Michael Storey

@marcellstrbich @realDonaldTrump @RobertKennedyJr @matthuffman1 @GovMikeDeWine Because HB 73 has nothing to do with PBMs.

If ⁦@realDonaldTrump⁩ & ⁦@RobertKennedyJr⁩ are working to reign in primary benefit managers (PBMs) than why is OH Senate President ⁦@matthuffman1⁩ and ⁦@GovMikeDeWine⁩ working to oppose HB 73 a bill to reign in PBMs? breitbart.com/politics/2024/…

@ASHP_EmergSci Blake and I will be presenting at ASHP Midyear 2024 on this topic - we welcome all to attend Tuesday at 4-5pm. I will also have a separate presentation Monday from 4:30-5:15pm on Gene Therapy Adverse Effect Management. Both of these presentations will provide more info!

I’d like to extend my thanks to Drs Shay and Storey for providing top level tips for engaging in the operational aspects of gene therapy which will be a certain area of exponential growth! Thanks to all who tuned in!

@ASHP_EmergSci @nephpharm Blake and I will be presenting at ASHP Midyear on this topic - we welcome all to attend Tuesday at 4-5pm in NOLA! I will also have a separate presentation Monday from 4:30-5:15pm on Gene Therapy Adverse Effect Management. Both of these presentations will provide more info!

Welcome to tonight’s @ASHP_EmergSci Journal Club with Drs Blake Shay and Michael Storey @storeyrx I’m @nephpharm and I will be your host. I work on the nanoformulation side- so I’m excited to understand the pragmatic challenges/opportunities at the operational level!🧬🧑‍💼💉🧪

@ASHP_EmergSci Developing USP 800 policies and procedures for compounding will be a key to success. Gene and cell therapies do not fit easily into current standards and guidelines for pharmacy. It is imperative to stay current on the latest news and publications on handling of G&C therapies.

Given the rapid growth associated with gene therapy, how can health-systems practice safe handling when current guidelines and standards are outdated?

@ASHP_EmergSci While most will not qualify as a hazardous drug per NIOSH, the toxicity could be considered “serotoxicity” or “immunotoxicity.” Health-system workers can mitigate this risk by wearing appropriate personal protective equipment when handling the therapy and patient waste.

@ASHP_EmergSci It is also important to discuss the numerous vials that will be drawn - often without closed system transfer devices.

How should health-system pharmacy leaders train their staff to handle gene therapy given the risk of occupational exposure?

@ASHP_EmergSci Clinically, centers must have plans for confirming eligibility, providing the infusion, managing adverse effects, and long-term follow-up.

What are some of the barriers experienced with gene therapy management from both an operational and clinical perspective?

@ASHP_EmergSci Operationally, centers must ensure appropriate cold chain storage, trained personnel, and SOPs are in place.  Financially, centers need to be engaged with confirming benefits, prior authorization, and payor contracting. These all need confirmation prior to ordering gene therapy.

@ASHP_EmergSci It is also important to familiarize yourself with single case agreements and your state Medicaid policies as this is nearly a requirement for each gene or cell therapy to ensure payment.

@ASHP_EmergSci Most importantly, I would include conversations about the cost and benefits to your community for offering these new therapies. While costly, this can be mitigated by advance discussion with senior leaders and finance prior to the therapy approval. Early communication is key!

What advice do you have for health-system pharmacy leaders that are trying to on-board offering gene therapy at their site? How can they approach discussions with institutional leaders on financial planning?

@ASHP_EmergSci asgct.org/publications/l…

@ASHP_EmergSci ASGCT provides a landscape report quarterly to keep up with new publications and FDA approvals. There are many updates coming forth about the handling and safety of viral vector based gene therapies. ASHP continues to provide programming on gene therapy in various formats.

What recommendations do you have for health-system pharmacy leaders to keep up to date with new gene therapy approvals?

@ASHP_EmergSci Ultimately, I firmly believe that the only viable way for health-systems to expect to be able to sustainably provide these products to patients is to receive revenue for providing them. A specialty pharmacy will not supply the infrastructure or personnel to provide this care.

@ASHP_EmergSci Buy and bill creates revenue to provide resources to manage these therapies - clinically and operationally. White bagging may appeal to an organization that does not have the financial support to offer the therapies but can manage the patients clinically and operationally.

What are the pros and cons of the buy and bill vs. white bagging purchasing strategies for gene therapy?

@ASHP_EmergSci They can cost between $500K to $5M dollars depending on many factors. They are available for 340B drug pricing, dependent on site eligibility and the site of care for the therapy. Some cellular gene therapies must be done inpatient, so 340b is not applicable.

Now for the $ questions... Approximately how much does gene therapy cost? Is gene therapy eligible for 340B pricing?

@ASHP_EmergSci Today, gene therapies are approved for a variety of diseases - spinal muscular atrophy has now had an approved gene therapy for 5 years this month, and more recent approvals for Duchenne muscular dystrophy and genetic hematologic diseases are now starting to be used.

What disease states are gene therapies most commonly used in?

@ASHP_EmergSci While the the first gene therapies were approved in Europe years earlier (with limited uptake), the first approvals in the United States were in 2017, for tisagenlecleucel for leukemia and lymphoma and voretigene neparvovec for a rare from of retinal dystrophy.

Let’s start with the basics. When was the first gene therapy FDA-approved? How many gene therapies are currently approved?

@sanofi Yes. All infants are at risk of RSV. Please make enough nirsevimab for all of them.

👶 Are all infants at risk from Respiratory Syncytial Virus? #RSV

@sanofi How do you win when you don’t even make enough of your own drugs? RSV season has not started yet. Meanwhile, you tell us you will not take any more orders for nirsevimab 100mg for the remainder of the season. Angry for my patients.

Today we share our Q3 2023 Results and details on the next chapter of our Play to Win strategy: spkl.io/60164oeJr