Table 1 Quotes of participants' perspectives on the adoption of an alternative financing model in Belgium

Overarching statement for any financing model

E1

1) “If you start lumping orphan drugs together for reimbursement like the majority of drugs, you will not get anywhere. If you adopt one criterium, a one-size-fits-all, people with special health needs that often experience difficulties, for which they do not have any liability, will be excluded. Not due to their disease but by way of distribution. So, if you want to categorize that disease, you will have to approach it in a different way. Otherwise, they do not stand a chance.”

Insulated orphan drug fund

E1

2) “An insulated fund would allow separate rules, considering rarity and the challenges of orphan drugs. Is it the only way to do so? I am not yet convinced myself. But it is a way to deviate from the standard that is applied elsewhere and that is certainly not fitting for assessment of orphan drugs.”

3) “If you consider a privately financed fund, you make patients dependent not on the government, something well-structured, but on the goodwill of the industry. You would wonder whether that is appropriate for the most vulnerable to be at the mercy of a private fund rather than public policy.”

D1

4) “How would you compare the reimbursement of a third-line oncologic that prolongs one patient’s life with four months compared to another therapy with five candidates that would obtain a different life quality? Through the same fund? You would still have discussion, isn’t it?”

5) “Does everyone get access to the fund or do people with five million euros in their bank account get the same treatment as someone who is unemployed?”

6) “The core is that it is such a mixed population with variations of pathologies. Secondly, for rare diseases and their therapies, there are often many uncertainties. Those are two main elements that you should cover. That would be challenging with something that is fixed for ten years. You will always need something dynamic.”

7) “If an orphan drug is prescribed in two or three centers, the files are always complete. It becomes a problem when a particular orphan drug is prescribed in 40 to 50 centers, because then there are a lot of people who have little experience prescribing some of those drugs which makes the files worse. A mechanism should be thought of to limit orphan drug treatments to one or two centers.”

I2

8) “I don’t think pharmaceutical companies would favor sponsoring such a fund. Instead, we should plead for an expansion of the medication budget to help as many patients through standard procedures.”

E1

9) “If you adopt a system of fair pricing, it would simplify making more budget available. If a disease is life-threatening and of high medical need, those fair prices could be set higher.”

Emergency fund: The Special Solidarity Fund (SSF)

C1

10) “The SSF should remain an exception. It is case-by-case, so really not for everyone with a rare disease. It is to answer needs when there is no structural solution or to give access to medicines not yet registered. For adults with a rare disease, costs are never fully covered. It is also a limited budget that could run out.”

I2

11) “It is a very laborious administrative procedure. Patients need to apply, usually with help of the hospital’s social services, but it is usually not applied for due to the tediousness and the significant delays.”

Private insurance

C1

12) “Access will not be uniform. Since, in Belgium, we highly consider the principle of solidarity, I do not think it is applicable in our country.”

E1

13) “This will de facto lead to a two-track policy. There would be decent public healthcare but the moment it becomes expensive and inaccessible, it would be handed over to private insurance. Making it accessible to only those who can afford it or who are administratively resilient enough to arrange it.”

14) “If everyone participated, that amount would remain relatively low, but if, for example, only 10,000 people register for it, that paints another picture”