. Experts stressed that screening captures 98 percent of cases, which means that its efficiency is extremely high, but this must not put to sleep the vigilance of physicians who are approached by parents with children with neurological problems and markedly delayed motor development. The second challenge is the transit from care in pediatric centers to adult neurological centers - the latter are too few in number and have so far specialized in treating other diseases and other patients. This problem, as emphasized in the discussion, requires a systemic solution, which should be proposed by experts. The task of the Ministry of Health and the National Health Fund, on the other hand, will be to make organizational and financial decisions.