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Time to Talk Cell Based Therapy Regulation

How well do you understand cell based therapy?

It’s complicated!

Given the pace of research and the number of already approved cell-based products it is now high on the list of essential knowledge for leaders in the pharma industry and for specialty pharmacies that want to play in that sandbox.

MSC-based cell therapy involves the administration of MSCs as a medicinal product. These cells are characterized by ways in which they can be used as a therapeutic agent in human treatments.

But, the mechanisms of MSC therapy are poorly understood. According to the NIH, MSC have a short survival time after infusion, it is believed that MSC pass on their effects to other cell types. The scientific stuff now goes on in language that is likely foreign for those without a doctorate degree. SO, this Report will increase your knowledge level, but only enough to make you dangerous.  

Here are some bullet points that you may want to consider —–

The Importance of Regulation in Ensuring Safety and Impact on Treatment Cost for Mesenchymal Stem Cell Therapies

CLICK here to access the full article

Regulation is essential to ensure the safety and efficacy of mesenchymal stem cell (MSC) treatments. It upholds high standards in production, application, and monitoring of these therapies. Without it, the risk of suboptimal quality and potential serious adverse effects increases.

Key Aspects of MSC Regulation:

Establishing guidelines for MSC isolation, expansion, and characterization.

Ensuring consistency, viability, and contamination-free cells in therapies.

Developing clinical trial protocols to assess safety and effectiveness in various medical conditions.

Role of Regulatory Agencies:

Agencies like the FDA mandate rigorous testing for new treatments.

Approval is required before widespread use of MSC-based therapies.

Regulatory Challenges and Impact on Treatment Costs:

While regulation is crucial for MSC treatment safety, it also contributes to increased costs. The rigorous testing and validation, adherence to Good Manufacturing Practices (GMP), and the need for sterile, high-quality production facilities add significant expenses.

Factors Contributing to Increased Costs:

Time-consuming and expensive clinical trials with extensive participant involvement.

Maintenance of GMP compliant facilities for MSC production.

Impact on Treatment Availability:

Lengthy regulatory approval processes can delay treatment availability to patients.

Balancing Safety and Cost in MSC Therapies:

Despite the challenges, the regulatory framework is essential for maintaining the integrity of MSC therapies. The increased costs are an investment in the treatment’s long-term success, building trust among patients, healthcare providers, and stakeholders in regenerative medicine.

Benefits of a Strong Regulatory Framework:

Ensures patient safety and high-quality care.

Contributes to the long-term success and trustworthiness of MSC treatments.

Our next Report will be on yet another Cell Based therapy!

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Time to Plan for the Cell & Gene Tsunami?

Quiz me this…..!

What new technology emerged in the 1970s that truly changed the world?…. Certainly, you immediately thought of ‘the computer’ in your top 5 answers.

So, what new technology is now emerging in the 2020s that will truly change the world of specialty pharmacy?….. tick… tick… tick… time’s up! The answer that many of you may have thought of is…. Cell and Gene Therapy.

Yep, C&G is gonna’ have a huge impact on pharmacy across the board. If you play it out to the extreme, there will be, one day, a cell or gene therapy (or some other magical potion or gizmo) that will cure every ache and ill out there. Bye bye pharmacy!

OK, that may be a pipe dream… but it might be best to start planning for the tidal wave as opposed to a mere changing tide.

The article below is on point with the changing tide in pharmacy. It is worth a read… both intellectually and practically from a business perspective.

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8 Trends to Watch in the Cell and Gene Therapy Market

Click Here to access the article

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FDA Approves Oral Tx for Rare CAH – Crenessity

…………. catching up on recent FDA approvals

The FDA recently approved a new therapy, Crenessity (crinecerfont) from Neurocrine Biosciences, Inc., to be used together with glucocorticoids (steroids) to control androgen levels in adults and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH).

Classic congenital adrenal hyperplasia is a rare genetic condition affecting the adrenal glands, which produce hormones such as cortisol and androgens. Patients with classic CAH do not produce enough cortisol and produce too many androgens. These patients require high doses of glucocorticoids because the glucocorticoids also help to reduce the excess levels of androgens. Crenessity works by reducing excessive adrenal androgen production, which helps reduce the amount of glucocorticoid treatment needed.

Crenessity was granted an Orphan Drug designation. The estimated prevalence of CAH is 1/10,000. Annual incidence ranges from 1/5,000 to 1/15,000.

Dosing for adults on therapy are 2 capsules daily. Crenessity is also available as an oral solution for children who are dosed based on weight.

Neurocrine has set the US list price for Crenessity at approximately $38,333 for a 30-day supply for adults ($460,000 annually). For pediatric patients weighing less than 20 kilograms, the 30-day supply is priced at about $19,167 ($230,000annually).

Crenessity is available through limited distribution. PantherRx Rare Specialty Pharmacy has confirmed that they were selected as a limited distribution partner.

CLICK HERE to access prescribing information

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FDA Approves New Treatment for Congenital Adrenal Hyperplasia  

CLICK HERE to read the FDA press release

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