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Around the nation: FDA proposes pathway to approve ultra-rare disease treatments


FDA has proposed a new flexible drug approval pathway to help increase access to treatments for ultra-rare diseases, in today's bite-sized hospital and health industry news from Illinois, Indiana, and Maryland.    

  • Illinois: The American Medical Association (AMA) plans to implement new maternity care coding starting next year. Currently, common procedural terminology (CPT) codes for pregnancy care have been global codes that bundle all medical services a patient receives across the different phases of pregnancy. However, AMA is replacing these global codes with a combination of evaluation and management codes, as well as new code categories that align to specific stages of pregnancy, such as labor management. According to Healthcare Dive, this is the first major change to the code structure for pregnancy services since the mid-1990s and aims to more accurately capture the more complex care patients now receive during pregnancy. "Things have changed over the last 30 years in terms of how women are getting maternity care, how it's being delivered — no pun intended — by OB-GYNs," said Daniel Halevy, an executive at Healthfirst, a nonprofit health insurer in New York. The new maternity care codes will go into effect on Jan. 1, 2027, and it will likely take insurers and vendors months to make the change since the current global codes are deeply embedded in healthcare reporting and billing systems. (Parduhn, Healthcare Dive, 3/2)
  • Indiana: Elevance Health has announced new management changes as it strengthens execution across its healthcare services company Carelon and its Health Benefits organization. Mark Kaye, the company's current EVP and CFO, will now oversee Carelon while Peter Haytaian, EVP and current president of Carelon, will become a special advisor to the company. Meanwhile, Felicia Norwood, EVP and chief health benefits officer, will now lead Elevance's consolidated Health Benefits organization. Health Benefits includes the individual, employer group risk-based, employer group fee-based, BlueCard, Medicare, Medicaid, and federal employee program businesses. "Over the last several years, we have continued to evolve how we operate to meet the changing needs of healthcare and deliver for our customers, members, and clients," said Elevance CEO Gail Boudreaux. "These management changes support disciplined execution and help us move with greater clarity and coordination as we continue to scale." (Morse, Healthcare Finance, 3/2)
  • Maryland: Last month, FDA announced new draft guidance for an approval pathway aimed at increasing access to treatments for ultra-rare diseases. The new approval pathway will provide "drug developers of ultra-rare disease therapies a path to accelerated or traditional approval based on the experience of individuals," according to FDA Commissioner Marty Makary. The proposal specifically focuses on genome editing and RNA therapies, but it may also be applied to other types of treatments. For approval under this pathway, drugmakers must identify the disease-causing abnormality, show that the treatment targets the root cause or proximate biological pathway, rely on well-characterized natural history data in untreated patients, and confirm successful target drugging or editing. "The idea with this pathway, or the plausible mechanism framework, is that it's going to make the approval of these treatments, these very targeted treatments, more efficient, more rapid, and allow companies, when it's appropriate, to enroll a smaller number of patients to generate the evidence for approval," said a senior FDA official. (Frieden, MedPage Today, 2/23; Choi, The Hill, 2/23)

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