Do cystic fibrosis patients have reason to be concerned?

Can it be said that cystic fibrosis patients are well cared for? Do they have a good drug program, good centers where they are treated, is the care comprehensive?

Yes, from my point of view as a patient organization, I can say yes. Patients with cystic fibrosis have been under good care for many years, well coordinated on the model of European models. What is being created in Poland at the moment, i.e. Centers for Rare Diseases, is actually developed much earlier in cystic fibrosis.

The problem is that such a cystic fibrosis treatment center dealing with some number of patients should not be in large medical centers, because of the individual problems of this disease. I'm even thinking here of cross-infection, that there should be special corridors set aside for these patients, which we can't afford in academic centers.

The question is how we, as a cystic fibrosis pilot and actually a model disease unit, will fit into the new laws, regulations that govern us. I hope that the Patient Empowerment Congress, which talks about empowerment, will somehow work positively and we will find a common denominator so that, something that has been worked out for a long time precisely in this unit because of the large population, because of the difficulty of this disease, can then result in better care for patients.

We can say the same with efficiency about the new regulations, the expansion of the drug program. These are great things that are being introduced by the Ministry's decision, but the problem of financing and not being afraid of medical centers to expand this program remains, because, however, it is optional, it is not mandatory.

Hospital directors taking this risk have to reckon with possible financial stagnation, and drugs in rare disease units are very expensive, and in these days, not necessarily fake news that talk about the financial deficit of the National Fund, can cause directors legitimate concern, because, however, it is a few million frozen money with no decision to recover it.

So this shared trust, this shared agility between policymakers, the payer, patient organizations, companies, wholesalers, guarantees good for the patient and great quality treatment. And that's what we would wish for entities like cystic fibrosis, but also for all rare disease entities for which there is treatment, and there are only 5% of them.

In addition to this kind of concern about funding, when it comes to the drug program: are there any limitations here, or could something be improved?

Certainly there are, and certainly it can always be better, as dozens of patients will not receive this treatment, but this is due to the difficulty that is cystic fibrosis. We have more than 2,000 mutations in this disease entity, which means we can't talk about treating a genetic disease that has one mutation and one image. Here we have 2,000 images, so there will always be a shortage, the research is not conducted on the entire group of gene defects, but for this most common group, so there will always be a shortage and those who don't will still reach for this drug until the company expands this.

Of course, there are solutions that we would call for, maybe this will happen soon. Let's hope that also on the occasion of the Polish presidency, our ministry would give such declarations, so that the solutions that the EMA introduces would already be implemented at the local level or expanded patient groups at the local level without the whole procedure, because this would give an acceleration of the introduction of a product that already functions on the market, and only expands its recipient group.