The methodology for defining the European Standards for the certification of Haemophilia Centres in Europe

Abstract

Introduction

Work Package 4 Development of the standardisation criteria of the European Haemophilia Network project has the main objective of implementing a common and shared European strategy for a certification system for two levels of Haemophilia Centres: European Haemophilia Treatment Centres and European Haemophilia Comprehensive Care Centres in the Member States of the European Union.

Materials and methods

An inclusive and participatory process for developing shared standards and criteria for the management of patients with inherited bleeding disorders has been carried out. The process has been implemented through four different consultation events involving the entire European community of stakeholders that significantly contributed in the drafting of the European Guidelines for the certification of Haemophilia Centres.

Results

The Guidelines set the standards for the designation of centres that provide specialised and multidisciplinary care (Haemophilia Comprehensive Care Centres) as well as local routine care (Haemophilia Treatment Centres). Standards cover several issues such as: general requirements; patient care; advisory services; laboratory; networking of clinical and specialised services.

Conclusions

The drafting of the European Guidelines for the certification of Haemophilia Centres was performed adopting a rigorous methodological approach. In order to build the widest possible consensus to the quality standards, the main institutional and scientific stakeholders have been involved. The resulting document will significantly contribute in promoting standardisation in the quality of diagnosis and treatment in European Haemophilia Centres.

Introduction

One of the work streams of the European Haemophilia Network (EUHANET) project is the promotion of standardisation of the quality of diagnosis and treatment in European Haemophilia Centres. EUHANET is a project funded by the Executive Agency for Health and Consumers (EAHC) of the European Commission¹. Within the EUHANET project, Work Package number 4 (WP4) Development of the standardisation criteria has the main objective of implementing a common and shared European strategy for a certification system for two levels of Haemophilia Centres (HCs): European Haemophilia Treatment Centres (EHTCs) and European Haemophilia Comprehensive Care Centres (EHCCCs) in the Member States of the European Union (EU MS).

The resulting output was the European Guidelines for the certification of Haemophilia Centres².

The main objective of this article is to describe the methodology adopted for an inclusive and participatory process of developing shared standards and criteria for the management of patients with inherited bleeding disorders as a model to be considered for implementation by other groups of rare diseases.

Materials and methods

Operational steps for the development of the European Guidelines for the certification of Haemophilia Centres

The different drafts of the European Guidelines for the certification of Haemophilia Centres (henceforth referred to as Guidelines) have been widely discussed with different European stakeholders (National Health Authorities, Health Professionals, Patient Organisations, EUHANET Project Partnership) at the time of several events and followed a very inclusive methodology of consultation using a bottom-up approach.

The activities of WP4 have been implemented through five steps:

1. review of literature on certification systems of HCs available in EU MS;

2. definition and sharing of principles and criteria to be considered for the development of requirement standards with EUHANET and other collaborating partners;

3. drawing up of a draft of the guiding document for consultation;

4. finalisation of the guiding document incorporating feedback received from the consultation process;

5. proposal of an evaluation system framework for EU HCs.

Review of literature on certification systems of HCs available in EU MS

The specific objective of step 1 was to conduct a survey of available standards and certification systems in EU MS. To reach this goal, and in particular to gather and validate information from official sources that may not be available through common search methods (published materials in official languages, grey literature, etc.), the WP4 team disseminated a preliminary questionnaire to 27 EU MS country stakeholders. 7 out of 17 returned questionnaires contained information about certification systems currently in place in the corresponding 7 countries.

Definition and sharing of principles and criteria to be considered for the development of requirement standards with EUHANET and other collaborating partners

In step 2, a cross-match analysis has been performed using all available materials, such as current national standards and certification systems as well as European key reference documents, including the European Haemophilia Principles of Care³ and EUCERD recommendations^4,5, with the aim of identifying common principles and criteria (i.e. scopes, approaches and rules) to be used as a basis for the draft of the Guidelines.

The WP4 team selected 7 criteria such as types of structures involved, types of diseases to be considered, etc. and disseminated a Questionnaire for evaluating the level of agreement on each of them. Table I shows the list of proposed criteria.

Table I.

List of criteria proposed.

Criteria	WP4 proposals
1 Types of structures involved	Standards for two levels of HCs, in relation to different functions assigned: Comprehensive Care Centres (CCC - providing specialised and multidisciplinary care and functioning as tertiary referral Centres) and HTC (providing local routine care).
2 Types of diseases	Haemophilia and allied inherited bleeding disorders, and acquired bleeding disorders
3 Age groups of patients	Adults and paediatric patients.
4 Reference sources	Standards developed on the basis of main European and national scientific evidence and recommendations.
5 Types of standards	Only “organisational” standards. Standards do not aim to take the place of scientific guidelines. HCs are requested to develop clinical protocols in adherence to available national and European scientific references, and to provide evidence of their correct and systematic application and supervision. General standards for quality system management (according to EUCERD recommendations). Quality indicators to be monitored and evaluated by the HCs to confirm that the clinical outcome reaches the requested standards (according to EUCERD recommendations) and related targets.
6 Assessment	Only standards that could objectively be assessed by objective evidence collected during audits.
7 Issues to be analysed and developed	General organisational requirements - Quality of the facility - Information about the Centre - Organisation and staffing - Policies and procedures - Record-keeping and data collection - Personnel appraisal and continuing education - Equipment - Supply and management of therapeutic products, reagents and medical devices - Quality planning, evaluation and improvement - Participation in institutional registries/databases related to inherited and acquired bleeding disorders - Participation in clinical research Patient care - Awareness, information and education of patients and their families - Diagnosis and therapy of haemophilia and allied inherited bleeding disorders, and all the forms of acquired haemophilia - Periodic clinical and multi-disciplinary review - Genetic services Laboratory Network of clinical and specialised services acting in conjunction with the haemophilia team (physiotherapy, orthopaedic, psychological, dental care, social, infectious diseases, etc.) Formal agreements between the CCC and HTC and between the HTC and third parties whose services impact patient management. Recommended quality indicators (patient care) that should be routinely measured and evaluated by the HCs.

The Principles and Criteria Questionnaire was launched during the European Haemophilia Consortium Annual Conference held in Prague on the 28^th of October 2012. Thus, WP4 collected 56 questionnaires from 26 EU and non-EU countries. The analysis of free text comments and answers showed a very high level of agreement on each of the criteria proposed. This agreement ranged from a minimum of 70% to a maximum of 90% on the total of the questionnaires received (Figure 1).

Figure 1.

Percentage distribution of answers for each of the seven principles/criteria proposed by the EUropean HAemophilia NETwork project - Work Package 4.

Drawing up of a draft of the guiding document for consultation

Once validated, the above-mentioned criteria were used in Step 3 in order to draw up the core draft of the Guidelines document through the following actions: i) definition of standards index and framework, ii) cross reference of EU MS standards, where available, and iii) development of standards.

An example of the resulting table of cross reference of EU MS standards is shown in the excerpt below (Table II).

Table II.

Example of cross-match analysis of some of the relevant documents used as a basis for the drafting of the European guidelines for the certification of Haemophilia Centres⁶–⁸.

EU principles of haemophilia care	EU MS 1	EU MS 2	EU MS 3	EU MS 4	EU MS n
NA	The cornerstone of the treatment of haemophilia is comprehensive care delivered by a multi-disciplinary and specialised team on a 24 hour basis. In practice this involves a core team consisting of the following personnel: - Haemophilia centre medical staff, who carry out routine and emergency treatment, … - Haemophilia nursing staff who co-ordinate much of the day to day treatment, …	Art. 6 The organisation of treatment centre staff always includes the following functions performed by one or more persons meeting the conditions laid down in Artic. 7: Physician coordinator. Physician specialist in internal medicine. Physician specialist in paediatrics. Physician specialist in clinical biology. ………………………….	NA	0.2.1 The Executive Management of the Centre defines and formalises responsibilities, mandates and assigns functions within the organisation…
Standard WP4 (proposal) The cornerstone of the treatment of haemophilia and other related bleeding disorders is comprehensive care delivered by a multi-disciplinary and specialised team. 1.4.1 The core team members of an EHTC/EHCCC consist of the following personnel: - medical staff, who carry out routine and emergency treatment and follow-up clinical reviews; - nursing staff, who co-ordinate much of the day to day treatment and supplies of coagulation factor concentrates; - laboratory staff, who provide a diagnostic and factor replacement monitoring service. - ……………………………………………………………………..

NA: not available

From the set of standards developed, 11 key standards were selected for further consultation through the Questionnaire on key points for the production of standards for HCs and for the development of a related evaluation system, that was presented in Warsaw during the Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). In particular, the issues that were brought to the attention of the European community of stakeholders were related to:

1. proposal for the names of the European Haemophilia Centres: European Haemophilia Treatment Centres (EHTCs) and European Haemophilia Comprehensive Care Centres (EHCCCs);

2. criteria for the designation of a HC as an EHCCC or an EHTC - number of patients/severity:

   1. forty severe haemophilia patients for EHCCCs;

   2. exclusion of patients with moderate and mild haemophilia, as well as those with von Willebrand disease and rare disorders, in the number of patients taken into consideration for EHCCC designation;

   3. no minimum number of patients required for designation of EHTCs;

3. procedures for the designation of EHCCCs/EHTCs:

   1. validity of the certification (3 years);

   2. designation of EHCCCs/EHTCs based upon self-assessment;

4. requirement standards:

   1. Provision of 24-h expert haemophilia medical cover for EHCCCs;

   2. Provision of 24-h medical cover by formalised arrangements with other departments and/or designated EHCCCs for EHTCs;

   3. Establishment of a formal relationship between EHTCs and EHCCCs;

   4. Guarantee of an integrated approach to patient multidisciplinary comprehensive care, also through a formal relationship between EHTCs and EHCCCs;

   5. For both EHCCCs and EHTCs, access to a laboratory that provides a minimum set of coagulation tests (Prothrombin time; activated Partial Thromboplastin Time; Thrombin time and mixing studies; Factor VIII and IX assays; Inhibitor screen; Fibrinogen and factor II, V, VII, X and XI assays; platelet aggregation; von Willebrand Factor multimers) and related monitoring, on the basis of formal agreements between the clinical and laboratory services;

5. availability of information related to EHTCs/EHCCCs on a public website.

More than two hundred European stakeholders participated in the survey. Agreement on each key point was shown in the great majority of the questionnaires received, with the exception of key point no. 2 (Figure 2). On this point, although there was agreement in over 50% of the questionnaires received, the comments received pointed out that in small population countries this threshold could represent a discouraging limitation for EHCCC designation. Moreover, the vast majority of respondents agreed to establish a minimum threshold number of patients (10) for the designation of an EHTC.

Figure 2.

Percentage distribution of answers for each of the key points proposed in the EUropean HAemophilia NETwork project - Work Package 4.

Finalisation of the Guiding document incorporating feedback received from the consultation process

In Step 4, the Guidelines document was revised in light of the results of the consultation process taking into account all relevant comments received. A new version of the Guidelines was then made available for further, final consultation. The final release of the document was approved on the 7^th of June 2013 and launched in Rome at the Italian National Institute of Health on the 11^th of July 2013 during the International Meeting Haemophilia Centre Certification Systems across Europe⁹ and the round table Haemophilia Centres of Expertise in Europe at the European Parliament on 17^th October 2013¹⁰.

Proposal of an evaluation system framework for EU HCs

Finally, in Step 5 the WP4 team outlined the proposal of a framework for the evaluation of applications to be received by European HCs during WP5 activities.

All the entire methodology about consensus building and consultation processes developed in EUHANET WP4 is represented in Figure 3.

Figure 3.

Flowchart of the consultation processes developed in the EUropean HAemophilia NETwork project - Work Package 4.

Results

The European Guidelines for the certification of Haemophilia Centres set the standards for the designation of two levels of European HCs: European Haemophilia Comprehensive Care Centres, that provide specialised and multidisciplinary care and function as tertiary referral Centres, and European Haemophilia Treatment Centres, that provide local routine care. The standards apply to both adults and paediatric patients. Their focus is on organisational issues and covers:

1. general requirements (facility; general policy and objectives; information about the HCs; organisation and staffing; policies and procedures; record-keeping and data collection; personnel appraisal and continuing education; supply and management of therapeutic products, reagents and medical devices; quality planning, evaluation and improvement; participation in registries; participation in clinical research);

2. patient care (awareness, information and education of patients and their families; diagnosis and therapy of haemophilia and other related bleeding disorders and all forms of acquired haemophilia; periodic clinical and multi-disciplinary review; genetic services; outcome indicators);

3. advisory services;

4. laboratory;

5. networking of clinical and specialised services.

Conclusions

The drafting of the European guidelines for the certification of haemophilia centres was performed adopting a rigorous methodological approach. The document came into being as a result of a synthesis of the experience accumulated by the EU MS on HC quality management and certification systems and European key reference documents (European Haemophilia Principles of Care, EUCERD recommendations). The drawing up of the Guidelines involved the main institutional and scientific stakeholders (EAHAD, EHC, EUHASS, EUHANET, EU and non-EU countries) in all the key steps of the project, in order to build the widest possible consensus to the quality standards to be implemented in European Haemophilia Centres.