Dr. A. Falek "New option in SMA treatment supports optimization of care"

Poland has been one of the leaders in the area of diagnosis and treatment of spinal muscular atrophy (SMA) for several years. We have both an extensive nationwide newborn screening program and a state-of-the-art drug program. Thanks to screening, it is possible to detect SMA in the first days of a child's life, which allows treatment to begin even before symptoms of the disease appear.

"SMA treatment in Poland is very well secured. We have a well-functioning drug program, and we no longer think about whether to treat patients, but how to do it in such a way that it is most convenient and with the best effect for patients and the system. We can say that the solution in Poland is a model. Now we are getting to the stage where new technologies are emerging and we are evaluating their systemic usefulness," Dr. Artur Fałek stresses.

Tablets as next step in drug program development

The introduction of an orally administered drug - in tablet form - into the drug program will be another step in improving SMA therapy. Although the therapeutic decision always remains in the hands of the doctor, the new form can significantly facilitate the daily functioning of patients and their families.

"The route of administration of the drug is important, but clinical circumstances are decisive in the first place. It is on the basis of these that the doctor selects the therapy. The introduction of an oral drug in the form of tablets, which - due to their pharmaceutical properties - have a longer shelf life than the oral solution currently used, will mean that the patient will be able to be supplied with the drug for a longer period of time, even six months. Patients won't have to show up at the treatment center just to pick up another dose of the drug from the hospital pharmacy, but will be able to come when the control schedule requires it. And in the SMA drug program, mandatory follow-up visits are scheduled every six months." - Dr. Artur Falek explains.

Benefits for the system: savings and reduced burden on centers

A positive recommendation by the President of the Agency for Health Technology Assessment and Tarification in October of this year regarding reimbursement of the tablet form shows the potential for savings for the payer.

"If the Agency sees such savings, there is no reason to undermine their calculations. If a form appears that carries the opportunity for savings, the greater the opportunity to include new programs, new drugs and patients. Let's remember that children with SMA are still being born, so patients in the drug program will increase. The savings are due to several reasons. Oral treatment - especially in tablet form - significantly reduces the number of hospitalizations required for other forms of therapy. The cost of an outpatient consultation, during which the patient is given the drug for use at home, is much lower than the cost of a one-day or multi-day hospitalization. In addition, the tablet form of the drug does not require the involvement of a hospital pharmacy," says Dr. Falek.

As the expert points out, cheaper treatment offers the chance to increase the financial capacity of the National Health Fund, and thus make it easier to select therapies and include new SMA patients in treatment. Equally important is the fact that the funds left in the NHF budget provide a potential opportunity to launch further innovative drug programs for patients with diseases other than SMA.

The decision is up to the doctor

The introduction of an SMA course-modifying drug in tablet form will not change the basic principle that the choice of therapy remains the decision of the treating physician.

"Patients who have been on the therapies in question will continue them, as long as there are no contraindications to their use. But there is a new, convenient option that may be the reason for choosing oral therapy," adds Dr. Artur Falek.

Thus, the new form of the drug can broaden the range of therapeutic options, ease the logistics of treatment and benefit both patients and the health care system as a whole.

Source: Press Release