Dr Clifford Goodman Discusses the Challenge of Valuing New, Expensive Therapies for Rare Diseases

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Legislation, advancing science, and more sophisticated patient advocacy has led to a proliferation of new therapies to treat rare diseases, and society has begun to indicate a willingness to pay for these more expensive therapies, said Clifford Goodman, PhD, senior vice president and director, Center for Comparative Effectiveness Research, The Lewin Group.

Transcript

How do decision makers need to change how they assess the value of a therapy as more expensive treatments start coming to market? And have you seen any changes in societal willingness to pay for these more expensive therapies?

The emergence and proliferation, largely for the good, of new therapies for rare diseases and even ultrarare diseases, is challenging how we evaluate healthcare, challenging how we even look at matters of cost effectiveness. A few main factors have contributed to this proliferation of therapies for rare diseases.

First of all, is legislation. The Orphan Drug Act has been wildly successful in providing incentives for pharma-bio companies to generate, to scope out, to research, and to generate new therapies for rare diseases. That has been augmented with very well-timed advances in the science. Our understanding of the human genome. Our understanding of how to get to the point where we can actually start editing genes. Advances in immunotherapies. So many great scientific advances.

And the third thing, actually, is patient advocacy. Patient advocacy is becoming wiser, more sophisticated, and better able to negotiate the policy making sphere. So, if you look at legislation, science, and patient advocacy, they’re contributing to this generation of therapies.

Now, we are now challenged to think a little bit differently about how we deal with these. For example, let’s take a look at cost effectiveness. In fact, what we’re learning from our patient advocates, our consumers, and society, overall, is that, in general, society is more willing to pay more for therapies for people who are otherwise in disposed or don’t have access to a workable therapy.

If I’m a patient, or family member, who has one of these conditions, and I know that their life is going to be short because there is no viable therapy out there, I want to have access to some sort of breakthrough therapy. Well, as it turns out, society gets that, too. Society is saying, “We’re more willing to pay.” And, so, you’ve seen groups, in particular NICE in the UK and a few others, including some organizations in the United States, start to say that cost per quality-adjusted life year thresholds can be raised to allow for these new sorts of therapies under those conditions.

Now, that’s very encouraging. We’re still going to have to manage our budgets, and we’re still going to be very concerned about the overall spending across rare diseases. Now, keep this in mind: until very recently rare diseases were just that. They were rare, which means our patient populations were small. We did not have very effect therapies for many of these. And there were fewer rare diseases that had been scoped out at all. Well, what do we have now? We have identified more rare diseases, and we have been able to develop more therapies for these rare diseases. We recognize that these therapies are things for which we want to pay. These therapies also extend the lives of people, so they’re racking up more healthcare costs. Whereas in the past, when a rare disease was a small or maybe not even distinguished blip on the radar screen of a payer, now there are more of them and those blips are bigger. So, we’re going to have to make some tough decisions.

But, overall, I think that society has weighed in on its willingness to rethink cost effectiveness and other criteria for developing and providing access to these new therapies for rare diseases.