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hard to pick up in a formal measure of their muscles. We had to expand our appreciation of how quality of life can improve even from small changes in muscle function.
It’s also hard to measure long-term effects on families. It’s a burden and a joy: A joy not to have the patients die, but a burden to care for them over the long haul. We worked with companies and patient groups to capture the impact on families. I feel the results are worthy to give to policy makers as they make their own assessments.
X: For Zolgensma, Novartis unveiled new data from its key STR1VE trial in the US last week—and disclosed that a patient death in a separate European trial was under review. (The death “was deemed possibly related to treatment by the investigator,” a Novartis spokesman told Reuters.) Does this change your assessment? [The story has been corrected to reflect that the death under review was in the European trial, not the US trial.]
SP: This is still preliminary, the only information we have is from the company and hasn’t been through peer review. But what we’ve seen so far would not change our conclusions materially. These patients have already benefited from a better outcome than they would have had otherwise. At a certain level of mortality, it’ll still make an overall positive net health benefit—when used in this population type 1. These are patients who have an extremely poor prognosis. But when used in presymptomatic patients—a mix of the most severe and less severe subtypes, it’s a different story. You’d want more data to understand the balance of risk and benefits.
X: One reason ICER and some drug companies disagree on a drug’s fair price is the difficulty in capturing the drug’s social benefits, such as a patient’s increased work productivity, or family members who don’t have to be full-time caregivers anymore. Are you, or is society, coming closer to a standard way to value these positive effects?
SP: I don’t think we’re close to consensus. But we’re having a real conversation, which includes the idea that we might not be able to quantify everything that matters, even if we tried. Luxturna was an interesting case. If it’s important to capture the influence of a treatment that allows people to return to work, does that mean we’re giving more credit to treatments for younger or working-age adults than for older people? That raises many important social and ethical questions. There will be tough decisions and tradeoffs.
X: Spark, Luxturna’s developer, came up with an analysis of Luxturna’s societal benefits. Are more companies trying to do the same? Do you find those analyses reliable?
SP: That’s one beneficial thing I see in the environment. More companies are willing to discuss in the public arena the justification of value and pricing of their treatments. Spark is a good example. We differed on how to judge the quality of life from improved vision. Their suggestions were to use jury trial decisions, and how much people were rewarded if their vision was damaged on the job. But if a neurosurgeon has loss of vision due to a work accident and gets compensated for that injury, is that the real way to value what a treatment would be worth?
There are different ways to do it. It’s a lot better than having a shouting match, with companies arguing for profits that are needed for future innovation versus the other side saying you’re ripping the public off, and so on, back and forth.