Highly Specialized Procedures

As the basis for a cross-border health action, we define highly specialized procedures (HSP) as those procedures that for a number of reasons i.e. economical, lack of expertise or awareness, are not available in all EU-MS, thus preventing the delivery of the best care for EU citizens suffering from a rare haematological disease (RHD) independently of their country of origin.

These HSP are classified as "under the scope" of the Directive 2011/24/EU if they are defined as standards of Care (SOC) and/or included in the national basket of health services for patients or "out of the scope" in the cases that they are still performed on academic or experimental environment. In these cases, the European cooperation can be produced on the research field.

HSP involve both interventions for diagnosis and for treatment, and their complexity can rely on technological advances or expertise of multidisciplinary team, or both.

Bone Marrow Transplant for Non-oncological diseases

Bone Marrow Transplantation (BMT) is a procedure widely implemented and standardized for oncological RHD, however expertise and capacity required for its performance on non oncological RHD differ from the medical perspective and present high inequalities on its availability across EU. Accordingly, ERN-EuroBloodNet identified BMT for non oncological diseases as a HSP priority for an action on the field of cross border health.

An online survey was conducted among the 56 ERN-EuroBloodNet HCPs covering non-oncological diseases from December 2018 to July 2019. The mapping exercise included:

• BMT need: To assess diseases for which the respondent consider the BMT for the correct management of the patients,
• BMT availability: To analyze for which non-oncological RHDs and patients' age the respondent's center offers the BMT,
• State of the art of BMT cross-border: To assess if referrals to other centers are considered when necessary and if a standardized procedure is in place in such cases.

A total of 48 responders from 44 HCPs of 14 MS answered the exercise. Comparison between need for BMT per disease declared by the HCPs and availabilty of BMT is shown in Table 1.

SCD is the condition for which the difference between need and availability of BMT is the highest, availability is 23,3 points below the need, followed by Thal in which availability of BMT is 10,8 points below the need. In 15 HCPs which consider BMT for SCD patients the procedure is not available. From them, 12 confirmed that they refer patients to another centre, 10 in the same country and 2 abroad. However, only 7 have a standardised procedure for referral of patients. In 13 HCPs which consider BMT for Thal patients the procedure is not available. From them, 11 confirmed that they refer patients to another centre, 9 in the same country, 1 in the same country or abroad, and 1 abroad. However, only 7 have a standardised procedure for referral of patients.

Information gathered has provided the evidence required for facilitating shaping public health policies addressing disease specific needs in the clinical management of non-oncological RHDs patients at the national level, while shedding light into the current EU status of highly specialized procedures identified of added value for the establishment of a cross-border referral system. In this context, a first pilot collaboration agreement for cross border health for BMT on SCD pediatric patients has been established between two ERN-EuroBloodNet members, one from Italy and one from Ireland.