Innovation in therapeutics should be weighed against risks

Source / Disclosures

Source:

Blumenkranz MS. Adventures in retinal innovation: Lessons learned from thirty years at Stanford and in “The Valley.” Presented at: Retina World Congress; May 12-15, 2022; Fort Lauderdale, Fla.

Disclosures: Blumenkranz reports being an equity holder in Adverum, BVI, Iveric Bio, Kala Pharmaceuticals, MacuSight, Oculeve, OptiMedica, Oyster Point, Peak Surgical and Presbyia.


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FORT LAUDERDALE, Fla. – Innovation in medicine must be weighed against the risk for patients and physicians, according to a speaker at the Retina World Congress.

Mark S. Blumenkranz, MDMMS, said innovation is not only systematically finding solutions to unsolved problems and unmet needs, but also doing it in ways that are practical and affordable.

Mark S. Blumenkranz

“Originality is necessary, but it’s not enough,” he said in a keynote address. “I think it requires discipline on the one hand and the ability to think both in the box and outside the box equally well.”

Over his 30-year career in medicine, Blumenkranz said he has learned that innovation comes with a few risks.

For patients, risks include safety and cost. Blumenkranz said patients could enter a clinical trial and end up in the placebo group, or the drug under review may have a safety issue.

“People make the case that the only difference between a drug and a poison is the dose,” he said. “You have to really look at what the standard of care is and the availability of alternative options.”

When dealing with untreatable diseases, such as those that could cause blindness in nearly all patients, Blumenkranz said the risks become less important than trying to reduce the cost of therapy or the frequency of therapy administration.

Methods such as cost-effectiveness analysis or incremental cost-effectiveness ratio are commonly used to price drugs, but Blumenkranz said they do not adequately account for risks.

“It’s important to recognize the context under which we assess risk,” he said. “Risk is all about risk and reward and alternatives. That’s really how we ought to be thinking of things. … It’s a good thing that we’re taking it seriously, but again, it goes without saying that with all opportunity comes risk. ”

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