Wonderfully succinct alternative payment model description on STAT.
We’ve developed fuller descriptions in NEWDIGS FoCUS at https://lnkd.in/dW7qyxxV. And you can calculate the financial impact and design your favorite solution using our “Individual Indication Workbook” at https://lnkd.in/d7ttUbNa
The outlook: With creativity we can provide equitable and affordable access.
Anywhere from 30,000 to 40,000 sickle patients on Medicaid might be eligible for one of the new sickle cell gene therapies. But their cost could prove to be an issue. https://trib.al/0J59l3U
This is a very interesting article. It highlights the need for full reinsurance protection for Gene Therapies. Just having protection for the cost of the drug or blood is not enough. Employers and captives need protect for the full episode of care, in this case several months of inpatient
care.
https://lnkd.in/gxruMCmK
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
September is #NationalSickleCellAwarenessMonth! Sickle Cell Disease (SCD) is the most common inherited blood disorder in the U.S., disproportionately affecting Black and Latino communities.
In late 2023, the FDA approved two groundbreaking gene therapies for SCD—Casgevy by Vertex and CRISPR Therapeutics, and Lyfgenia by bluebird bio—offering new hope for those living with this condition. While this is a huge step forward, there's still work to be done to make these treatments more affordable, less burdensome, and accessible for all.
Learn more about these advancements: https://lnkd.in/gBE9jEFp#raiseawareness#yourCBHN#blackhealth#healthequity#jointhefight#healthcare
Biotech | Medicine | Translational | Market Intelligent | Business Dev. | Help Each Other Success | Med Device | The Community Guy | Speaking for himself
First autologous CD34+ hematopoietic stem/progenitor cells therapy, genetically modified by Lenti-transduced ASMA gene, approved today by FDA to treat Metachromatic Leukodystrophy (MLD).
The trial recruitment of MLD is in EU, https://lnkd.in/eZ3PpuHZ
The trial sponsor published the Ph1/2 clinical trial result in Lancet in 2022: https://lnkd.in/eRejuC_J
This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from mutations in the gene encoding the Arylsulfatase A (ARSA) enzyme. The medicinal product consists of autologous CD34+ hematopoietic stem/progenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors.
Treatment with arsa-cel resulted in sustained, clinically relevant benefits in children with early-onset MLD by preserving cognitive function and motor development in most patients, and slowing demyelination and brain atrophy.
Congrat toUK based Orchard Therapeutics
Today, we approved the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). https://lnkd.in/epcpJtrT
MLD is a debilitating, rare genetic disease affecting the brain and nervous system caused by a deficiency of an enzyme called arylsulfatase A, leading to a buildup of sulfatides or fatty substances in the cells.
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
Love your comments! Potential candidates and those who pay for them need to be empowered with:
1. Today’s Gene and Cellular Therapies are not shown to be curative, but temporary. Side effects can be fatal and most certainly risk infertility.
2. Should the recipients go on to be able to have biological children, they should still be expected to pass down the mutated genes.
3. Under most circumstances, today’s gene therapies are not superior to current standards of care.
4. Once a gene therapy is received, we are ineligible to receive a newer, better one in the future, for at least 25 years and counting.
5. There are grand rebates that are being returned to the carriers, but aren’t being passed-thru to the actual payers for the 80% of self-funded employers.
6. Manufacturers also have outcome based contract or warranties that again are returned to ASO carrier and not payers.
**It is a fascinating and inspiring time in medicine, but we have only started growing mold on a Petri dish, we have not created the penicillin of gene therapies yet.
medTRANS Insurance, LtdComplete Captive Management Services
Founder/Chief Medical Officer at Advanced Medical Strategies
Gene therapy is often thought of as a "miracle cure". Some of these treatments are extremely ardous to go through; case in point; gene therapy for sickle cell treatment. Almost 1 year after approval, ~30 patients have begun the process to receive it. This article details the first commercial patient to receive it.
I'm always asked how adoption rates will be for these multi-million dollar therapies, especially since many of them are given to children. The side effects will give many parents reason pause and consider moving forward.
https://lnkd.in/gsz3CEND
Cell and gene therapies are revolutionizing how clinicians treat previously intractable diseases such as cancers, blindness, and metabolic disorders. In this tech note, examine integration site analysis techniques that are a critical component of patient safety testing of these therapies: https://hubs.ly/Q02LmRR20#ClinicalTrials#CellTherapy#GeneTherapy#PatientSafety
#CureSickleCellNow
There are many misconceptions about sickle cell disease.
First, social determinants of health have a huge impact on disease burden. This was and is a health disparity.
Second, many believe that it has now been "cured" by gene therapy and gene editing. The reality is that most patients do not have access to care where they could become eligible these treatments, and only a small percentage meet eligibility requirements.
<50% is not a cure.
Unfortunately, these misconceptions are also common among expert reviewers for research funding. I quote the words of a reviewer in talks now from a grant application submitted just before the approval of the genetic therapies...
We study transfusion related complications in sickle cell disease. Most adults have these complications!
Huge news for sickle cell warriors and the blood and biotherapies community:
The U.S. Food and Drug Administration approved the first two gene therapies to treat sickle cell disease on Friday.
These single-dose therapies are approved for adult and adolescent patients ages 12 and older. https://bit.ly/3RdHaFe#SickleCell#GeneTherapy#SCD#Biotherapies
Today, we approved the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). https://lnkd.in/epcpJtrT
MLD is a debilitating, rare genetic disease affecting the brain and nervous system caused by a deficiency of an enzyme called arylsulfatase A, leading to a buildup of sulfatides or fatty substances in the cells.
