Editors' (re)View: Advancing mental health treatment; CMS proposes financial incentives for gene therapy access

April 12, 2024
Pharma Manufacturing editors Karen Langhauser and Andrea Corona comment on the notable happenings in the pharma industry from the week of April 8

Advancing mental health treatment

This week, Bristol Myers Squibb shared positive news from a phase 3 trial of its schizophrenia drug, KarXT, picked up from Karuna Therapeutics last year.

KarXT — which has a September PDUFA date — is a potentially revolutionary schizophrenia treatment, marking the first major pharmacological innovation in the field in decades.

While recorded descriptions of illnesses that sounded like schizophrenia go as far back as ancient Egypt, the disease was first identified as a mental illness by German psychiatrist Emil Kraepelin in 1887, who referred to it as 'dementia praecox.' Swiss psychiatrist, Eugen Bleuler, coined the actual term ‘schizophrenia’ in 1910.

Anyone who has dug into the past of mental illness treatment knows it’s quite grim. Throughout history, people who likely suffered from diseases such as schizophrenia were burned as witches, locked in asylums, and had ice picks driven through their eye sockets as a form of ‘treatment.’

Modern-day treatments, while far more humane, still are not perfect. The meds — which are necessary to control the disease — come with side effects, including drowsiness, sedation, uncontrolled muscle movements and weight gain, that often cause people to stop taking the drugs.

But KarXT — which is an oral combo of xanomeline and trospium chloride taken twice daily — differs from existing drugs in terms of its mechanism of action. While traditional antipsychotics target dopamine receptors in the brain, KarXT targets the muscarinic neurotransmitter system (M1 and M4 neuroreceptors), which is involved in cognition. Because it does not directly block dopamine receptors, many of the negative side effects of traditional treatments are eliminated.

While KarXT is by no means a cure, the outcomes from the trial analysis, where more than 75% of participants achieved >30% improvement in symptoms, indicate a much-needed step forward in mental health treatment and may also lead to breakthroughs in other areas of psychosis. —Karen Langhauser

CMS proposes 2025 financial incentives for sickle cell disease gene therapy access

This week, the Centers for Medicare & Medicaid Services (CMS) outlined an updated 2025 proposal for the inpatient prospective payment system (IPPS), aimed at expanding patient access to emerging medical technologies, including gene therapy for sickle cell disease (SCD).

Under the new proposal, CMS plans to implement new technology add-on payments (NTAP)  specifically designed to support the adoption of gene therapies. These payments are crucial for hospitals, especially when introducing expensive but potentially life-changing treatments like gene therapy for sickle cell disease.

The CMS is proposing an increase in the NTAP percentage from 65% to 75%. This adjustment would apply specifically to gene therapies used for SCD treatment, pending approval. The proposed increase would start next year and last through the 2 to 3 year newness period of the therapy.

Earlier this year, the Biden administration announced that SCD had been selected as the initial focus of the Cell and Gene Therapy Access Model, also set to launch in 2025. This model aims to expand access, and reduce costs for vulnerable populations, coinciding with broader efforts to cut prescription drug costs. 

Medicaid, which covers 50-60% of SCD patients, faces nearly $3 billion in annual health system costs due to SCD episodes. The Cell and Gene Therapy Access Model will involve CMS in negotiating outcomes-based agreements with manufacturers to link pricing with improved health outcomes for Medicaid recipients.

The financial support for gene therapy highlights a shift towards embracing personalized medicine. By financially incentivizing hospitals to incorporate such advanced therapies, CMS aims to make these treatments more accessible to the patients who need them most. — Andrea Corona 

About the Author

Karen P. Langhauser | Chief Content Director, Pharma Manufacturing

Karen currently serves as Pharma Manufacturing's chief content director.

Now having dedicated her entire career to b2b journalism, Karen got her start writing for Food Manufacturing magazine. She made the decision to trade food for drugs in 2013, when she joined Putman Media as the digital content manager for Pharma Manufacturing, later taking the helm on the brand in 2016.

As an award-winning journalist with 20+ years experience writing in the manufacturing space, Karen passionately believes that b2b content does not have to suck. As the content director, her ongoing mission has been to keep Pharma Manufacturing's editorial look, tone and content fresh and accessible.

Karen graduated with honors from Bucknell University, where she majored in English and played Division 1 softball for the Bison. Happily living in NJ's famed Asbury Park, Karen is a retired Garden State Rollergirl, known to the roller derby community as the 'Predator-in-Chief.' 

About the Author

Andrea Corona | Senior Editor