bluebird bio’s Post

Earlier this week the CMS Innovation Center unveiled the Cell & Gene Therapy (CGT) Access Model – a new demonstration designed to promote the use of outcomes-based agreements (OBAs) between state Medicaid agencies and the manufacturers of newly approved gene therapies to treat severe Sickle Cell Disease with the goal of improving Medicaid patients’ access to these life-changing treatments. My initial reaction: cautious optimism. While the model details released raise as many questions as they answer, one thing is clear – Centers for Medicare & Medicaid Services recognizes the need to modernize coverage and reimbursement policies to match the pace of medical innovation and address troubling health disparities. The CGT Access model could be a path to ensuring SCD patients who rely on Medicaid can benefit from gene therapy. That’s IF Medicaid agencies and manufacturers decide to apply and IF CMS and manufacturers can agree on the terms of a multi-state OBA. I’m excited to see that the model reflects many of the Alliance for Regenerative Medicine’s recommendations. -      First, it’s voluntary. OBAs help payers overcome the risk of a significant upfront investment in exchange for long-term but uncertain benefits; however, they are not the only payment mechanism and may not be appropriate for all CGTs. We’ve stressed that manufacturers need flexibility, not mandates, to support access and innovation. -      Second, it has guardrails to ensure patients have appropriate access: states must “appropriately reimburse” for the gene therapy, align coverage across fee-for-service and managed care organizations, adopt uniform prior authorization standards, and contract with out-of-state providers. If implemented well, common access standards can reduce the administrative burden on patients, their treating clinicians, and manufacturers. -      Third, it enables manufacturers to provide travel and lodging assistance for patients receiving gene therapy and their caregivers and pay for fertility preservation treatment (something federal anti-kickback statute has thus far limited). This will help mitigate well-documented barriers that make it especially difficult for low-income patients and women of color to receive care.   -      Finally, it could create significant efficiency for both manufacturers and states. Many state Medicaid agencies lack the sophistication and resources to develop and administer OBAs, so having CMS do the heavy lifting might make sense. And manufacturers might save considerable time and resources by negotiating with one entity as opposed to multiple states. I’ll be closely following the implementation of the Access Model, and I hope that what unfolds will affirm the transformative value of gene therapies and not devolve into a short-sighted attempt to simply slash prices. ARM will continue to work with our members and CMS to promote sustainable reimbursement for and timely, equitable access to cell and gene therapies.

Sickle cell disease can be debilitating for patients with a severe form of the disease. This includes frequent episodes of joint pains, chest pain and shortness of breath, fatigue, and yellowing of the skin. It can also have life-changing consequences or complications, such as stroke, increased infections, bone damage, and kidney damage. This disease primarily affects individuals of African American descent because of its genetic inheritance and origin. In all, about 100,000 Americans have sickle cell disease. I'm proud that CMS is taking action to help patients with sickle cell disease via the new Cell and Gene Therapy (CGT) Access Model! Learn more here and below: https://lnkd.in/eJkehMsF 👇🏾

A short memory might be good sometime as you forget you did the interview, but it becomes a brutal awakening when seeing it posted on LinkedIn. 😅 #cellandgenetherapy #processintensification #umetrics #ai #ml #designofexperiments #multivariatedataanalysis #advancedtherapies

Learn about today’s top challenges in #CellAndGeneTherapy and how to solve them from Johan Hultman at this year’s #ESACT. Explore more about Sartorius cell and gene therapy solutions: https://sar.to/5t6ox #SimplifyingProgress #InThisTogether #AdvancedTherapies #ESACT2024

From regulatory implications to immunogenicity testing, delve into every corner of the cell and gene therapy landscape in bioanalysis in our podcast series, The new frontier: the bioanalysis of cell and gene therapies. Listen to episode 1 - now live onsite >>> https://hubs.ly/Q02mpdss0

Brilliant session on the role of MSC apoptosis in functional effects on monocyte/macrophages in mediating MSC therapeutic effects @Dazzi_lab @DazziFrancesco @JamesAnkrum and Tracy Heng, excellently chaired by Dan Weiss ISCT, International Society for Cell & Gene Therapy #ISCT2024

On #thisDayInScience in 1989, Dr. Steven Rosenberg and his team made history by successfully transferring foreign gene-containing cells into a human, marking the birth of gene therapy. Today, this technique treats various diseases, including cancers. #GeneTherapy #MedicalHistory Read this post and more on my Typeshare Social Blog: https://lnkd.in/gfj4Jn8X

As the spread and scale of #AdvancedTherapies increases, efficient and effective processes which ensure #patients are referred into specialist centres are essential to avoid any impact on treatment efficacy. Learn more about the UK patient referrals pathways for different advanced therapies, and the recommendations for improvement, in the ATTC Network's upcoming webinar: 'Evaluation of the Patient Referral Pathways for Gene Therapy and CAR-T in the UK' on the 24th January. Register here: https://buff.ly/3NnbYCm #CellTherapy #GeneTherapy #AdvancedTherapy #Patient #Access #ATMPs

I am humbled by the dedication of families seeking life-changing therapies for their loved ones. Patients drive our relentless efforts in researching and developing gene therapies. Recently, a family's sacrifice to join a gene therapy trial for their son's Canavan disease in Dayton, Ohio, showcases the profound impact of clinical trials. Canavan, an ultrarare genetic disease, affects myelin cells covering neurons' axons with a bleak prognosis and no approved treatment currently available. Clinical trials play a pivotal role in evaluating the safety and efficacy of these therapies for potential approval. I extend my heartfelt gratitude to the families for their unwavering trust, the researchers for their groundbreaking discoveries, the clinical scientists for their dedication, and the companies behind the development and manufacturing of these life-saving treatments. Read more about this inspiring story here: [Link to the article] #GeneTherapy #ClinicalTrials #RareDisease #MedicalResearch

Another amazing story of hope and transformation comes from the Children’s Hospital of Philadelphia, where groundbreaking Gene Therapies are offering new possibilities for those living with sickle cell disease. This is more than treating symptoms, it's about rewriting futures and potentially curing a condition once deemed lifelong. Yet, to ensure that these breakthroughs become accessible to all who need them, continued investment in Cell and Gene Therapy research is essential. This is the future of medicine, and it’s within reach. With the right resources, we can turn today’s medical miracles into tomorrow’s standard of care. https://lnkd.in/eHVFn6iV