Global Cell Therapy Figures Warn of ‘Disastrous Consequences For Science’

What are Cell and Gene Therapies?

Gene therapy. These concern  the transfer of a therapeutic or working gene copy into specific cells  in order to repair a faulty gene copy in an individual.

 Cell therapy – These involve the injection of living cells into the patient to treat a certain condition. – An example being T cells (immune system cells) which improve immune response and the patient’s ability to fight cancer

 The International Society of Cellular Therapy (ISCT) has decided to speak out regarding oncoming health budget cuts in the US due to fears of the consequences for science, clinical care and patients in the US and internationally. The group believes that Trump’s proposed  unprecedented budget cuts to US governmental scientific agencies including the NIH (National Institutes of Health) and NSF (National Science Foundation) could have wide ranging and potentially life threatening ramifications. 

Whilst ISCT does not commonly comment on the government budgets of individual countries, in this specific case, because of the potential widespread global impact of these propositions, ISCT vigorously opposes cuts to the US scientific agencies.

 The ISCT global leadership representing stakeholders involved in cell therapy around the world believes many of the cuts proposed for the NIH by the Trump administration will directly and dangerously impact the international cell therapy, medical and scientific communities. 

 The ISCT President, Dr. Catherine Bollard highlights the example of the proposed elimination of the Fogarty International Center for Advanced Study in the Health Sciences, the NIH program that promotes and funds medical research and training outside the US. For 50 years, the Center has been instrumental in bringing together the international medical and scientific communities to address global health issues such as HIV/AIDS and Ebola. 

 Dr. Bollard believes this is but one example of how the proposed cuts will significantly impact the international scientific community and further erode standards of safety and efficacy in the delivery of treatments to patients globally. 

 Cell and gene therapies are part of a fast moving field with many new drugs progressing to market. Over the last two years the space has gained speed and caught the eye of big pharma , and they are now collaborating with the academic-hospital partnerships who initially dominated the space.

When asked about emerging trends in line with the future of cell and gene therapies, Steven Bradshaw European Director of Market Access Solutions (Europe) noted: “In 2016 new medicines will transform patient care in a number of diseases, including innovative therapies for Alzheimer’s disease, autoimmune diseases and a number of cancers and orphan diseases”, yet the market remains wide open.

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“Although we don’t expect to see any new gene or cell therapies – therapies that offer the potential promise of cure in some cases – to launch in 2016, this is a critical area for industry and payers to access.

The reason being is that these technologies disrupt not only the treatment landscape, but also how medicines have been traditionally evaluated by payers and how they are paid for and delivered. 

“The concept of upfront payments are not popular for payers when such therapies carry price tags even greater than Glybera and for increasing numbers of patients, and when often the efficacy / [cost benefit balance is not revealed for years], in some cases. Performancedriven, annuity based payments, risk pools, mortgages or even licensing for payers (similar to software licensing in the IT world) are all under discussion, but it will be for the manufacturers and not payers to establish these.

“Manufacturers need to be especially proactive in establishing innovative payment schemes and should focus on thinking outside the box or learning from other industries by multistakeholder consultation to avoid delay of access [to] effective therapies.” 

Payment schemes are just one trend of focus that is surfacing within the cell and gene therapy manufacturing stratosphere. Eyes in the market are also set on aspects such as: overcoming technical hurdles to get discoveries to perform, transporting product to market faster and optimising the use of closed system processing.