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Cell and gene therapy is a rapidly growing market that is expected to reach $10 billion in the future. $106 billion by 2033But significant obstacles remain before the field can reach its full potential, said Fran Gregory, vice president of new therapeutics at Cardinal Health.
Cutting-edge treatments hold the promise of being one-off cures if the industry can overcome access and information issues, as well as the prohibitive price tags that have upset traditional pharmaceutical payment models and put the treatments out of reach for many patients. Gene therapies such as Orchard Therapeutics’ $4.2 million Remmeldy and CSL Behring’s and uniQure’s $3.5 million Hemgenix are currently Most expensive medicines Globally, to payers Rethinking traditional payment models.
While cell and gene therapies are often associated with cancer treatments, the more than 20 therapies approved by the FDA also include products for hematology and neuromuscular diseases, including rare conditions with no other treatment options, Gregory said.
a Tufts Medical Consortium The FDA estimates that by 2032, there could be 66 or more product indications approved, and by 2027, there could be 41 disease conditions with cell and gene therapy options. Allogeneic and In vivo CAR-T Options are evolving and may offer advantages over traditional CAR T-cell therapy.
To achieve this, manufacturers need the consent of healthcare providers and patients, which safetyensuring access and affordability.
Barriers to adoption
Many cell and gene therapies are available only through manufacturer-certified providers and facilities — usually academic medical centers in cities far from potential patients — and Gregory said access is limited because patients often have difficulty traveling to treatment facilities.
Further complicating the situation, local doctors may be hesitant to give up on patients and prefer to treat them in their own clinics, and doctors and patients may not even know that treatment is an option.
Fran Gregory, vice president, emerging therapies, Cardinal Health
With permission from Cardinal Health
Patient out-of-pocket expenses also vary widely.
“The situation is really diverse,” Gregory said. “Some patients have low out-of-pocket costs, while others face a huge financial burden.”
Some foundations offer financial assistance, but depending on insurance coverage, some people may not be able to afford treatment.
Manufacturer-sponsored programs don’t help people with government-funded insurance like Medicare or Medicaid, Gregory said, but that’s not the case: Medicaid coverage is determined independently by states, for example, so two Medicaid patients with the same condition may have different access to treatment.
Increasing Solutions
“As the market evolves, we’re finding a lot of solutions to a lot of these problems,” Gregory said.
Among them, the Centers for Medicare and Medicaid Services: Cell and Gene Therapy Access ModelsThe bill, set to be introduced in January, aims to create value-based agreements between states and cell and gene therapy companies to lower prices and create performance-based payment incentives.
“These are fantastic products and in many cases life-changing and life-saving, so we want to continue to support them.”
Fran Gregory
Vice President, Emerging Therapies, Cardinal Health
Gregory supports requiring manufacturers to clarify guidelines about medical necessity, and individual companies like Kite say they Accrediting more community oncology clinicsIt’s where many cancer patients seek treatment.
To prepare products for market, Cardinal Health is taking steps to provide insurers with information about costs, risks and benefits and to help manufacturers work with insurers early in the process, Gregory said.
“It’s very difficult to determine if these products are covered by a particular insurer or a particular health plan when you look online,” she said. “Having clear treatment guidelines and medical necessity guidelines would save a lot of time for providers and patients.”
Innovative payment models They may also allow installment payments or offer guarantees or refunds if treatment is unsuccessful.
“Manufacturers and payers need to work together to come up with ways to mitigate these huge, one-time costs to health plans,” Gregory said.
This field is likely to see significant advances over the next few years.
“We are pleased to see the FDA continue to support cell and gene therapy products,” she said, but progress will depend on support from investors, payers and the medical community.
“These are incredible products and in many cases life-changing and life-saving, so we want to continue to support them,” Gregory said.
