Pan-European Patient Network for Information on Rare Diseases and Orphan Drugs - PARD 3 -

Transcription

1 EUROPEAN COMMISSION DIRECTORATE GENERAL HEALTH AND CONSUMER PROTECTION Pan-European Patient Network for Information on Rare Diseases and Orphan Drugs - PARD 3 - A project conducted by Under the Programme of Community Action on Rare Diseases Contract n SPC Intermediate Activity Report July 2003

2 Table of contents I - INTRODUCTION... 3 II - EXECUTIVE SUMMARY...4 III - PROGRESS REPORT... 6 IV - NEXT STEPS V - LIST OF ANNEXES

3 I - Introduction This document describes the work carried out under the project entitled "Pan- European Patient Network for Information on Rare Diseases and Orphan Drugs", internally referred to as "PARD 3 project". The objectives of the "PARD 3 project" are: - to gather information needed to build a public policy on rare diseases, - to improve the access to quality information on rare diseases and orphan drugs. This is the third part of a long-term project initiated by Eurordis in the framework of the Programme of Community Action on Rare Diseases /2003, the objective of which is to create and animate a European trans-national network on rare disorders, geared at patient organisations and patients. The report begins with an executive summary of the project status and provides an updated timeline of activities. It then describes the project management, the activities successfully completed and the progress made to date. The report then maps out remaining activities to be carried out in order to achieve the expected results. Detailed information can be found in the annexes. 3

4 II - Executive Summary th Since December 30, 2003, the following results have been achieved in the framework of the PARD 3 project. 2 European workshops held involving partners from 12 countries Questionnaire created for quantitative survey and validated at workshop Questionnaire submitted to provider to prepare for issue to partners and contacts and for translation into 7 languages Interview template created for qualitative survey Uniform procedure developed to ensure equable interview process across borders Involvement of Eurordis Directors and/or Steering Committee members as seconders in interview process Submission of interview texts to interviewees for validation Synthesis of interviews begun Clear indicators of good practice derived from interview process Clear indicators of barriers to obtaining information from interview process Clear indicators of different(but valuable) practices in different countries Refining of questionnaire for quantitative survey in light of qualitative survey results Bench mark of good practices and key elements for guidelines identified at second workshop First European Awareness Week on Rare Diseases conducted, introducing several partners to the process of press releases, media handling and use of websites as a marketing tool in addition to involving their members at grass-roots level Collection of 31 patient profiles which will form the basis of a continuing process and record of patient experience in the field of rare diseases Monthly meetings of the steering committee to monitor progress, develop and refine future planning Programme for Pilot sessions drafted Programme for National Alliance workshops to develop improvement plans at National and European level drafted for discussion and validation at third European workshop in Paris in October 2003 In the light of past experience and allowing for summer holidays, redrafting of the final phase of the project timetable to ensure realistic goals and achievable outcomes for our partners 4

5 Below is a visual overview of the activities either already conducted or to be conducted until project completion. Action Jan. 03 Feb. 03 Mar. 03 Apr. 03 May 03 Jun. 03 Jul. 03 Aug. 03 Sep. 03 Oct. 03 Nov. 03 Dec. 03 Jan. 04 Feb. 04 Mar. 04 European Workshop in Paris Qualitative survey European Workshop in Namur European Awareness Week on Rare Diseases Quantitative survey Pilot test of charter and manual European Workshop in Paris National workshops Dissemination 5

6 III - Progress Report 1. Team Project Leader Lesley Greene, Eurordis President until My 24 th, 2003 Project Coordinator Claire Marichal, Eurordis Project and Administrative Officer Coordinator (from May 2003) François Houÿez, Eurordis Project Manager, Access to Care Additional Steering Committee members Elisabeth Kampmann-Hansen, Director, CSH (Danish Centre on Rare Disorders) Michele Lipucci di Paola, Eurordis Director Anne Schaetzel, General Delegate, Maladies Rares Info Service Yann Le Cam, Eurordis Chief Executive Officer Between the submission of the project and its approval by the European Commission, Ms. Youngs, the proposed Project leader, changed jobs and was appointed Director of Public Affairs at the UK Dyslexia Association. As dyslexia is not a rare disease and as her new job was very demanding, it was decided to appoint a new project leader having the experience and skills to take over the project without any additional delays. As long as the Webportal based on PARD2 project was not fully operational, it was felt that a more effective way of implementing the staff resources available for this project would be to postpone the recruitment of an Information manager / Webmaster to Summer 2003, and meanwhile to recruit a project manager to assist the project coordinator and project leader, which was done in May At the same time, the project coordinator was off work for three weeks, which necessitated the involvement of the Eurordis Chief Executive Officer for that period. Additionally, some changes were made to the originally proposed Steering Committee in order to involve national helplines as much as possible. 6

7 2. Project monitoring Throughout the project, the Steering Committee is in charge of supervising the implementation of the project. Meetings take place once a month, mostly by phone. It has helped draw a new timetable and sequencing of actions. 3. Revised schedule The PARD 2 project - Contract SI (2001CVG4-807) - carried out by Eurordis in 2002 was continued with the agreement of the European Commission th th (Amendment n 1 signed on December 10, 2002) until December 29, 2002, thus postponing the start of the PARD 3 contract while still keeping the same th deadline of December 30, 2003, i.e. 12 months instead of 15. th The PARD 3 project is therefore planned to be conducted from December 30, th 2002 to December 30, A revised schedule was therefore designed to take into account the later start of the project and build around set events. (see next page) 7

8 Original timing Phase I - Survey - December February 2003 Survey of information practices and identification of national best practices (fact finding survey: questionnaires sent to 500 organisations, national meetings and in-depth interviews in 10 countries, analysis). Trial test of Web-Site with survey questionnaire posted on-line to reach out of the audience in the EU and non-eu States. 2 days Workshop in Paris with partners involved in this Network to present conclusions benchmark best practices and identify key elements for the guidelines. A further outcome of the workshop will be the validation of an action plan for the European Awareness Week on Rare Diseases in Phase II Best practices and support package - March-April 2003 Development of guidelines on best practices and a support and training package. 2 days workshop in London for consensual validation of guidelines and package and final co-ordination of European Awareness Week. Phase III - Implementation May - September European Awareness Week On Rare Diseases (envisaged May 2003) coordination and support to national preparation - use web-site as Pan- European co-ordination tool and implementation. - Pilot test of guidelines and of support and training package in recent alliances and accession countries, 2 days in 2 countries. - Development of national information improvement action plan - National workshop in each of 10 network countries to develop national information improvement plan Phase IV - Evaluation and Synthesis - October 2003 Workshops around the 2 nd European Conference on Rare Diseases and Disabilities in Paris, in partnership with the French Rare Diseases Alliance involving all the national alliances and information helplines, and European Commission representatives to Share all the National Improvement Action Plans with a view to integrating best practices. Based on experience gained, revise and get final validation of the Support & Training Package; identify the possibility of future European Integrated Projects on information to patients on rare disorders and orphan drugs; Evaluate achievements and added value of the 1 st European Awareness Week on rare diseases and plan action for the 2 nd one; Identify essential recommendations and findings of the project. Phase V - Dissemination Nov - Dec 2003 Preparation, validation and publication of a guideline on producing and assessing quality of information to patients on rare diseases and orphan drugs. Interactive interface on web-site for dissemination of the guide and feedback gathering. Updated timing Quant.: 09-11/03 Qual.: 03-08/03 Paris Workshop: 17-18/01/03 to prepare survey and EAWRD 05-09/03 Namur Workshop: 23-25/05/ /05/ /09/03 27/10/03-15/01/ /10/ /2004 8

9 4. Activities achieved to date 1. Paris Workshop (17-18 January 2003) This first workshop had three objectives: design the quantitative questionnaire, validate the methodology of the quantitative and qualitative phases and plan the first European Awareness Week on Rare Diseases. We indeed preferred to ask our partners for their inputs at design stage rather than at validating stage. Date : January 2003 Location : Paris, France Chaired by : Lesley Greene (Project Leader) 18 participants : 1-2 participants per partner (Denmark, Italy, France, Spain, Sweden, UK, Belgium, The Netherlands, Estonia), CEO and staff. (5 participants were sponsored by Eurordis). The project coordinator first gave an overview of the project and the team, emphasising the objectives, the means and the role of the partners and briefly presenting the survey. Participants were split into five groups to study the structure and wording of the quantitative questionnaire as well as the organisation of the fieldwork and the methodology of the qualitative phase. After the four groups had reported back the results of their discussions, the European Awareness Week was discussed. (See Annex 1a for agenda, Annex 1b for preparation of Awareness Week, Annex 1c for Quantitative questionnaire and Annex 1d for Workshop report) 2. Qualitative survey (April-July 2003) A survey of information practices was the key action to have a first view of the situation so as to build from there, identifying good practices, disseminating training packages and encouraging countries to develop national improvement plans. As we started contacting market research institutes to discuss the survey with them, we progressively realised that it would be helpful to conduct the qualitative phase first, instead of starting with the quantitative phase, as planned in the initial project. The results of the qualitative survey indeed helped us to refine the quantitative questionnaire. We therefore conducted 31 in-depth interviews in nine countries in April and May 2003, using the same methodology in each case. (See Annex 2a for instructions to interviewers, Annex 2b for information to respondents, Annex 2c for the list of interviews and Annex 2d for the template of the qualitative interview reports.) 9

10 A first synthesis was provided to the participants in the Namur workshop of May The transcription work continued until July when interviewers and respondents were asked to validate the interview reports. We plan to conduct additional interviews by phone in the coming weeks in Sweden and Portugal, either because some organisations could not be interviewed during our visits or, in the case of Portugal, because the original trip had to be postponed. Cooperation with Orphanet and NEPHIRD The cooperation initiated with Orphanet during the PARD 2 project is continuing. Additionally first contacts have been taken with NEPHIRD during the present project. Both networks have been approached to contribute to the survey conducted by Eurordis. During the qualitative phase of the survey an interview was conducted at the National Centre on Rare Disorders in Italy. During the quantitative phase of the survey, Orphanet and NEPHIRD will mainly contribute by providing a link from their own website to the quantitative questionnaire posted on the Eurordis website. The paper version of the questionnaire will also be sent to the patient organisations listed in the Orphanet directory. 3. Namur Workshop (23-25 May 2003) During the Paris workshop of January 2003, the participants decided to organise the next meeting around the Eurordis General Assembly to be held in Namur, Belgium on May 24 th, 2003, as it was a unique opportunity to gather so many organisations for two days. Date : May 2003 Location : Namur, Belgium Chaired by : Lesley Greene (Project Leader) 25 participants : 1-4 participants per partner (Denmark, Italy, France, Spain, Sweden, UK, Belgium, Hungary, Germany, Greece), CEO and PARD 3 Steering Committee. As this meeting took place just after the series of in-depth interviews in 9 countries, the aim of this workshop was to report on the qualitative survey, analyse its results and validate the work process from interviews to guidelines. It was essential for the participants to define what was considered as good practices and what practices should be avoided, as well as what obstacles paved the way to good information and how to overcome them. The final step consisted in drafting a tentative structure for the manual. (See Annex 3a for the agenda, Annex 3b for the list of participants by working group and Annex 3c for the workshop report.) 10

11 4. European Awareness Week on Rare Diseases (24-31 May 2003) The preparation of the first European Awareness Week on Rare Diseases started in June 2002 at the National Workshop of National Alliances held in Barcelona when the National Alliances were consulted about the type of event we should organise. It was then further discussed at the Workshop held in Paris in January 2003 and at PARD 3 steering committees early th Finally, an announcement was sent to Eurordis members on May 6, 2003 and an announcement was posted on the Eurordis website to facilitate its coordination and implementation. th The European Awareness Week was launched in Namur on May 24, 2003 on the st occasion of the Namur Workshop, and lasted until May 31, covering events both at Eurordis and country level. (See Annex 4a for the announcement to members, Annex 4b for the article to be published in the next Eurordis newsletter, Annex 4c for the announcement made on the Eurordis website, Annex 4d for personal profiles of rare diseases and 4e for an example of balloon tag used for the balloon release in Namur.) 11

12 IV - Next steps of the project Our first goal is to reach the expected results: - Results of the fact-finding survey - Identification of best-practices for improved information and access to information for patients - Guidelines on producing and assessing quality of information on rare diseases and orphan drugs (Charter) - Know-how transfer through support and training package particularly for new national alliances and accession countries (Manual) - Strengthening of co-operation between existing national structures - Development of at least 2 new national alliances of patients' organisations - Further development of Web-prototype into an interactive platform structuring the network - First European Awareness Week on Rare Diseases. We have therefore created a realistic schedule for the remaining part of the project based on experience from the first half of the year. (See Annex 6 for the latest detailed project timetable as of end of July 2003.) 1. Development of a charter and a manual (August-September 2003) A charter and a manual are two of the main tools we wish to develop. These tools will be based on the qualitative interview reports and the discussions of the Namur workshop. The charter lists the basic rules that need to be followed when handling information on rare diseases. As to the manual, it lists good practices and gives advice on how to start and manage a helpline. A synthesis of the situation by country will also be made to be used during the national workshops of November Pilot sessions (September 2003) The charter and manual will be tested in recent alliances and accession countries. We decided to organise such events in at least two countries. Initial contacts have been made with Greece, Hungary and Portugal. We will discuss the organisation of these workshops with our contact persons in these countries in order to ensure that the programme is adapted to their own situation for participation and for results to be optimal. The objective is to help create a national alliance and/or a helpline project. 12

13 3. Quantitative Survey (September-November 2003) The questionnaire discussed with the partners during the Paris workshop has been refined and re-submitted to the market research companies for them to confirm their quotes. We selected Research International and agreed on a timetable for the survey. (See Annex 5a for the latest draft of the questionnaire, Annex 5b for the offer from the selected provider and Annex 5c for the new agreed schedule.) Because of the holidays, we shall start the fieldwork in September in order to ensure as high a response rate as possible. The survey will be conducted in 13 countries and translation will be done by our partners in the various countries. Its aim is to provide a more quantitative idea of the state of information services and practices and to highlight obstacles to a good access to information in the various countries. 4. Paris Workshop (17-18 October 2003) As the national workshops will occur in November 2003, the programme of the Paris workshop, to be organised just after the 2nd European Conference on Rare Diseases and Disabilities, has been slightly changed. We plan to report on the qualitative survey, prepare the national workshops, validate the charter and manual based on the experience of the pilot sessions and evaluate achievements and added value of the 1st European Awareness Week on rare diseases in preparation for future awareness weeks. We will invite Orphanet, NEPHIRD, EFPIA, EuropaBio and European Commission representatives. 5. National Workshops (27 October - 15 January 2004) National workshops will take place in 10 countries from October 27 th to November 30 th, i.e. after the Paris workshop in order to leave partners more time to organise these events. Their main goal is to develop improvement plans at national level. Each one-day meeting will therefore study the situation at country level and suggest an improvement plan at association, country and European levels. Partners will be asked to report result, using a template to ensure that results are comparable. Analysis will follow until January The synthesis of the national improvement plans, initially planned at the October workshop, will be made accessible to Eurordis members through the passwordprotected area of the new Eurordis web portal. 13

14 6. Dissemination (January-March 2003) The last phase of the project will be dedicated to the dissemination of: - A survey and report on best practices of information and access to information of rare diseases patients - Support and Training package for national patient organisations - The publication of a guide on producing and assessing quality of information to patients on rare diseases and orphan drugs and its wide distribution to reach 2000 patient organisations stakeholders. However we will also post on the Eurordis website as much information as possible from the end of August onwards, i.e. as soon as the Eurordis Webmaster / Information Manager joins the team. Dissemination through the Internet will not only increase the outreach of the project but also enable to update the documents regularly as other good practices are identified. 14

15 IV - List of annexes N Type of document Page 1a Paris Workshop (January 17 th -18 th, 2003): agenda 16 1b Paris Workshop: Preparation of the European Awareness Week 18 1c Paris Workshop: PARD 3 Quantitative Questionnaire 22 1d Paris Workshop: Report 24 2a Qualitative Interviews (April May 2003): instructions to interviewers 27 2b Qualitative Interviews: information sent to respondents 34 2c Qualitative Interviews: list of interviews 37 2d Qualitative Interviews: template of qualitative interview reports 40 3a Namur Workshop (May 23 rd -25 th, 2003): agenda 46 3b Namur Workshop (May 23 rd -25 th, 2003): participants by working groups 49 3c Namur Workshop (May 23 rd -25 th, 2003): workshop report 51 4a 4b 4c 4d European Awareness Week (May 24 th -31 st, 2003): announcement to members European Awareness Week (May 24 th -31 st, 2003): article published in Eurordis Newsletter European Awareness Week (May 24 th -31 st, 2003): announcement made on Eurordis website European Awareness Week (May 24 th -31 st, 2003): personal profiles of Rare Diseases e European Awareness Week (May 24 th -31 st, 2003): balloon tag 116 5a Quantitative Survey: latest draft of questionnaire 118 5b Quantitative Survey: offer from selected provider 127 5c Quantitative Survey: schedule Project Timetable as of end of July

16 1a. Paris Workshop (January 17 th -18 th, 2003): agenda 16

17 1b. Paris Workshop: preparation of the European Awareness Week 18

18 1c. Paris Workshop: PARD 3 Quantitative Questionnaire 22

19 1d. Paris Workshop: report 24

20 2a. Qualitative Interviews (April May 2003): instructions to interviewers 27

21 2b. Qualitative Interviews (April May 2003): information sent to respondents 34

22 2c. Qualitative Interviews (April May 2003): list of interviews 37

23 2d. Qualitative Interviews: template of qualitative interview reports 40

24 3a. Namur Workshop (May 23 rd -25 th, 2003): agenda 46

25 3b. Namur Workshop (May 23 rd -25 th, 2003): participants by working groups 49

26 3c. Namur Workshop (May 23 rd -25 th, 2003): workshop report 51

27 4a. European Awareness Week (May 24 th -31 st, 2003): announcement to members 61

28 4b. European Awareness Week (May 24 th -31 st, 2003): article published in Eurordis Newsletter 63

29 4c. European Awareness Week (May 24 th -31 st, 2003): announcement made on Eurordis website 66

30 4d. European Awareness Week (May 24 th -31 st, 2003): personal profiles of Rare Diseases 68

31 4e. European Awareness Week (May 24 th -31 st, 2003): balloon tag 116

32 5a. Quantitative Survey: latest draft of questionnaire 118

33 5b. Quantitative Survey: offer from selected provider 127

34 5c. Quantitative Survey: schedule 136

35 6. Project Timetable as of end of July

36 This report was produced by a contractor for Health & Consumer Protection Directorate General and represents the views of the contractor or author. These views have not been adopted or in any way approved by the Commission and do not necessarily represent the view of the Commission or the Directorate General for Health and Consumer Protection. The European Commission does not guarantee the accuracy of the data included in this study, nor does it accept responsibility for any use made thereof.