SMA: Members are asking, the ministry is responding

In recent days, Marcelina Zawisza (Left) informed about the submission of an interpellation by a group of deputies. The deputy pointed out that although the very decision to finance Zolgensma from public funds (the so-called gene therapy) for children up to 6 months of age is great news, it is doubtful that older children who meet the body weight criterion provided by the manufacturer could also benefit from treatment. - This is what is done, for example, in the United States, where cases of administering the drug to children up to 2 years of age are described - she reminded.

But the MPs of Razem put questions to the ministry beyond the dispute over the age criterion in the drug program. They want to know, among other things:

- Due to the fact that children over 6 months of age are not included in the B 102 drug program, does the minister of health envisage a separate path for financing Zolgensma treatment from public funds in justified cases of patients over 6 months of age;

- In this case, in the opinion of the ministry, is it possible to reimburse the Emergency Access to Drug Technologies (RDTL)?

- In such a case, in the opinion of the ministry, is it possible to finance such treatment from the therapeutic and innovative subfund of the presidential Medical Fund?

They are also asking for answers to the questions to which the Ministry of Health, at the time of announcing the decision to cover two new drugs for SMA, answered at least partially. Minister Adam Niedzielski said, for example, that there would be no double funding (gene therapy and nusinersen). However, members of the Left want to know whether in the case of children qualified for treatment with Zolgensma, when the response of the body after administration of the drug is weak or no, it will be possible to treat them with Spinraza (nusinersen) or Evrysdi (risdiplam), whether the administration of Zolgensma will completely block it, and whether For children who have received or will receive Zolgensma on a non-reimbursed basis, it will be possible to continue the treatment with nusinersen that has already been started.

This week, the first responses of the Ministry of Health to the interpellations submitted by MPs on this matter in August were published on the parliamentary website. Deputy Minister Maciej Miłkowski, responsible in the ministry for drug policy, in response to an interpellation by Katarzyna Osos (KO) - the deputy asked, inter alia, o the possibility of extending the inclusion criteria to the drug program - explains the substantive basis on which gene therapy reimbursement is based. Reading the explanations suggests that the Ministry of Health does not intend to open any gate.

- In conclusion, it should be emphasized that the Ministry of Health pays a lot of attention to the problems of patients suffering from SMA, which results in systematic implementation of solutions for this group of patients. At the same time, the wealth of the state budget and the enormity of health needs of Polish citizens should be taken into account, which makes it impossible to meet the expectations of all patients. However, acting within the framework of the applicable regulations and carefully balancing the interests of all patient groups participating in reimbursement, as well as placing special emphasis on the discipline of public finances, multidirectional measures are taken to provide patients with the greatest possible number of treatment options. At the same time, it should be emphasized that in implementing the state's drug policy, the Minister of Health follows the principles of evidence-based medicine (EBM-Evidence-Based Medicine) and health technology assessment (HTA - Health 10 Technology Assessment), which ensures transparency and rationality of decisions made. on the allocation of public funds - we read in the reply of the deputy head of the Ministry of Health.