The US department of Health and Human Services (HHS) will look at new ways of fast-tracking approval of drugs for rare diseases, as health secretary Robert F Kennedy Jr (RFK Jr) called out the current US healthcare landscape that profits from life-long treatments.
“We are going to continue to figure out new ways of accelerating approvals for rare disease drugs and treatments. We are going to make this country the hub of biotechnology innovation,” RFK Jr said during a US Food and Drug Administration (FDA) roundtable on cell and gene therapy on 5 June.
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Those in attendance included cell and gene therapy experts, industry representatives, and US health agency chiefs.
Cell and gene therapies, many of which are a one-time treatment, have marked a significant advancement for certain rare diseases. This modality can offer long-lasting and even permanent treatment for certain conditions. The approval process for cell and gene therapy candidates is tricky, however, given their high-cost and oft-debated benefit-risk ratio.
RFK Jr did not question their importance, and acknowledged panellists’ calls for faster regulatory processes.
“We’re going to do everything in our power to sweep away the barriers from getting those solutions to market,” the health secretary stated.
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By GlobalDataShares in cell and gene therapy-specialist companies rose in afternoon trading on 5 June. Sarepta Therapeutics, developer of gene therapy Elevidys (delandistrogene moxeparvovec-rokl) for Duchenne muscular dystrophy (DMD), was up 1.79% in pre-market trading on 6 June.
RFK Jr said that funds freed from recent cuts across the HHS would be redirected to “spawn, foster, and fortify innovation”, ensuing the US does not lag behind other countries in drug development.
“We want to make sure we keep this country competitive with all other nations, a hub of this kind of technology. We want to make sure we are getting rid of the regulatory impediments [that stop drugs getting to market],” RFK Jr stated.
Scalability and cost hurdles
Also at the proceedings was National Institutes of Health (NIH) director Jay Bhattacharya. His views on the treatment modality are important, given he oversees, traditionally, the largest funder of biomedical research in the world. Bhattacharya centred his remarks around sickle cell anaemia. Though he did not mention the products by name, the two FDA-approved treatments for this red blood cell disorder are Vertex Pharmacueticals’ Casgevy (exagamglogene autotemcel) and bluebird bio’s Lyfgenia (lovotibeglogene autotemcel).
“I never imagined in my lifetime we’d have the capacity to cure sickle cell anaemia,” Bhattacharya commented.
Bhattacharya did, however, allude to the difficulty in scaling cell and gene therapy products – an area he wants changed.
“The challenge is how do we make these advances scalable and affordable,” the NIH director said.
Bhattacharya said that cell and gene therapy products are financially acceptable based on life-long models for a patient. Treatments have been criticised for their ultra-high price point, often costing millions of dollars, though evidence suggests that this is still less than the financial burden the disease creates over a patient’s lifetime.
“It will take a regulatory framework that makes sure that the [therapies] that do advance for patient care are the ones that are actually safe and have excellent evidence behind them,” he added.
In one of the fierier remarks from the roundtable, RFK Jr said: “At every level, doctors, other providers, our hospitals, the pharmaceutical companies, the insurance companies, all make money by keeping us sick. We need to fundamentally change that so that we are focusing on curing disease and making people better.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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