K. Lisowska: For hematooncology patients, access to innovative therapies means "to be or not to be."

At the end of March, the Agency for Health Technology Assessment and Tarification published a list of highly innovative drug technologies (TLIs). It includes only 8 of the 33 technologies considered under the process described by the provisions of the Medical Fund Law. Only two anti-cancer therapies are on the list, and none for the treatment of blood cancers is included in the document. In that case, are no new therapies worthy of funding?

I am unable to understand why this happened. The Medical Fund was a promise of improvement for all oncology patients and was supposed to provide access to modern treatment. Meanwhile, it turned out, none of the molecular indications in hematooncology appeared on the list. As a result, our patients are deprived of the chance for innovative treatment. There are very few of them, so we are very concerned about this, and the question arises as to why they did not receive it.

What does the lack of access to innovative therapies mean for these patients?

For hematooncology patients, it's a matter of "to be or not to be." Every next therapy is a chance to extend life by another months or years. The patient can thus wait until the next molecule is developed to treat his disease. There are not many of us, because blood cancers are classified as rare diseases. Sometimes I get the impression that we are still somewhere at the back of interest. On the one hand, we actually have a great number of drugs available for reimbursement and drug programs, and in this respect we can't actually add anything. The last two years have been very important for our patients in this regard. However, there is still a group of patients who are waiting for access to modern treatment, under drug technologies. These patients number in the dozens every year, and we feel that they are left out of the distribution of access to drug technologies.

Which patients are these? Which of them most urgently need access to modern therapies at the moment?

Patients who are waiting for these drugs are most often those who have already had stages of first-, second- or third-line treatment, but the cancer has returned and they need another therapy - such as mosunetuzumab for patients with follicular lymphoma who have undergone at least two treatments and have had no response or the cancer has returned. At the moment, such patients in Poland no longer have access to modern, effective treatment that could have a long-term effect. Melanoma patients are very much hoping for reimbursement of ruxolitinib , which has already been used for years in myelofibrosis patients. For young myelofibrosis and hyperplasia patients, on the other hand, ropeginterferon alfa-2b is still lacking.

Since last year, we have also been running a support group for patients with aTTP (acquired thrombotic thrombocytopenic purpura); there are dozens of these patients in Poland. They too do not have reimbursed treatment. As I mentioned earlier, medicines for our patients are expensive, and there are few patients in rare diseases. Their voice is simply not heard!

What is the current situation of hematooncology patients.

This year and the previous year have brought major changes in the treatment of acute myeloid leukemia and, in part, in lymphomas. Beneficial changes for myelofibrosis patients came into effect at the beginning of the year. The reimbursement of oral azacitazine in acute myeloid leukemia is also a huge success. We hope for more reimbursements, even though these are very expensive therapies. I think the problems with getting them on the lists come from this as well. There are no cheap drugs in our diseases, but that's why the Medical Fund was created, to make them available to patients.