The medical costs do not begin to capture the economic burden. Many adults with sickle-cell disease are disabled to some degree, and many have brain damage, making it difficult for them to work. Family members often wind up as caregivers, and so the economic burden ripples outward.

“In sickle cell, we are not providing great care and we are paying a lot,” said Dr. Ted Love, chief executive at Global Blood Therapeutics.

A spokeswoman for Novartis said: “We’ve taken a thoughtful approach to the price of Adakveo, balancing the innovation it brings to the treatment of sickle cell disease, the benefits it can provide to patients, and the importance of ensuring that appropriate patients have access to it.”

But Dr. Sarpatwari is leery of companies’ cost-benefit analyses, which he said are based on limited evidence and assume that the drug makers ought to be able to extract a maximum price for the treatments, without regard to actual development costs or any taxpayer support that may have been involved.

David Mitchell, founder of Patients for Affordable Drugs, an advocacy group, said patients and insurers should not agree to just any price for these medications.

“Drug companies want us to ask this question: What are we willing to pay to ease the pain and challenge of living with sickle cell?” he said. “When it’s your child facing the disease, or your friend in unbearable pain, the answer is ‘anything.’”

But that’s the wrong way to approach pricing, he added, and the more appropriate question is: What amount should drug companies make on these drugs?