EBMT NEWSLETTER | November 2017 | Volume 59 - Issue 5

Important dates
Dear friends and colleagues,
The EBMT is fully determined to create new directions for the future, and we are proud to play a key role in the scientific and medical community, helping to give our field a global outlook.
The recent approval of two CAR T-cell products is a true revolution in our field. Scientifically, we have never been in a better position to advance treatments in life-threatening diseases. Basic scientific research fuelled by the tools of molecular biology and genomics, has generated unprecedented knowledge. We can now manipulate and genetically engineer many of the cellular effectors that allow for long-term disease control and cure. And, increasingly, we know how to personalize therapy to the unique genetics of the tumour and the patient. The advent of CAR-T cells will enrich the therapeutic armamentarium. It also represents a great opportunity to rejuvenate the cellular therapy field. However, the post-regulatory appraisal will allow investigation of the real-life clinical effectiveness, and testing the capacity of all stakeholders to invent new models for affordability.
EBMT is committed to allow access of all patients to CAR-T cell therapy.  However, the announced price tags for such products appear to be quite prohibitive. Also, the latter does not include the cost of patient care in the weeks following administration. This includes, among others,  those of prior lympho-depleting chemotherapy, and the management of potentially severe complications, such as cytokine release syndrome or neurological toxicities that may add up another substantial expenditure on top of that of the cellular therapy product itself. The advertised prices are way above the cost of an allogeneic stem cell transplant procedure in most EBMT centres, as well as of the already inflated price for other immune-oncology drugs. Reimbursement of CAR-T cell products will be hard-pressed harsh in most developed countries, not to mention those with low- to middle-income.
Handling of CAR-T cells on a routine basis will require a strong culture of quality assurance in the delivery of complex therapeutic procedures, as exemplified by the accreditation process that demonstrates compliance of transplant programs with the FACT-JACIE International Standards for Hematopoietic Cellular Therapies. Thus, the EBMT community is well prepared to meet the medical challenges raised by the use of innovative cellular therapies. However, we fear that cost concerns could lead to unacceptable delayed access for patients who are in immediate need.
We, at EBMT, believe in the “academia-pharma” strong partnership for the good of our field. With this in mind, EBMT is committed to identifying organizational and technical solutions to lower costs, and to promoting original reimbursement strategies through discussions with manufacturers, regulators, payers and patients’ associations. Besides efficacy and safety, accessibility and affordability are our mantra to tackle this new era, for harnessing innovation, and transforming patients’ care and health systems.
Over the long term, we will be judged by our capacity to defend our patients’ interests. EBMT promises to keep playing this role all year around. You can, as usual, count on my energy. It is always an honour and a pleasure to serve EBMT and its members!

In the meantime, please do not forget to follow the EBMT news on Twitter @TheEBMT and @Mohty_EBMT.
Best regards,
Mohamad Mohty
EBMT President
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