Swiss pharmaceutical giant Novartis made history as the first company to win FDA approval for a CAR-T therapy in the United States. Novartis announced that its genetically modified autologous (“self-derived”) immunocellular therapy, Kymriah, will cost $475,000 per treatment course. Shortly thereafter, Kite Pharma announced the approval of its CAR-T therapy, Yescarta, in the U.S. with a list price of $373,000. While these prices are expensive, they are far from trendsetting.
Cell Therapy Pricing and Products
In this article:
- Cell Therapy Cost
- Pricing of Approved Cell Therapy Products
- Wound Care Therapy
- Cartilage Based Cell Therapy
- Intravenous Cell Therapy
- Cell-Based Gene Therapy
- Cost of Stem Cell Therapy
- Autologous vs. Allogeneic Stem Cell Transplants
- Cell Therapy Pricing
Cell Therapy Cost
Pricing of cell therapies is controversial because most cell therapy products are priced exponentially higher than traditional drugs. Unfortunately, most drugs can be manufactured and stockpiled in large quantities for off-the-shelf use, while cell therapies involve living cells that require a different approach to commercial-scale manufacturing, transit, stockpiling, and patient use.
To date, the highest priced treatment has not been a cell therapy, but a gene therapy (Glybera). At the time of its launch, Glybera was the first gene therapy approved in the Western world, launching for sale in Germany at a cost close to $1 million per treatment.[1] The record-breaking price tag got revealed in November 2014, when Uniqure and its marketing partner Chiesi, filed a pricing dossier with German authorities to launch Glybera. Unfortunately, Glybera was later withdrawn from the European market due to lack of sales.
Following the approval of Glybera, Kymriah, Yescarta, and more than a dozen other cell therapies, conversations surrounding pricing and reimbursement have become a focal point within the cell therapy industry.
Pricing of Approved Cell Therapy Products
In contrast to pharmaceutical drugs, cell therapies require a different pricing analysis. Below, price tags are shown for approved cell therapy products that have reached the market (prices in US$) and for which there is standardized market pricing.
Pricing of Approved Cell Therapy Products:
Apligraf by Organogenesis & Novartis AG in USA = $1,500-2,500 per use [2]
Carticel by Genzyme in USA = $15,000 to $35,000 [3]
Cartistem by MEDIPOST in S. Korea = $19,000-21,000 [4],[5]
Cupistem by Anterogen in South Korea = $3,000-5,000 per treatment [6]
ChondroCelect by Tigenix in EU = ~ $24,000 (€20,000) [7]
Dermagraft by Advanced Tissue Science in USA = $1,700 per application [8],[9]
Epicel by Vericel in the United States = $6,000-10,000 per 1% of total body surface area [10]
Hearticellgram by FCB-Pharmicell in South Korea = $19,000 [11]
HeartSheet by Terumo in Japan = $56,000 (¥6,360,000) for HeartSheet A Kit; $15,000 (¥1,680,000) for HeartSheet B Kit (*Each administration uses one A Kit and 5 B Kits) [12]
Holoclar by Chiesi Framaceutici in EU = Unknown (very small patient population)
Kymriah by Novartis in USA = $425,000 per treatment[13]
Osteocel by NuVasive in USA = $600 per cc [14],[15]
Prochymal by Osiris Therapeutics and Mesoblast in Canada = ~ $200,000 [16]
Provenge by Dendreon and Valeant Pharma in USA = $93,000 [17], [18]
Spheroxby CO.DON AG in EU = $9,500 – $12,000 (€8,000 – €10,000) per treatment [19]
Strimvelis by GSK in EU = $665,000 (One of world’s most expensive therapies) [20],[21]
Temcell by JCR Pharmaceuticals Co. Ltd. in Japan = $115,000-170,000 [22]
*Pricing of TEMCELL is $7,600 (868,680 ¥ per bag), with one bag of 72m cells administered twice weekly and 2m cells/kg of body weight required per administration[23]
Yescarta by Kite Pharma in USA = $373,000[24]
Wound Care Therapy
As shown in the list above, wound care products tend to have the lowest cell therapy pricing, typically costing $1,500 to $2,500 per use. For example, Apligraf® is created from cells found in healthy human skin and is used to heal ulcers that do not heal after 3-4 weeks ($1,500-2,500 per use), and Dermagraft is a “skin substitute” that is placed on your ulcer to cover it and to help it heal ($1,700 per application).
Interestingly, Epicel is a treatment for deep dermal or full thickness burns comprising a total body surface area of greater than or equal to 30%. It has higher pricing of $6,000-10,000 per 1% of total body surface area, because it is not used to treat a single wound site, but rather used to treat a large surface area of the patient’s body.
Cartilage-Based Cell Therapy
Next, cartilage-based cell therapy products tend to have mid-range pricing of $10,000 to $35,000. For example, Carticel is a product that consists of autologous cartilage cells (pricing of $15,000 to $35,000), CARTISTEM is a regenerative treatment for knee cartilage (pricing of $19,000 to $21,000), and ChondroCelect is a suspension for implantation that contains cartilage cells (pricing of $24,000). In July 2017, the EMA in Europe also approved Spherox as a product for articular cartilage defects of the knee with a pricing of $9,500 – $12,000 (€8,000 – €10,000) per treatment.
Intravenous Cell Therapy
The next most expensive cell therapy products are the ones that are administered intravenously, which range in price from approximately $90,000 to $200,000. For example, Prochymal is an intravenously administered allogenic MSC therapy derived from the bone marrow of adult donors (pricing of $200,000), Provenge is an intravenously administered cancer immunotherapy for prostate cancer ($93,000), and Temcell is an intravenously administered autologous MSC product for the treatment of acute GVHD after an allogeneic bone marrow transplant (pricing of $115,000-170,000).
Cell-Based Gene Therapy
Finally, many of the world’s most expensive cell therapies are gene therapies, ranging in price from $500,000 to $1,000,000. For example, Kymriah is the first CAR-T cell therapy to be FDA approved in the United States (pricing of $475,00 per treatment course). Strimvelis is an ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (pricing of $665,000).
Cost of Cell Therapy
Although these generalizations do not hold true for every cell therapy product, they explain the majority of cell therapy pricing and provide a valuable model for estimating cell therapy pricing and reimbursement. This information is summarized in the following table.
TABLE. Pricing Scale for Approved Cell Therapies
Autologous vs. Allogeneic Stem Cell Transplants
Another point of reference is also valuable. The RIKEN Institute launched the world’s first clinical trial involving an iPSC-derived product when it transplanted autologous iPSC-derived RPE cells into a human patient in 2014. While the trial was later suspended due to safety concerns, it resumed in 2016, this time using an allogeneic iPSC-derived cell product.
The research team indicated that by using stockpiled iPS cells, the time needed to prepare for a graft can be reduced from 11 months to as little as one month, and the cost, currently around ¥100 million ($889,100), can be cut to one-fifth or less.[25]
Cell Therapy Pricing
While many factors contribute to cell therapy pricing, key variables that can be used to predict market pricing include:
- Whether the cell therapy involves genetic manipulation
- Type of cell therapy product
- Whether it is an autologous vs. allogeneic treatment
Another compounding factor is market size, because wound healing and cartilage replacement therapies have significant patient populations, while several of the more expensive therapies address smaller patient populations.[26]
To learn more about this rapidly expanding industry, view the “Global Regenerative Medicine Industry Database – Featuring 1,050+ Companies Worldwide.”
What variable do you think influence the cost of cell therapies? Share your thoughts in the comments below.
[1] $1-Million Price Tag For Glybera Gene Therapy: Trade Secrets”. Available at http://blogs.nature.com/tradesecrets/2015/03/03/1-million-price-tag-set-for-glybera-gene-therapy. Web. 21 Aug. 2017.
[2] 2017 Apligraf ® Medicare Product and Related Procedure Payment, Organogenesis. Available at: http://www.apligraf.com/professional/pdf/PaymentRateSheetHospitalOutpatient.pdf. Web. 3 Mar. 2017.
[3] “CARTICEL® (Autologous Chondrocyte Implantation, Or ACI)”. Available at: https://www.painscience.com/articles/cartilage-repair-with-carticel-review.php. Web. 3 Aug. 2017.
[4] Cartistem?, What. “What Is The Cost Of Cartistem?” Available at: http://www.stemcellsfreak.com/2015/01/cartistem-price.html. N.p., 2017. Web. 3 Mar. 2017.
[5] “Cartistem”. Kneeguru.co.uk. Available at: http://www.kneeguru.co.uk/KNEEtalk/index.php?topic=59438.0. Web. 3 Aug. 2017.
[6] Stem Art, Stem Cell Therapy Pricing. Available at: http://www.stem-art.com/Library/Miscellaneous/SCT%20products%20%20Sheet%201.pdf. Web. 3 Mar. 2017.
[7] Are Biosimilar Cell Therapy Products Possible? Presentation by Christopher A Bravery [PDF]. Available at: http://advbiols.com/documents/Bravery-AreBiosimilarCellTherapiesPossible.pdf. Web. 3 Aug. 2017.
[8] Artificial Skin, Presentation by Nouaying Kue (BME 281). Available at: www.ele.uri.edu/Courses/bme281/F12/NouayingK_1.ppt. Web. 3 Mar. 2017.
[9] Allenet, et al. “Cost-effectiveness modeling of Dermagraft for the treatment of diabetic foot ulcers in the french context.” Diabetic Metab. 2000 Apr;26(2):125-32.
[10] Epicel Skin Grafts, Sarah Schlatter, Biomedical Engineering, University of Rhode Island. Available at: http://www.ele.uri.edu/Courses/bme281/F08/Sarah_1.pdf. Web. 31 July. 2017.
[11] Nature. (2011). South Korea’s stem cell approval. [online] Available at: http://www.nature.com/nbt/journal/v29/n10/full/nbt1011-857b.html. Web. 3 Sept. 2017.
[12] Novick, Coline Lee. Translated version of the first two pages of Terumo’s Conditionally Approved HeartSheet NHI Reimbursement Price. [Twitter Post] Available at: goo.gl/YGCh6z. Web. 21 Sep. 2017.
[13] Fortune.com. (2017). Is $475,000 Too High a Price for Novartis’s ‘Historic’ Cancer Gene Therapy? [online] Available at: http://fortune.com/2017/08/31/novartis-kymriah-car-t-cms-price/ Web. 8 Sept. 2017.
[14] Skovrlj, Branko et al. “Cellular Bone Matrices: Viable Stem Cell-Containing Bone Graft Substitutes”. The Spine Journal 14.11 (2014): 2763-2772. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4402977/. Web. April 12, 2017.
[15] Hiltzik, Michael. “Sky-High Price Of New Stem Cell Therapies Is A Growing Concern”. Available at: http://www.latimes.com/business/hiltzik/la-fi-hiltzik-20151010-column.html. Web. 1 Sept. 2017.
[16] “Counting Coup: Is Osiris Losing Faith In Prochymal?, Busa Consulting LLC. Available at: http://busaconsultingllc.com/scsi/organelles/counting_coup_prochymal.php. Web. 3 Aug. 2017.
[17] “Dendreon Sets Provenge Price At $93,000, Says Only 2,000 People Will Get It In First Year | Xconomy”. Available at: http://www.xconomy.com/seattle/2010/04/29/dendreon-sets-provenge-price-at-93000-says-only-2000-people-will-get-it-in-first-year/. Web. 3 Mar. 2017.
[18] “Dendreon: Provenge To Cost $93K For Full Course Of Treatment | Fiercebiotech”. Available at: http://www.fiercebiotech.com/biotech/dendreon-provenge-to-cost-93k-for-full-course-of-treatment. Web. 3 Mar. 2017.
[19] Warberg Research. CO.DON (CDAX, Health Care). Available at: http://www.codon.de/fileadmin/assets/pdf/03_Investor/Research_Report/2017_07_24_CO.DON_Note_Warburg_Research_englisch.pdf. Web. 21 Sept. 2017.
[20] “GSK Inks Money-Back Guarantee On $665K Strimvelis, Blazing A Trail For Gene-Therapy Pricing | Fiercepharma”. Available at: http://www.fiercepharma.com/pharma/gsk-inks-money-back-guarantee-665k-strimvelis-blazing-a-trail-for-gene-therapy-pricing. Web. 3 Mar. 2017.
[21] Strimvelis”. Wikipedia.org. Available at: https://en.wikipedia.org/wiki/Strimvelis. Web. 13 Aug. 2017.
[22] “Mesoblast’S Japan Licensee Receives Pricing For TEMCELL® HS Inj. For Treatment Of Acute Graft Versus Host Disease”. Mesoblast Limited, GlobeNewswire News Room. Available at: https://globenewswire.com/news-release/2015/11/27/790909/0/en/Mesoblast-s-Japan-Licensee-Receives-Pricing-for-TEMCELL-HS-Inj-for-Treatment-of-Acute-Graft-Versus-Host-Disease.html. Web. 3 Mar. 2017.
[23] “TEMCELL® HS Inj. Receives NHI Reimbursement Price Listing,” JCR Pharmaceuticals Co., Ltd. News Release, November 26, 2015. Available at: http://www.jcrpharm.co.jp/wp2/wp-content/uploads/2016/01/ir_news_20151126.pdf. Web. 3 Mar. 2017.
[24] “Kite’s Yescarta™ (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy”. Business Wire. Web. 19 Oct. 2017.
[25] “Riken-Linked Team Set To Test Transplanting Eye Cells Using Ips From Donor | The Japan Times”. The Japan Times. N.p., 2017. Web. 23 July. 2017.
[26] LinkedIn Comment, by David Caron. Available at: https://www.linkedin.com/feed/update/urn:li:activity:6316277496551665664/. Web. 21 Sept. 2017.