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The rare disease climate in Europe

Published on 21/12/17 at 10:05am

Continuing on from this week's earlier feature on the rare disease situation in the US, Pharmafile moves its attention onto the debate in Europe – by speaking to Simone Boselli, Public Affairs Director, EURORDIS.

There are now a lot of discussions occurring at a country-level regarding the price of medicine – those for rare diseases, in certain examples, can be at the higher end of the scale. How is this impacting patient access?

Of course, access implies affordability and availability – in this sense, we do see an impact. We see this through our own research, many patients within the rare disease community are having difficulty accessing treatment but also accessing care. It’s all together.

We have found, on the treatment side, that there are a lot of misconceptions between manufacturers, payers and the rest of the community. In the public, there is sometimes the idea that any orphan drug that comes to market always bears a hefty price tag and that this is unsustainable; the reality, which we are outlining, is different. A study emerging from the UK, for instance, found that only 20% of drugs for rare diseases cost more than €100,000. We found, in general, across Europe an average of therapies’ listing price being a more reasonable figure of between €30,000 and €40,000 per patient – and, as we know, this listed price is not often paid after negotiations.

When you take into account the fact that medicines for rare diseases are, by nature, done for a smaller population and with far more obstacles to developing treatments, for instance, for clinical trials, it’s a very different picture of the pricing landscape.

Starting from these assumptions, we do also acknowledge that there can be some problems, in the sense that there are some issues with the pricing strategies that are employed by the industry, in certain cases, that are straining the relationship with the payers. The major problem there is the lack of data, and available data, at the time of marketing authorisation and pricing reimbursement discussions because many of the new drugs are based on small pools of patients.

Is the issue then a few individual cases of aggressive pricing strategies that are creating an impression of a wider problem?

There are two phases to this story: the question of high prices began seriously with the ‘poster child’ for the wider high prices story – the case of [Gilead’s] Sovaldi, which triggered a much greater discussion on pricing. The problem with an aggressive pricing strategy is finding out what the market can bear and pricing their drugs accordingly, rather than looking at the value the medicine is bringing.

What we are advocating for is that there needs to be greater transparency on how the price is set, as opposed to just stating: “This is the price we have set”. In some cases, we have seen that the prices of well-established medicines have been raised by four or five digit figures and this is not understandable, if you ask me. In these scenarios, the price is set for investors rather than for public health purposes and this is not the norm, but they are the ones that are capturing the imagination of the public.

How do you work to separate the impression that these prices can become linked to rare disease advocacy?

Headlines are always looking at the list prices and certain drugs can have in excess of six figures, and it does make good headlines. However, when you look at the total budget impact in countries across Europe – for example, if you look at Italy – rare disease drugs make up just 0.6% of the total pharmaceutical budget. When put in perspective, more or less across Europe, that has also been quite stable for a number of years and a few published studies have pointed towards the costs having plateaued.

When we look to the future, when we talk more about advanced therapies – for example, gene therapies or cell therapies – we see prices that are well in excess of €100,000. However, if you look at the recent news from NICE that approved gene therapy for the bubble boy condition by GSK, it has a price of €594,000 but was deemed cost-effective. Of course, there are only 15 patients that are born with this disease so the overall budget impact is relatively minimal but the value that is brought to the families involved is huge.

With such improvement, what are the hurdles that are still in place to improving access?

Contrary to what has happened in the US, in Europe, we have a fragmented market; we have 28 different markets and we see that, in terms of access, for orphan drugs then there is an 18 to 24 months delay between market authorisation and full access. That is quite significant and where we see the real problem – what is available from a regulatory stand point and then from a pricing and reimbursement standpoint. The regulation, per se, is not so much the problem – yes, it can be ameliorated, but the problem lies more downstream.

If we have a fair understanding of the problem, what do you believe are the solutions?

The solution is in early dialogue between all stakeholders. You see that from a regulatory standpoint there are now more early dialogues taking place between them and manufacturers to arrive at an appropriate pricing strategy. Companies are now beginning to look at the data whilst discussing with the regulator what, further downstream, the HTAs and payers will want to look at. I think there’s an opportunity that we can speed up access through this.

I have seen in recent research that UK is not that bad at providing access to rare disease medicine compared to other countries – it’s just too long for everyone. If you’re at 18 months, that’s almost two years to gain access to an approved medicine.

Another angle that has to be looked at is increasing flexibility, particularly from manufacturers and payers, in the type of agreement secured. For instance, we could have a type of agreement that provides a certain amount of the price upfront, but then the rest can be linked to agreed endpoints where data can enter and where they can justify the price that has been asked in the first place. What is the problem is that there is not much experience of these flexible approaches; we see some companies and some payers being innovative, but it is not being seen across Europe.

You need to look at the difference in the manufacturing landscape. Novartis can afford, with its strength, to be a bit more innovative and spread the load. When we are looking at smaller companies that are sometimes mono- or bi-product, it is much more difficult to do. However, payers may potentially gain confidence from the decision to discuss with Novartis or other companies’ alternative models of payment – that is a hope we have.

How much influence do patients now have in the development and on access issues, regarding orphan drugs?

We see now that the patient’s voice is being meaningfully taken into account and our community has always been aware of the importance of being involved, being educated and being able to provide meaningful contribution that goes beyond our own experience with a disease. We see that that shift is fundamentally happening as we observe how medicine is delivered, distributed and the attention that is being given to side-effects. This latter point means that attention is being paid to make therapies be more bearable. One of the things that we are putting forward is the creation of community advisor reports from patient associations and looking to expand over the next two years. It will provide patient communities with the opportunity to dive into a dialogue with manufacturers.

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