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Rare disease medicines access faces a perfect storm

Published on 18/12/17 at 11:37am

With access to rare disease treatments coming under increasing attention, both in the EU and the US, took the time to speak to two individuals on either side of the Atlantic for their take on the issue.

We first spoke to Patricia Ferland Weltin, Chief Executive Officer and Founder of the Rare Disease United Foundation to gauge the mood in the US – particularly in the light of President Trump’s tax reforms that could hit tax credits for the development of rare disease medicine.

How is the current climate for rare disease treatment and access in the US?

It’s scary. I think we’re really at a crossroads – there’s a lot going on that we should have seen coming a long time ago. I have two kids with rare diseases, so for me this isn’t just what’s happening to the community, it’s what happens personally in my life. The Orphan Drug Act, which is really our only hope for incentivising orphan drug treatments is on the chopping block – I didn’t think I’d ever see this, but here we are.

What is your view on what the FDA is doing to bring rare disease treatments to the patients that need them?

I think that we had a lot of hope for [FDA Commissioner] Scott Gottlieb when he came in. We were happy to see that they cleared up that backlog, which is what they said they would do.

Of course, I think Gottlieb, as a Republican, is going to enforce less regulation – that’s a Republican platform. Do I think that’s a good thing? In the rare disease space it’s different – because we’re talking about so few treatments, I don’t think it really applies to us. For us it’s more about incentives and funding to the FDA so that they can process all these applications.

I was disappointed that they didn’t approve PTC Therapeutics’ Duchenne muscular dystrophy drug ataluren. The approval from Sarepta with eteplirsen, where they worked through the approval process and it was very contentious – that approval paved the way. I think that when they approved it, they were saying: “We can’t use the same methods for orphan treatments that we do for regular treatments.”

How confident do you feel about seeing access to treatments improving in the future?

It’s very difficult to be confident with the climate we’re in right now. For the first time in a long time, with drug pricing and now the Orphan Drug Tax Credit, we’re getting opposition from both sides of the aisle. So, this is not a general backlash against the pharmaceutical industry – now, it’s particularly orphan therapies because they’re so expensive.

I’ve spoken with Senator [Bernie] Sanders’ health aide, and she said that they were very concerned with the high price of orphan drugs and they hate to see patients leave their home because they have to pay for treatments. But what they don’t understand is that the majority of patients are moving homes because they [don’t] have the treatments. And I explained that to her.

It’s expensive not to have a treatment, so, although I agree that pricing is an issue, I think that kind of misguided thinking is going to hurt us eventually, because they’re clearly not getting the message accurately on what this life is like. Our government officials don’t hear enough from patients, I’m the only one working strictly in rare diseases as an advocacy organisation that lives this life.

I’ve had grown men crying to me because they’re losing their homes. For people with rare diseases, it’s more than likely we’d have to leave the state to talk to specialists and get our treatment. And no one is helping to support the regular cost of travel and those kinds of medical expenses. They don’t see that – we’re not sending the right message to Congress about what’s happening to the rare disease population. But they’re willing to go ahead and take drastic measures when they shouldn’t be.

Do you think it is a fundamental misunderstanding of the situation?

Absolutely, Senator Sanders’ health aide had absolutely no response to what I said to her. I don’t blame anyone, because before my children got sick, I would never have imagined how horrible this life is – that my child would get sick, and not only would they not be treated, but they wouldn’t be able to see a doctor who understood it. We’re doing our best to get that message across, and we’ve done a great job with the medical community and the research community, but we have not been able to reach our government.

There are 30 million people in the US with a rare disease, but if you look at the direct impact – and this is a very conservative estimate – I would say that 60 million people are affected. It affects every parent, and it goes further, and often other family members get involved: grandparents, aunts, uncles, siblings are directly affected. The burden is much, much greater than we speak about. But no one is going to believe that this many people can be suffering, and our government is doing a really great job of not acknowledging that suffering, and they’re about to make it worse. They’re ready to just cut the Orphan Drug Tax Credits, which is a huge incentive for therapies.

Do you think the US should pursue an outcomes-based pricing model, rather than focusing on bringing prices down, as Trump has promised?

I was excited to see the CEO of Shire talking about value-based pricing. I think if we don’t start talking about pricing, someone else is going to do it for us. Because Shire is a leading rare disease pharmaceutical company, I think that they’re going to set the tone for pricing in the future with a value-based system.

I think a move towards a value-based system needs to be talked about more and if it’s not, it’s just going to get worse. The reputation of the pharmaceutical industry is not good to begin with – there’s not a lot of trust from the general patient population, but we’re not the general patient population, we’re a completely different animal, and we need to be treated that way.

There are huge price tags on some of these drugs which look outrageous, but we never understand the research that goes into them from the beginning – how it’s so difficult and how difficult clinical trials are in the rare disease space. A drug that only affects 20,000 kids or fewer is a lot different, there’s a lot more expense involved.

Do you think fundamental change is needed in order to effectively tackle these issues?

I think we need to be recognised as a community as a whole rather than each individual disease group, and that’s very, very hard to do because there are just so many diseases. It’s a problem that has so many working parts that there needs to be some kind of agency specific to rare diseases that involves every single stakeholder: payers, pharma, patients, the FDA, the researchers, the medical community. There are just too many unmet needs that it needs to be cohesive and needs to be seen as one issue and addressed under one roof by all stakeholders. We’re definitely at a crisis point here because that has not happened.

How likely do you think the weakening of tax credits for orphan drugs is to pass, and how does this change your outlook moving forward?

I don’t think it’s going to pass in the Senate. I hope it doesn’t, but I do think that we need to see it as a warning that it’s even a possibility. I do think it needs to be taken very, very seriously, and we need to regroup and start rethinking the way we do things so that this possibility doesn’t happen again. Now is the time to act. It can’t be business as usual if this doesn’t pass.

The same week, featured an interview with Simone Boselli, Public Affairs Director at EURORDIS, for his take on the EU climate.


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