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In a response to a survey, caregivers of people with spinal muscular atrophy identified the risk of severe adverse events and the need for permanent ventilation as the most important factors in treatment decisions. Access to treatment, including cost and availability, ranked third.

What Do SMA Caregivers Want?

Keith Loria is a contributing writer to Medical Economics.

Mouse models of liver, kidney and lung fibrosis showed that genetic deletion of MERTK prevented fibrosis

MERTK Inhibition As a Catch-All Antifibrotic Strategy

If approved, donidalorsen would be a first-in-class RNA-targeted medicine for hereditary angioedema. The agency’s goal date is August 21, 2025. 

FDA Sets Goal Date for Donidalorsen in HAE

Here’s what you missed this week on Managed Healthcare Executive.

MHE Week in Review – Taming Healthcare Costs, Gene Therapy Coverage Challenges and More

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, insights, and strategies to pursue value-driven solutions.

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Hub services are expanding functions to facilitating access to needed therapies or devices and addressing barriers to adherence.

Pharma’s Role in Addressing Social Determinants of Health

Studies show that prolonged mechanical ventilation (PMV) is associated with many financial and clinical risks, including, higher rates of ICU-acquired weakness (ICUAW) and readmission.[i]

Researchers attempting to address this dilemma have found that transferring PMV patients to settings that specialize in ventilator weaning could be beneficial for those whose conditions are otherwise stable.

Clinical and Financial Outcomes at Kindred Hospitals

Kindred Hospitals provide specialized acute care and rehabilitation for medically complex patients, and have a particular expertise in treating ventilator patients

Data show that the average 30-day readmission are significantly lower at Kindred than at traditional inpatient hospitals for Diagnosis-Related Groups (DRGs) 003, 004, and 207 with prolonged mechanical ventilation

DRG 207: Respiratory system diagnosis with ventilator support >96 hours.

DRG 003: ECMO or tracheostomy with MV >96 hours or principal diagnosis except face, mouth and neck with major O.R. procedures.

DRG 004: Tracheostomy with MV >96 hours or principal diagnosis except face, mouth and neck without major O.R. procedures.

At Kindred, our high-level staffing allows us to accept critically ill patients on mechanical ventilation once they have reached a stable condition. Pulmonary patients benefit from physician-led interdisciplinary care, pulmonologist consultations, 24/7 access to respiratory therapy, and physical, occupational, and speech therapy.

We also have established care initiatives that promote recovery, including:

Early mobility for all patients, even those on mechanical ventilation

Disease-specific care certifications in Respiratory Failure from The Joint Commission

At a Kindred Hospital, PMV patients benefit from specialized and efficient care in a facility focused specifically on their needs. 

With the significant risks associated with PMV, transferring these patients to a Kindred Hospital once they are stable is an important strategy in improving patient outcomes, and reducing readmission risk and cost.

To learn more about how we can help reduce readmissions, visit kindredmanagedcare.com



https://pubmed.ncbi.nlm.nih.gov/26242743/

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7515670/

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6852505/

https://pubmed.ncbi.nlm.nih.gov/28403404/

 The Advisory Board. The Post-Acute Care Pathways Explorer: State Average Outcomes by MS-DRG, Accessed August 2024; Systems Outcomes Report, MS-DRG, Kindred Hospital Medicare IDs, Q4 2022-Q1 2023.

Reducing Readmissions for Ventilator Patients Through Early Transfer to Kindred Hospitals

If priced between $143,900 and $273,700 per treatment cycle, tabelecleucel (tab-cel) would be a cost-effective cell therapy to treat a post-transplant complication, according to an 

 from The Institute for Clinical and Economic Review. Tab-cel is currently being reviewed by U.S. regulators to treat adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD).

Lymphoproliferative disease is a rare but life-threatening 

 after organ or stem-cell transplants. Many cases are associated with Epstein-Barr virus, accounting for more than 75% of post-transplant lymphoproliferative disease, according to one estimate. Most people have been exposed to Epstein-Barr, but the immune suppression drugs used after a transplant can reactivate the virus.

There is no treatment available specifically for Epstein-Barr virus related post-transplant lymphoproliferative disease, but Rituxan (rituximab) and chemotherapy are often used to treat the disease.

“EBV+ PTLD is a rare and often fatal cancer associated with solid organ transplant and stem cell transplant,” ICER’s vice president of research Foluso Agboola, MBBS, MPH, said in a 

. “Unfortunately, approximately half of EBV+ PTLD cases are refractory to initial treatment or relapse, resulting in a poor prognosis. The limited evidence on tabelecleucel suggests that it provides important clinical benefits in patients with relapsed refractory EBV+ PTLD, extending survival for patients who otherwise do not usually survive beyond a few weeks to months, with few harms.”

Tab-cel is an allogeneic, T-cell immunotherapy that, if approved, would administered intravenously for three doses per cycle for a minimum of two cycles. It would be available as an off-the- shelf, T-cell therapy.

The biologics license application for tab-cel is being review by U.S. regulators, and an FDA decision is expected by Jan. 15, 2025. Developed by Atara Biotherapeutics, tab-cel will be marketed in the United States by Pierre Fabre Laboratories, according an 

 between the companies in December 2023. Based on this agreement, all tab-cel manufacturing, clinical, and regulatory activities have been transitioned from Atara to Pierre Fabre Laboratories.

Tab-cel is available in Europe with the brand name Ebvallo and marketed by Pierre Fabre.

ICER has given tabelecleucel a rating of A, indicating the T cell therapy has a high certainty of substantial net health benefit. The analysis indicates that treatment with tab-cel extends survival for patients who otherwise do not usually survive beyond a few weeks to months. The data indicate that the therapy appears to induce partial or complete remission in a substantial proportion of patients with relapsed/refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease.

ICER’s health-benefit price benchmark is a price range suggesting the highest U.S. price, net of rebates and discounts, a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients receive from that treatment.

The BLA is supported by data from more than 43 patients treated with tab-cel across multiple life-threatening diseases, including the single-arm ALLELE study that met its primary endpoint. Interim data from this study found that 22 of 43 (51.2%) Epstein-Barr virus related post-transplant lymphoproliferative disease patients achieved an objective response rate. The median duration of response was 23 months, and the median overall survival was 18.4 months.

In ICER’s analysis, tab-cel resulted in high quality of life years (QALY), equal value of life years (evLY) gained, and life years gained over a lifetime horizon. Based on a placeholder price of $287,500 per 35-day treatment cycle, the incremental cost-effectiveness of tabelecleucel was approximately $183,449 per QALY gained and $156,668 per evLY gained.

Because tab-cel is an off-the-shelf product, the ability to treat patients early would be an advantage, ICER notes. Currently available T cell therapies have lengthy manufacturing times, which can delay treatment.

ICER officials, however, cautioned that the cost-effectiveness findings could be an optimistic estimate given the limited clinical evidence available. The actual cost-effectiveness will be dependent on its actual price and the survival benefit of treatment. The analysis notes that few patients have been treated have been followed for five years.

“Additionally, it is not clear how generalizable the data from the ALLELE study are, since the small sample size and short duration of the trial may obscure differences in treatment effect by transplant type,” the reported noted.

Long-term safety data are also needed to assess potential harms. ICER officials noted there were four events of acute graft versus host disease (GvHD) in three patients reported in the U.S. expanded access program that were considered by investigators to be possibly related to tabelecleucel.

ICER has given tabelecleucel a rating of A, indicating the T cell therapy for Epstein-Barr virus related post-transplant lymphoproliferative disease has a high certainty of substantial net health benefit and would be cost-effective if priced between $143,900 and $273,700.


Formulary Watch

Clinical

ICER Gives Cell Therapy for Post-Transplant Complications High Rating

In a session that discussed the pipeline and a new FDA-approved treatment option for patients with nonalcoholic steatohepatitis (NASH), speakers at the annual meeting of the Academy of Managed Care Pharmacy in New Orleans debated how managed care organizations and health plans can think about coverage for these newer therapies.

Both Donna Cryer, J.D., and Stephanie Spence, Pharm.D., CSP, agreed that there is unmet medical need for patients given the progressive nature of NASH, a severe form of fatty liver disease. NASH is a leading cause of liver-related mortality, especially for those with hypertension and type 2 diabetes. NASH affects between 1.5% and 6.5% of U.S. adults.

Some experts are now calling the disease metabolic dysfunction-associated steatohepatitis, or MASH. But as the speakers pointed out, an ICD 10 code has not yet been established for MASH. The transition to a new code could take some time.

Last month, the FDA granted accelerated approval for Rezdiffra (resmetirom), the first drug approved specifically for NASH. It was approved based on a surrogate endpoint. Treatments that have been used for NASH have included lifestyle modification and off-label use of drugs therapies such as semaglutide or tirzepatide, drugs that are approved to treat diabetes and weight loss.

Rezdiffra is an oral therapy approved based on data from the ongoing phase 3 MAESTRO-NASH trial. At 52 weeks, Rezdiffra achieved liver histological improvement endpoints: resolution of NASH and reduction of liver fibrosis, two surrogate endpoints. It has shown promise in improving liver scarring and even resolving NASH in some patients after one year. 

Spence, who is director of specialty pharmacy at CareFirst BlueCross BlueShield, which serves 3.5 million people in Maryland, the District of Columbia and Northern Virginia, said that payers’ prior authorization for Rezdiffra could consider either noninvasive methods or biopsy as way of  diagnosing NASH, as well as requiring treatment for comorbid conditions, lifestyle modifications and abstinence from alcohol.

Additionally, Spence mentioned that prescribing would be appropriate through specialty providers. “Primary care doctors are overwhelmed by the prospect of managing such a large population with this condition.”

She also suggested that because of the cost of Rezdiffra, payers should consider value-based contracts with manufacturers. The drug's maker, Madrigal Pharmaceuticals, announced in March after the FDA approved Rezdiffra that it would price drug to cost $47,400 per year. 

“There are opportunities, especially as we look at the NASH treatment pipeline, to bring some innovation to the contracts that we have with manufacturers, specifically around outcomes,” said Spence, who suggested that there are still questions about where Rezdiffra fits into NASH treatment. “Are we actually preventing the progression of NASH with these therapies? Or are we just tacking on another therapy?" she asked.

During the question and answer part of the session, Donna Cryer, founder and CEO of the patient organization Global Liver Institute, addressed the importance of payers following therapies’ approved labels. For example, she pointed out that Rezdiffra’s label does not require biopsies; instead, patients can be diagnosed with ultrasound imaging or blood tests for biomarkers of liver fibrosis. “I would not want to see a prior authorization require a biopsy since that is not on the label,” Cryer said.

Additionally, Cryer said that the requirement to abstain from alcohol also is not on the label. “The nomenclature of this disease was to create a category separate from alcohol-related liver disease. Certainly, patients should be counseled about the impact of alcohol on the liver. But it’s important to patients to explain to families that this is not about alcohol." 

Spence said it is common to require abstention from alcohol for liver diseases because of its impact on the liver. “It’s also in the study design [of Rezdiffra]. There is room for discussion but it is a standard criteria for drugs that are modifying the liver.”

She also said payers should review requiring patients to step through current treatment options, especially with the GLP-1, before being prescribed Rezdiffra

Cryer, however, suggested that requiring steps could make it harder for patients to access needed therapy. “I see this as an opportunity rather than something required. I think this is an opportunity to optimize people’s metabolic profile."

Even with the approval of Rezdiffra, Cryer and Spence agree that a huge need for additional treatments and equitable access remains. Therapies in development are targeting various aspects of the disease, including inflammation, fibrosis and bile acid regulation. They offer the potential to slow or reverse disease progress.

Semaglutide, currently approved as Ozempic to treat diabetes and as Wegovy for weight loss, is in a phase 3 trial for a NASH indication. It is a GLP-1 agonist, which have been used off label for NASH. Other drugs in phase 3 trials include:

Azemiglitizone, an oral thiazolidinedione

Belapectin, an intraveneous galectin inhibitor

Efruxifermin, a subcutaneous fibroblast growth factor mimetic

The pipeline for NASH treatments is robust, with six therapies in phase 3 development. Speakers at the annual AMCP meeting discuss Rezdiffra, the first drug approved specifically for NASH, and how to think about coverage issues.

Providing Access to Rezdiffra for NASH and the Therapies to Come | AMCP 2024

The best strategy for treatting optics neuritis, the importance of complete elimination of subretinal fluid and many other long-held beliefs in ophthalmology were challenged during a session Saturday focused on breaking down myths at the annual meeting of the American Academy of Ophthalmology (AAO) in Chicago.

The moderator of the session, Sruthi Arepalli, M.D., noted that new research findings can chip away at cherished notions about diagnosis and treatment that were once held firmly as true.

“Things change with time as more data comes,” said Arepalli, an assistant professor in the Department of Ophthalmology at the Emory School of Medicine in Atlanta.

Optic neuritis: To treat (and test) or not to treat (and not to test)

John J. Chen, M.D., Ph.D., a neuro-ophthalmologist at the Mayo Clinic, said the belief that optic neuritis tends to get better on its own stemmed from a landmark study published in the 

Presenters at a myth breakers session at the American Academy of Ophthalmology took aim at some prevailing notions.

Maureen Hennessey, PhD, CPCC, CPHQ

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