Milliman’s Post

New webinars on #CMS' cell and gene therapy access model pilot program! Cell and #genetherapies have revolutionized the disease trajectory for many patients and families with rare diseases. These potentially life-altering treatments also come with a big upfront pricetag. How to appropriately account for these upfront costs in our fragmented healthcare system, how to streamline the contract negotiation process among manufacturers and state #Medicaid agencies - and how to appropriately price these therapies given remaining questions about long-term safety and durability of effect - are big, hotly debated questions right now. The Centers for Medicare & Medicaid Services just announced the details for their first access model pilot, focused on sickle cell disease, to help solve some of these questions and hopefully alleviate access challenges. Yet, many questions about the implementation of this pilot remain. #CMMI held the first webinar discussing the new model yesterday, and is planning another one (for states that contemplate participating in the model) tomorrow. More information about the pilot and the webinar registration link can be found here: https://lnkd.in/eTA2uyVD

September is #NationalSickleCellAwarenessMonth! Did you know that Sickle Cell Disease affects about 100,000 people in the US, with more than 90% of them being African American? Sickle cell disease affects the hemoglobin in your red blood cells, causing them to turn sickle-shaped. This can lead to anemia, pain, infections, and serious complications. It's important to be aware of the symptoms and seek proper testing. But there's hope - new developments in gene therapy and drug treatments could greatly improve the quality of life for those living with this condition. Let's spread awareness and support those affected by sickle cell disease! #SickleCellAwareness #Healthcare #GeneTherapy #CommunitySupport

The US needs a high risk pool for gene therapy. It should be administered by CMS with negotiated pricing and outcomes based contracts. There are over a dozen $2M plus treatments coming on line in the next few years. Many are for rare diseases, but some, like the new treatments for sickle cell disease have tens of thousands of potential beneficiaries. Nine State Medicaid programs are exposed to the tune of $70 to $80 Million each. These drugs are life changing, but priced for lifetime benefit. No single State program or commercial insurance plan should bare these costs in a single year. This is a crisis we can still get ahead of if we act quickly.

The Centers for Medicare & Medicaid Services introduces the Cell and Gene Therapy Access Model. Primarily addressing sickle cell disease, this innovative initiative aims to enhance treatment accessibility for Medicaid enrollees with rare and severe diseases. The model, set to launch in 2025, anticipates transforming the lives of over 100,000 Americans affected by this genetic blood disorder. Chiquita Brooks-LaSure, CMS Administrator, emphasizes the potential of gene therapies in treating sickle cell disease, offering a chance for healthier lives and potentially avoiding associated health issues. This groundbreaking approach not only prioritizes individuals' well-being but also has the potential for long-term cost savings, benefiting states and taxpayers alike. https://bit.ly/3SMm5nl #medicaid #genetherapy

Cell and gene therapies are a growing class of medicine designed to treat complex diseases, including genetic diseases. Through a federal contract, AIR will help the Centers for Medicare & Medicaid Services implement and monitor a new program that makes these transformative therapies more accessible to #Medicaid recipients. This work will test whether this new access model improves health outcomes for people on Medicaid, reduces costs, and fosters health #equity. https://lnkd.in/gNWEbuV3

Cell and Gene Therapy Access Model explained. #HHSSickleCellDiseaseSummit #CellAndGeneTherapyAccessModel #SickleCell #SickleCellAwareness #SickleCellDisease

The current cost of novel therapies for sickle cell disease reaches into the millions, but potential relief may be coming via CMS. They recently proposed an increase in reimbursements for two groundbreaking gene therapies, hopefully increasing access to treatments for the debilitating effects of this blood disorder. Beginning in the 2025 fiscal year, CMS will boost the reimbursement rate from 65% to 75% during the therapies' initial 2-3 year "newness" period. With the new reimbursement strategy, CMS is addressing concerns about the affordability and accessibility of these treatments. This is great news for the estimated 100,000 Americans and others around the world who are affected by this blood disorder. #medicaid #medicare #cms #sicklecell #sicklecelldisease #crispr #cellandgenetherapy #celltherapy #genetherapy #cgt

In recognition of both Retinitis Pigmentosa (RP) and Age-Related Macular Degeneration (AMD)/Low Vision Awareness Month, I want to take a moment to shed light on the reality of living with various forms of visual impairment.     Patients with X-linked retinitis pigmentosa (XLRP), a progressive form of vision loss, typically experience night blindness in childhood and reach permanent blindness by their mid-forties, with no available treatment options.     Patients with geographic atrophy (GA) are typically 60 years of age or older and experience progressive blindness, which may cause challenges in completing everyday tasks like reading, cooking, and driving. Their only available option is monthly or bimonthly intraocular injections that may put them out of commission for several days per year.     At Johnson & Johnson Innovative Medicine, we’re taking a different approach to redefine the future for these patients.      This requires bridging R&D and commercialization to address accessibility challenges, redefining our approach to retina drug delivery through the adoption of transformative gene therapies, and optimizing information-sharing through innovative patient registries.     Looking ahead, we’ll continue to meet patients where they are and unlock the solutions they need and deserve, never settling until retinal disease is behind them.    #RetinitisPigmentosaAwarenessMonth #AMDAwarenessMonth #LowVisionAwarenessMonth  #Retina

New technology add-on payment (NTAP) policy changes in CMS's just-released 2025 Medicare hospital inpatient final rule include: changing the cutoff date for "newness," potentially allowing more therapies to qualify for a third year of NTAP payments; and increasing the NTAP percentage for certain sickle cell disease gene therapies from 65% to 75%. Please contact me if you would like Fortrea's help assessing the impact of changes in the rule. https://lnkd.in/ewAD3D_f #fortrea #consulting #marketaccess #policy #reimbursement #CMS #Medicare #NTAP #IPPS

Are you living with SCD? Do you have a relative, or friend living with SCD? Current Research in the field is greatly promising, not only in the development of new therapies but offering hope for CURES that can be made available to many more people. Learn more about gene therapies and other sickle cell disease treatments: https://bit.ly/3sI8D9t #Geneandcelltherapies #SCDcures #Blooddisorders