The nascent field of gene therapy, which hit its stride this year, has unlocked an opportunity for tackling sickle cell that was inconceivable just years ago, experts said. While hydroxyurea, the de-facto standard of treatment since its 1998 approval, can meaningfully reduce the frequency of pain crises, potentially curative treatments offer hope of eliminating crises altogether.

Over the weekend, ASH released its highly awaited full set of new clinical guidelines for sickle-cell treatment — the most comprehensive slate of recommendations to date — with even more nearing completion.

Since the passage of the Orphan Drug Act of 1983 provided financial incentive for pharmaceutical companies to focus on rare diseases, experts have been troubled by a lack of consistency in evaluation of clinical trials. The new guidelines come as drugmakers such as GBT, Novartis and Bluebird establish themselves in this space, offering researchers a uniform, measurable framework for trials to demonstrate the value of their experimental treatments.

All stakeholders agreed that this is only the beginning. Orphan drugs are estimated to comprise one-fifth of global prescription sales by 2024, according to EvaluatePharma, and blood is the leading therapeutic area by sales and market share.

As with any new drugs, uncertainties on cost loom. But experts swiftly rejected the idea that those concerns should dampen the renewed energy around tackling sickle cell, and said attempts to do so may be rooted in prejudice — given sickle cell’s disproportionate impact on black communities.

Therapies for cancers, cystic fibrosis and hemophilia are routinely priced in the hundreds of thousands and even millions, Osunkwo said. The fact that those treatments are widely celebrated as worthwhile endeavors, while the cost of gene therapy for sickle cell is under a microscope before even winning approval, is “stigmatizing” and rooted in “conscious bias,” she said.

Both Novartis and GBT said they are actively talking with payers to facilitate coverage for their drugs, both priced around $100,000 per year, and are taking steps to shoulder some of the burden with their own patient support centers.

“We should have at least another two drugs, if not more, by next year,” Osunkwo said. “The sickle cell community is riled up, ready to participate in clinical trials and do what it takes to get more tools in their treatment toolbox. And they’re ready to speak out about how unfair health care and research has been to their cause.”



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