Making a Commercially Successful Gene Therapy

April 4, 2019

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Developing a commercially successful gene therapy is challenging, and requires balancing several different considerations. Having a clinical effective therapy is necessary, but not sufficient to ensure product success. In addition to this, reimbursement, quality and regulatory considerations, and manufacturing also must be considered.





With the development and approval of a several therapies in the last few years, reimbursement is now the primary bottleneck in the successful commercialization of gene therapies. Gene therapy is a one-time, potentially curative treatment which generally comes at a very high cost. This contrasts with most current therapies which require ongoing treatment.


This change in treatment regime can prove taxing to payers (such as insurance companies and national health care providers) and is an area where considerable innovation is needed as payment models diverge from value based pricing towards more outcome based models.


For example, in the US, the list price for Yescartaand Kymriah is $373,000 and $475,000 per patient respectively1,2. The costs for Luxturna is $425,000 per eye, making them among the most expensive drugs available on the market today3. Novartis has suggested a risk-sharing approach where they will absorb the cost of Kymriah if it does not work on the patient2.



Regulatory Agency and Approval Process


In recent years, regulatory agencies have been particularly active in enabling gene therapies to navigate the regulatory pathway with the development of the advanced therapy medicinal product (ATMP) and regenerative medicine advanced therapy (RMAT) designations in Europe and the US respectively. These designations along with both Breakthrough Therapy and Orphan Drug designation, which are also frequently applied to cell and gene therapies, allow for an expedited regulatory pathway for drugs that demonstrate the ability of cure or modify serious, life threatening diseases that are currently without existing effective treatments. 


These designations can lead to a shorter time to approval and it is estimated that breakthrough therapy products are approved within an average 5.2 years from IND application, compared to 7.4 years for a non-breakthrough therapy. RMAT designations have so far been granted to >30 drugs indicating the popularity of using this route4.



Implications for Manufacturing


Manufacturing sufficient product with the necessary quality is an area that requires significant effort and knowledge. The challenges around reimbursement and the potential for shorter approval times, both put pressure on the development of an optimal manufacturing processes where the cost of manufacturing needs to support any proposed selling price; and process development time is short.


There are several examples of where the balance has between these various factors has not been struck successfully. Glybera was approved by the European Medicines Agency in October 2012 making it the first virus based gene therapy on the market. Unfortunately, despite a price of €1 million, the cost of manufacturing was too high to make a profit and, with a very low patient demand the product was withdrawn from the market in 2017 after only 1 patient was treated5



The Future


While there are many challenges to overcome to successful commercialize a gene therapy drug, the results across different gene therapies are phenomenal, revolutionary, and lifechanging. The widely documented benefits and patient success stories continue to grow. 


Ashanthi DeSilva, was the first patient of gene therapy in the world. She participated in the first clinical trials, experiencing first-hand the success of trials to the development to commercialization6. Many more gene therapy success stories are documented. 


An advocate for gene therapy, Ashanthi shares her powerful story. Listen here:


Learn more in the next gene therapy blog: How Does Genetic Material Get Delivered to The Patient?





1, The FDA Approves a Second CAR-T Therapy, Cheaper than Novartis’, 19 October 2017

2 Fast Company, This Pioneering $475,000 Cancer Drug Comes With A Money-Back Guarantee, 31 August 2017 

3 CNN, Gene therapy for rare retinal disorder to cost $425,000 per eye, 3 January 2018   

4 Key Considerations for Gene Therapy Commercialization, Cell Culture Dish, 12 September 2018

5 Pharmaphorum, Glybera, the most expensive drug in the world, to be withdrawn after commercial flop, 20 April 2017 

6 Biotechnology Innovation Organization, Ashanthi DeSilva—Very First Gene Therapy Patient, 7 June 2018


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Clive Glover – Director, Cell & Gene Therapy

Dr Clive Glover is the Director of Cell & Gene Therapy at Pall. Clive holds a PhD in Genetics and has over 15 years of experience in cell and gene therapy. His work has been published in numerous scientific journals and he has presented at many conferences.
Dr Clive Glover is the Director of Cell & Gene Therapy at Pall. Clive holds a PhD in Genetics and has over 15 years of experience in cell and gene therapy. His work has been published in numerous scientific journals and he has presented at many conferences.
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