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Valuation Challenges and Ethical Implications of Cures
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Valuation Challenges and Ethical Implications of Cures

Laura Joszt
As new treatments come to market that have a substantial impact on diseases, or even cure them, the healthcare system is facing the challenge of how to value these treatments. A panel of experts highlighted what evidence there needs to be, methods of valuing therapies, and the ethical implications of having cures.
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As new treatments come to market that have a substantial impact on diseases or even cure them, the healthcare system is facing the challenge of how to value these treatments. A panel at the International Society of Pharmacoeconomics Outcomes Research 23rd Annual International Meeting, held in Baltimore, Maryland, highlighted what evidence there needs to be, methods of valuing therapies, and the ethical implications of having cures.

At the start of the panel, John Watkins, PharmD, MPH, pharmacy manager, Formulary Development, Premera Blue Cross, highlighted the questions payers have regarding cures with high prices, such as what is the evidence, is it really a cure, is the effect durable for a lifetime, and can the price be justified?

The evidence will be dependent on the population available, he said. There will never be a large randomized controlled trial for an ultrarare disease. In addition, whether or not the treatment is truly a cure needs to be clear. Can the patient go on to live a normal life? For instance, can a child who is cured of hemophilia A go on to play football like any other child?

One of the biggest challenges is knowing whether the effect is durable for a lifetime since all follow-up has been for a fairly short period of a few years, so far. Which means payers have to extrapolate those results for a lifetime.

“We’re still looking for the invention of the time machine so that I can go forward to a 50-year horizon and observe what happens to the person…” Watkins said.

In addition, the definition of value has become overcomplicated, stated Mark Sculpher, PhD, professor of Health Economics, Centre for Health Economics, University of York. There is the value of hope and the value of assurance, but, in his opinion, there are no technical answers to those things. They can be determined differently in different health systems.

He also discussed one way of paying for these expensive treatments through a smoothing of costs over time. Instead of having a large cost upfront, there would be loans from the government or mortgages for manufacturers to smooth the short-term costs for future health gains.

“We’re going have profound evidential uncertainty when we’re making initial decisions about funding: ie, at the launch of these products,” said Sculpher. “What we’ve seen over the last 5, 10 years is the evolution of quite nuanced policy responses to that.”


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