OBJECTIVES: In April 2016 European Medicines Agency (EMA) approved an autologous CD34+ ex vivo gene therapy product for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). As this product would be administered in some specialist accredited transplant centers through Europe, classic reimbursement criteria should be revised.

METHODS: Available EU and national legislation has been searched in order to identify available negotiation tools for the following two cases: a)integration of gene therapies under the general framework of a national policy (leading to national pricing and reimbursement) b) adaptation of the benefit package provided by reimbursement funds to gene therapies and c) study of the existing administrative procedure of approval and reimbursement in the field of transplantations of human tissues, which presents important similarities to the ex vivo gene therapy studied.

RESULTS: According to Regulation (EC) no 883/2004 a prior authorization is needed in all cases of programmed treatment in another EU Member State (MS). The authorization shall be accorded where the treatment in question is among the benefits provided for by the legislation in the MS where the person concerned resides and where he cannot be given such treatment within a time-limit which is medically justifiable, taking into account his current state of health and the probable course of his illness. Another approach is as all candidates for the gene treatment will be treated in an accredited center in other MS discounts and price-volume agreements could be applied according to national legislation.

CONCLUSIONS: The lack of a common framework for the reimbursement of gene therapies could lead to the non-reimbursement of new therapies. Furthermore, the development of specialized centers throughout MS could be more efficient than the development of a network in each MS. That perspective leads national healthcare systems in joint negotiation procedures.