In the past few years, there has been a spurt in clinical development of advanced therapies, including cell therapies, gene therapies and tissue engineering products. Several cell-based medicinal products have been approved in the U.S., Europe, China, Japan, South Korea, India, Canada, Australia and New Zealand. A number of start-up companies, hospitals, universities, and large biotech and pharma companies have started testing hundreds of cell-based product candidates in clinical studies.
With a visible acceleration in development, the advanced therapy field has evolved from a curiosity to a vibrant arena for competition.
The clinical data from different geographies is exciting and the manufacturing is becoming more and more cost-effective, with a plethora of companies providing new solutions. With new product approvals, the general public, patients and investors alike have embraced advanced therapies as a reality.
With competition on the rise, we know that the question is no longer, if we have products, but which will be the ones with a competitive advantage. Cell therapy products, CAR-T cell therapy products, gene therapy products and tissue-engineered products are reaching the market place almost each year.
FDA and EMA Approvals
To date, the FDA has approved 16 cell-based therapies. Among them, 11 are cell therapies; four are gene therapies and one is a tissue-engineered product. The European Medicines Agency (EMA) has thus far approved 13 cell-based therapies. Among them three are cell therapies, six are gene therapy products, and four are tissue-engineered products.
Six products have been approved by both the FDA and EMA. At the same time EMA has withdrawn approvals for four of the already approved products.
Additionally, FDA officials predict that the agency may have to approve 10 to 20 cell and gene therapy products every year.
Pricing of Gene Therapies, Cell Therapies and Tissue Engineered Products
While only a few cell-based drugs have been approved by FDA and EMA, hundreds of therapies are under preclinical and clinical development and expected to reach the market in the foreseeable future. Currently, the high cost of advanced therapies limits affordability for public and private payers and hinders patient access to treatment for many of the life-threatening diseases.
Unlike the other biologics, the price of gene therapies costs more ($357,309 to $1,206,751) than cell therapies ($110,920–$814,780) and tissue-engineered products ($18,950–$93,432).
One reason for the high cost of gene therapies therapies is that they often can be sold to only a small number of patients. For instance, ADA-SCID is a rare disease that affects between one in 200,000 and one in 1,000,000 children. For this indication, GSK’s Strimvelis is administered at a single specialist center in Italy. Strimvelis is the first ex-vivo stem cell gene therapy to treat patients with the rare disease, ADA-SCID.
While the exceedingly high prices may incentivize the development of cell-based therapies, they also limit accessibility and prevent challenges.
Global Regions for Advanced Therapy Development
The Asia-Pacific region is as active as the U.S. in developing and marketing cell-based medicinal products. The countries in this region with approved cell-based medicines include South Korea, India, Japan, China, Australia, and New Zealand.
The major healthcare drivers of this region include the aging population, transplant needs for organ donation, and the emergence of personalized medicine.
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