Overcoming the Challenges and Complexities of the Personalized Medicine Supply Chain

Mar 17, 2017

While healthcare logistics is complex for all types of medical shipments – gene, cell and immunotherapy medicines present their own special challenges.

 

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The last decade has seen an increased emphasis on research and development into personalized medicines, including cell, gene and immunotherapy treatments – a trend that is expected to continue due to the forecasted 8.74% CAGR of the personalized medicine, targeted therapeutics, and companion diagnostics market to $149 billion by 2020.

1These lofty expectations are due in large part to advancements in science and the availability of more sophisticated diagnostic tools, which are giving the medical community a better understanding of the human genome and make it easier to detect genetic mutations effecting an individual patient.

Specifically, 2016 witnessed the first ex-vivo stem cell gene therapy to be approved by the European Commission to treat patients with the rare disease ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). Martin Andrews, Head of the Rare Disease Unit at GlaxoSmithKline, calls the successful approval of Strimvelis “the start of a new chapter in the treatment of rare and genetic diseases.”2

If Strimvelis truly delivers upon its initial success, it could signal a disruptive new phase in medicine, where a one time gene fix or treatment replaces a life long dependence on taking a medicine and continuous treatment. Already, several hundred gene therapies are in development with the hope of providing a true cure for one of the 5,000 rare diseases caused by the error in a single gene3.

The high potential that personalized medicines, including gene and cell therapies, have demonstrated for curing various types of cancers has led to a significant increase in the investment into these treatments, at both the institutional and industry level. According to intelligence firm Informa, the total financing in gene and cell therapy companies in 2015 came to US$10.8B, which represented a 106% increase over 2014.4 While institutional investment into cell and gene therapies is both necessary and significant, having the knowledge and understanding of the new technologies, represented by industry and corporate investment, gives this developing technology platform validity.5

The high potential that personalized medicines, including gene and cell therapies, have demonstrated for curing various types of cancers has led to a significant increase in the investment into these treatments, at both the institutional and industry level. According to intelligence firm Informa, the total financing in gene and cell therapy companies in 2015 came to US$10.8B, which represented a 106% increase over 2014.4 While institutional investment into cell and gene therapies is both necessary and significant, having the knowledge and understanding of the new technologies, represented by industry and corporate investment, gives this developing technology platform validity.5

 

 

Therefore, the increase in corporate investment from US$58.9M in 2013 to US$2.432B in 2015,a colossal 4000+% jump over a two-year period, demonstrates a belief from large pharma that cell, gene, and immunotherapies can deliver upon their early promise6. This has recently been demonstrated in the numerous corporate partnership and acquisitions within the cell and gene therapy space.Which include Celgene’s recent US$1 billion investment in a ten-year collaboration with Juno, Amgen’s and Kite’s combined US$1.1B collaboration to advance CAR-T therapies7, and Pfizer’s desire to become the leader in gene therapy through their acquisition of Bamboo Therapeutics, Inc. for US$150M in August 2016.8

The Personalized Medicine Supply Chain: Maintaining Viability and Safety

While healthcare logistics is complex for all types of medical shipments – gene, cell and immunotherapy medicines present their own special challenges. It begins at the medical facility where a patient’s blood, plasma, white blood cells, tissue or tumor samples are harvested, during the process of apheresis, leukapheresis, or a biopsy. Samples are then transported to a Contract Development and Manufacturing Organization (CDMO) or processing facility where the personalized medicine is created for a specific patient. Once the cell, gene, or immunotherapy is prepared, it is returned to the medical facility or patient infusion site for administration. To ensure adherence to the strict regulations of the life science industry, to maintain the integrity of a therapy in transit, and the overall safety of a patient, the supply chain solution developed for both the clinical development and commercialization of a personalized medicine must take the following into consideration:

 

Time and Temperature Sensitivity

Since they can involve the transport of either cryo frozen or living cells, CAR-T and immunotherapy trial logistics are highly temperature and time sensitive. For one thing, multiple temperature profiles can exist for within one trial, with temperature requirements ranging from -196°, -80°, -20°, 2-8°, to 15-25° Celsius. That means any supply chain solution must be flexible enough to accommodate different temperature requirements to and from the site, CDMO, or lab.

Time is always an issue where clinical trials logistics are concerned, however, many of the personalized medicines currently under development have shortened stability timelines of 6 to 48 hours, which requires their transport and processing, or administration to occur within a highly abbreviated time frame. To ensure that these cell, gene and immunotherapy shipments arrive on time and within the correct temperature, careful pre-planning must take place. This includes: 

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