THE MARKET FOR
CELL THERAPY
MANUFACTURING
Strategies for Cell Therapy Product Pricing, Cost
Control, Reimbursement, Distribution, & More
BioInformant
Worldwide, LLC
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TABLE OF CONTENTS
1. Introduction to Cell Therapy Manufacturing……………………………………….p.4
2. Leading Cell Therapy Companies……………………………………………………p.6
2.1. List of Cell Therapy Companies Worldwide
[Table] List of Cell Therapy Companies Worldwide by Location and Type
2.2. Leading Cell Therapy Companies
2.1.1. ReNeuron
2.1.2. Mesoblast Ltd.
2.1.3. Asterias Biotherapeutics
2.1.4. TiGenix NV
2.1.5. Cellectis
2.1.6. Cynata Therapeutics
2.1.7. Cytori Therapeutics
2.1.8. Astellas Pharma, and Subsidiary Ocata Therapeutics
2.1.9. Gamida Cell Ltd.
2.1.10. Pluristem Therapeutics
3. Approved Cell Therapy Products……………………………………………………p.41
3.1. Apligraf (Organogenesis, Inc. & Novartis AG)
3.2. Carticel (Genzyme)
3.3. Cartistem (MEDIPOST)
3.4. ChrondoCelect (TiGenix NV)
3.5. Cupistem (Anterogen)
3.6. Dermagraft (Advanced Tissue Sciences)
3.7. Epicel (Vericel)
3.8. Hearticellgram-AMI (FCB Pharmicell)
3.9. Holoclar (Chiesi Farmaceutici)
3.10. Osteocel (NuVasive)
3.11. Prochymal (Mesoblast)
3.12. Provenge
3.13. Strimvelis (GSK)
3.14. TEMCELL (JCR Pharmaceuticals Co. Ltd., Licensee of Mesoblast Ltd.)
4. Pricing Analysis for Cell Therapy Products…………………………………………p.60
4.1. Pricing of Approved Cell Therapy Products
[Table] Pricing of Approved Cell Therapies
[Table] Pricing Scale for Approved Cell Therapies, by Type
4.2. Reasons for High Cell Therapy Product Costs
4.2.1. High Cost of Manufacturing
4.2.2. Need to Recoup Developmental Costs
4.2.3. Cash Flow and Quantity of Cash Reserves
4.2.4. Need for Return on Investment (ROI)
4.2.5. High Cost of Delivery
4.2.6. Lack of Comparative Studies as Evidence for Reimbursement Scheme
4.2.7. Lack of Competition
4.2.8. Potential for High Utility Patient Outcomes (Cures)
4.2.9. Small Market Size
5. Cost-Control for Cell Therapy Products...…………………………………………..p.69
5.1. Cost of Goods (COGs) Components
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5.2. Automation as a Cost Control Measure
[Table] Cost Per Million of Clinical-Grade MSCs Expanded by Different Methods
5.3. Effect of Process Development and Process Change on COGs
5.4. Managing Cash Flow
5.5. Role of Cell Therapy CDMOs
6. Time Frames for Cell Therapy Product Development…….………………………..p.80
6.1. Pre-Market Challenges
6.2. Persevering Through Lengthy Developmental Timelines
6.3. Navigating the Regulatory Environment
6.4. Timeline from Phase I to Commercialization
7. Reimbursement of Cell Therapy Products…………………………………..………p.85
7.1. Securing Reasonable Reimbursement
7.2. Encouraging Adoption
8. Distribution Channels for Cell Therapy Products…..……………………………...p.90
8.1. Addressing Distribution Logistics
8.2. Allogeneic vs. Autologous Therapies
9. Market Trend Analysis - Key Trends Impacting the Marketplace……………..…p.93
9.1. Regulatory Issues
9.1.1. FDA Guidelines for HCT/Ps
9.1.2. 21st Century Cures Act)
9.2. Global Trends
9.2.1. Accelerated Approval Pathway in Japan
9.2.2. Prolific Partnering Between Cell Therapy Companies and Japanese
Pharmaceutical Companies
9.2.3. Cell Therapy Products (and Pricing) Resulting from the Japanese Regulatory
Framework
10. Technologies Impacting the Cell Therapy Manufacturing Market…..…………p.109
10.1. Closed-System Manufacturing
10.2. Automation of Cell Therapy Manufacturing Processes
10.3. Automation of Data Management
10.4. Bioreactor Technologies
11. Market Potential for Autologous vs. Allogeneic Manufacturing………………….p.113
[Table] Allogeneic vs. Autologous Product Development Among Leading Cell Therapy
12. Cell Therapy Manufacturing Challenges and Considerations…………………….p.117
13. Conclusions…………………………………………….………………………………p.120
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1. Introduction to Cell Therapy Manufacturing
Cell therapy is the use of cells and tissues to regenerate, repair, and enhance human health.
Traditionally, the modern medical system has relied on a pharmaceutical based approach. This
lead to the rise of the U.S. Food and Drug Administration (FDA) which was founded in 1906,
making it more than one-hundred years old. Unfortunately, our approach to human health has not
experienced a dramatic shift over the past 100+ years, and as a society, we still tend to rely on a
drug-based approach.
Although variations exist, drugs generally work by binding to receptors on the cell surface or by
exerting an enzymatic effect to regulate the rate of an internal chemical reaction.
Pharmaceuticals can frequently modulate the human system with great effect, but rarely do they
have the capacity to regenerate or restore entire tissues or systems within the human body.
In contrast, cell therapy works
because cells are powerful factories
that can exert therapeutic effects
through a wide range of strategies,
including honing to sites of injury,
exerting paracrine effects, and in
some cases, differentiating into new
types of cells and tissues. This
versatility makes cell therapy
extremely powerful and gives it the potential to reverse previously untreatable diseases. Needless
to say, it is an exciting time in history.
For this reason, cell therapy – or more likely, cell therapy in combination with pharmaceuticals –
will be the future of human health.
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One of the major issues with moving cell therapy
products from “bench to bedside” has been
manufacturing bottlenecks. The heterogeneous nature
of cell therapy products has introduced manufacturing
complexity and regulatory concerns, as well as scale-
up complexities that are not present within traditional
pharmaceutical manufacturing.
Furthermore, much of cell therapy manufacturing now involves patient-specific cell therapies,
where cells for an individual patient are processed one batch at a time. Ten years ago, it was a
common belief that making individual batches of a therapy for each patient would never provide
an economical business model. Rather, allogeneic therapies would better align with the
pharmaceutical model, because universal cell populations could be prepared in advance to later
be prescribed to patients in need, much like drug compounds have been administered for
decades.
Nonetheless, clinical data in support of patient-specific therapy has been compelling, so the
pressure is on cell therapy industry to manufacture these therapies on a larger scale. It is now
understood that both autologous and allogeneic cell therapies will contribute important
therapeutic solutions. There are opp
