Best practice guidelines in the context of rare disease ERNs · Best practice guidelines in the...
Transcript of Best practice guidelines in the context of rare disease ERNs · Best practice guidelines in the...
Best practice guidelines in the context of rare disease ERNs.
Domenica TaruscioNational Centre for Rare DiseasesIstituto Superiore di Sanità, Italy
Definition: Health care guideline(RARE-Bestpractices
Glossary)
Health care guidelines are systematically developed statements by a systematic review of evidence
and an assessment of the benefits and harms of alternative care options,
which assist providers, patients and stakeholders to make informed decisions about appropriate health care for specific circumstances, including clinical interventions, public health activities, or government policies.
Health care guidelines provide recommendations that describe in detail what the recommended action is
and under what circumstances it should be performed.
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Content of this presentation
• Health care guidelines and health care guidelines on rare disease in Europe: present scenario
• Health care guidelines and the EC directive on patients’rights in cross-border healthcare and decisions
• Linking EU directive and decisions with RARE-Bestpractices goals and outputs
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Health policies of individual European member are becoming more interconnected and interdependent
1) for the movement of patients and health professionals across national borders
2) for the creation of European Reference Networks
Important consequences on the quality and safety of health services
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• A mapping exercise illustrates the divergent status of guideline production in the EU (Legido, 2013)
• A more recent Rare-Bestpractices mapping exercise illustrate the considerable diversity in the extent to which EU MS have established systems to develop guideline on rare diseases
Clinical guidelines for chronic conditions in the European Union. 2013 Eds Legido-Quigley H. et al. The European Observatory on Health Systems and Policies-Observatory Series 30
Health care guidelines and health care guidelines and on rare disease in Europe: present scenario
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Within EU all countries are showing some interest in developing and implementing health care guidelines
However there is great variability among countries. Most have fragmentary initiatives led by enthusiasts
Few countries have well-developed systems in place to develop health care guidelines
Clinical guidelines for chronic conditions in the European Union. 2013 Eds Legido-Quigley H. et al. The European Observatory on Health Systems and Policies-Observatory Series 30
Health care guidelines and health care guidelines on rare disease in Europe: present scenario
Which is the level of engagement in guideline production in EU?
Countries with well established activities (for example long-standing programme, tradition)
e.g. Belgium, England, France, Germany, Netherland, Finland, Norway, Sweden, Italy Denmark, Spain, Czech Republic
Countries making progress in the development of health care guideline
e.g. Luxembourg, Latvia, Hungary, Malta, Ireland
Countries “recently adopting” some guidelines or where this are in the planning stages e.g Slovenia, Greece
Clinical guidelines for chronic conditions in the European Union. 2013 Eds Legido-Quigley H. et al. The European Observatory on Health Systems and Policies-Observatory Series 30
Health care guidelines and health care guidelines and on rare disease in Europe: present scenario
Who is responsible for guidelines production in EU?
Guideline produced by a central agency
England, Finland and Luxemburg
Multiple actors involved and there is a central component in place
Austria, Cyprus, Czech Republic, Denmark, Estonia, France, Germany, Hungary, Italy, Latvia, Lithuania, Malta, Norway, Romania, Spain and Sweden
Multiple actors produce guidance without central coordination
Belgium, Bulgaria, Greece, Ireland, Netherlands, Poland, Portugal, Slovakia, Slovenia and Switzerland
Clinical guidelines for chronic conditions in the European Union. 2013 Eds Legido-Quigley H. et al. The European Observatory on Health Systems and Policies-Observatory Series 30
Health care guidelines and health care guidelines and on rare disease in Europe: present scenario
Health care guidelines and health care guidelines and on rare disease in Europe: present scenario
Is there any regulatory basis for the development of guidelines?
The majority of countries has not legal framework
HAS has the statutory duty to develop guidelines and Nice England too. The Directorate general for health in Portugal
There are any formal process to assess the quality of the guidelines? (Example AG
REE II)
The countries with long-standing programme have also system to assess the quality of the guideline produces (England, Finland, Norway)
Clinical guidelines for chronic conditions in the European Union. 2013 Eds Legido-Quigley H. et al. The European Observatory onHealth Systems and Policies-Observatory Series 30
Health care guidelines and health care guidelines and on rare disease in Europe: present scenario
The value of the legal mandate is inconclusive.
Spencer and Walshe suggested that having a legal requirement for quality improvement strategies was an important driver of progres, along with the activiites of professional association sHowever there are example of laws that exist but are not implemented (Italy) or there are highly developed systems without any legal basis (The Netherland)
A study (Burgers, 2003) analyzing 86 guidelines from 10 European countries in a quest to identify predictors of high-quality guidelines, found that guidelines produced within a guidelines programme and by governamental agency scored better than their counterpart. To ensure high quality, guidelines should be produced within a structured and coordinated program
Spencer E, Walshe K. National quality improvement policies and strategies in European healthcare systems. Qual Saf Health Care. 2009 Feb;18 Suppl 1:i22-7. doi: 10.1136/qshc.2008.029355. PubMed PMID: 19188457; PubMed Central PMCID: PMC2629881.
Burgers JS, Cluzeau FA, Hanna SE, Hunt C, Grol R. Characteristics of high-quality guidelines: evaluation of 86 clinical guidelines developed in ten European countries and Canada. Int J Technol Assess Health Care. 2003 Winter;19(1):148-57. PubMed PMID: 12701947.
This European novel dynamic scenarioand RARE-Bestpractices
Rare-Bestpractices mapping exercise★ illustrate the divergent status of guideline on rare diseases in EU
★The survey is lead by Italian Chochrane Centre in collaboration with the Istituto Superiore di Sanità as part of in the RARE-Bestpractices work
This European novel dynamic scenarioand RARE-Bestpractices
13 EU MS have not developed national programme on rare disease health care guidelines yet
3 EU MS have already national programme on rare disease health care guidelines (one on rare tumors)
4 EU MS have plans for developing national programme on RD guidelines in the near future
This European novel dynamic scenarioand RARE-Bestpractices
Why?
Scarcity/absence of evidence makes guidelines on RD useless
Economic reasons
Adopt/adapt guideline developed by other agencies/organizations
Delegate the role of guideline development to other bodies (scientificsocieties, research institutes)
It is not a priority for our national health programme
Reference centre will be designated to do it in the framework of national planfor RD
Health care guidelines and health care guidelines and on rare disease in Europe: present scenario
Health care guideline
on rare diseases
Linking with the directive on patients’ rights
in cross-border healthcare
The directive on patients’ rights in cross-border healthcare
The directive intend to provide greater clarity on the rules governing patients travelling abroad to receive treatment.
It give EU Citizens the right to obtain from abroad any care not requiring a hospital stay without advance authorization
Member stay may create an authorization system to enable them to manage patient flow and avoid threat to the financial and operational sustainability of their own system
In both cases patients will be entitled to be reimbursed up to what would have been paid for if the care was provided at home
Legido-Quigley H, Passarani I, Knai C, Busse R, Palm W, Wismar M, McKee M. Cross-border healthcare in the European Union: clarifying patients' rights. BMJ. 2011 Jan 17;342:d296. doi: 10.1136/bmj.d296. PubMed PMID: 21242212
EU Directive and decisions
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Article 12 European reference networks
The Commission shall support Member States in the development of European reference networks between healthcare providers and centres of expertise in the Member States, in particular in the area of rare diseases
These criteria and conditions shall ensure, inter alia, that European reference networks:
(i) have knowledge and expertise to diagnose, follow-up and manage patients with evidence of good outcomes, as far as applicable;
(ii) follow a multi-disciplinary approach;
(iii) offer a high level of expertise and have the capacity to produce good practice guidelines and to implement outcome measures and quality control;
(iv) make a contribution to research;
EU Directive2011/24/EU of the European Parliament and of the Council of 9 March 2011 on
the application of patients’ rights in cross-border healthcare
Setting out criteria and conditions that European Reference Networks and healthcare
providers whishing to join a European Reference Network must fulfil:
4) To fulfil the requirement set out in point (iii) of Article 12(4)(a) of directive 2011/24/EU)
offer a high level of expertise and have the capacity to produce good practice guidelines and to implement outcome measures and quality control; the Network must:) Develop and implement clinical guideline and cross-border patient pathways
5) To fulfil the requirement set out in point (iv) of Article 12(4)(a) of directive 2011/24/EU) “make a contribution” to
research the Network must: a) Identify and fill the research gap
7) To comply with the requirement set out in point (v) of Article 12(4)(a) of directive 2011/24/EU (“organize and teaching training activities”): (a) identify and fill training gaps
EU Commission Decision10.3.2014C(2014)1408 final
EU Commission Decision10.3.2014C(2014)1408 final
To be ERN member Develop and use
clinical guidelines and patways in their area
of expertise (1)
Develop and implement clinical
guideline and cross-border patient
pathways
Identify and fillthe research
gap
Identify and fillTraining gap
“The network must”
(1) From Criteria and condition for applicants for membership of a
network ((e)(iv) Annex II)
EU Commission Decision10.3.2014C(2014)1408 final
Directive, Decision Guideline on rare diseases in EU MS………………………. linking with
RARE-Bestpractices
.
www.rarebestpractices.eu/
Platform for sharing best practices
for the management
of rare diseases
Project funded EU funded (7FP)
Coordination: National Centre for Rare
Diseases-Istituto Superiore di Sanità, Rome
Duration: 2013-2016
The mandate of the RARE-Bestpractices is
knowledge management to support
evidence-informed decision-making
of the rare disease community &
also to support ERNs
We do this by creating a platform with
resources and tools
to facilitate access and interpretation of the
rare diseases synthesis of evidence
Knowledge Management Platform(www.rarebestpractices.eu)
IT PLATFORMRAREGUIDELINES
RAREGAP
TRAINING(Courses and
Tools)
The RARE-Bestpractices Platform
Knowledge Management Platform(www.rarebestpractices.eu)
IT PLATFORM
RAREGUIDELINES
RAREGAP
TRAININGCourses & Tools
The RARE-Bestpractices Platform
For the benefits of guideline developers and of guideline users
• Database of health care guidelines
• Database of uncertainties / research recommendations
• Standard and tools for guideline development on rare diseases(test GRADE in rare disease context, standard of reporting, toolfor CoI management, for International collaboration, formanagement, for patient involvment etc..)
• A plan of RARE-Bestpractices training courses
For the benefit of rare disease guidelinedevelopers
Increase trasparency
Facilate sharing of information (evidence table, literature search)
Facilitate adaptation of guidelines
Facilitate co-production of guidelines (by multiple guidelinedevelopers, agencies, patient advocacy groups…..ERNs?)
For the benefit of rare disease guideline users
Systematically created health care guidelines are extremelyuseful, invaluable for clinicians and policymakers
Clinicians:
Health care guideline allow clinicians to make appropriate clinical decisions (diagnosis, treatment), and communicate these decisions transparently to patients and families.
Policymakers
Rigorous guidelines also allow policymakers to determine whether a given treatment provides value to stakeholders, so resources can be allocated appropriately.
Resource allocation is particularly important when treatment options are very costly, which is often the case in rare diseases.
RAREGUIDELINE
Contains only guidelines appraised for quality using a validated tool AGREE II
thereby allowing those using the database to identify which guidelines are of high quality.
Inclusion criteria:Guidelines are identified through a structured process of searching known sources of guidelines including existing organizations (e.g. Guidelines International Network) and databases (National Guideline Clearinghouse, Orphanet).
RAREGUIDELINE
RAREGUIDELINE as a resource
Guideline usersRetrieve information to help ensure best possible careInform decision makingSelect the best quality guidelines for their contextProvide feedback/input to guidelines
Guideline developersIdentify existing guidelines and their quality & identify gaps
Collaborate – e.g. invite/collate comment on draft guidelinesDisseminate their new guidelines
Avoid duplication of effort in producing guideline
Information specialists Responsible for developing search strategiesUse as search resourceGenerate bibliographies
RAREGAP is the research recommendation database of the RARE-
Bestpractices project
The role of research recommendations is to highlight uncertainty in
existing knowledge and translating this uncertainty into proposals for
future research
Research recommendations are identified from existing high quality
systematic reviews.
Low methodological rigor may result in the identification of false
uncertainties where further research is not required.
RAREGAP contains details of research recommendations identified in
Cochrane reviews of rare diseases
Training courses and tools
based on the RARE-Bestpractices work to promote the development of trustworthy health care guidelines for rare diseases
and their use across Europe and to support the establishment of European Reference Networks
Istituto Superiore di Sanità
Rome (Italy)Istituto Superiore di Sanità
Rome (Italy)
2 Courses for
guideline developers
Development of
treatment guidelines
10-11-12 February 2016
Milan
Development of
diagnostic guidelines
September 2016
Milan
Cochrane Italy
Challenges, facilitators and opportunitiesfor the production and use of guidelines in rare diseases
Facilitators
Directive 2011/24/EU
supports ERNs, which must have the capacity to produce good practicesguidelines
Public funds available formethodological research in guidelines
i. GRADE working group’ EU-funded DECIDE project
ii.RARE-Bestpractices EU - fundedproject
iii. COMET initiative
IV. DECIDE
Opportunities
MS are at a decisive point in establishingthe criteria to ensure the transparentand effective functioning of ERNs
MS should consider the importance and devote resources
to build efficient systems and capacities for the production of trustworthy guidelines, according tointernational standards
Ms could adopt a cooperative approachto optimize guideline production and implementation across countries
Challenges
Guideline production and implementation is far from satisfactory in the EU
Few Countries have well-established systems in place sustained byregulations that providemechanisms for qualityassurance, implementation and useof clinical guidelines
Many countries still rely on sporadic initiatives, others lack the capacityfor evidence-basedguideline development
Legido-Quigley et al. 2012
Morciano et al. 2015; Menaka et al. for the RARE-BestPractices Consortium, 2015
Challenges, facilitators and opportunitiesto the production and use of guidelines in rare diseases
Opportunities
ERNs can be a concrete opportunity for initiating processes of cooperation among MS in producing trustworthy guidelines, according tointernational standards
Potential results of this cooperation
- shared health care guidelines
- identifying research gaps
- formulate, prioritize and comunicate research recommendations
to researchers and research funders
Menaka Pai et al. for the RARE-BestPractices Consortium, 2015
The relationship between clinical care, research and guidelines
Standards for Developing Trustworthy Clinical Practice Guidelines
1. Establishing transparency
2. Management of conflict of interest; independency
3. Group composition: multidisciplinary and balanced, comprising a variety of
methodological experts and clinicians, and key affected groups. Patient preferences
4. Systematic review of the existing evidence
5. Provide ratings of both the quality of evidence and the strength of the
recommendations
6 Recommendations should be articulated in a standardized form
7. External reviewers should comprise a full spectrum of relevant stakeholders,
including scientific and clinical experts, organizations (e.g., health care, specialty
societies), agencies (e.g., federal government), patients, and representatives of the
public.
8. Updating (IOM 2011)
P - Population: defining the population of interest is a major challenge (smallnumbers of patients, diagnostic uncertainty, etc.)
I- C - Intervention and comparator : there is often only one treatment for anygiven RD.
In many cases, use of placebo is not an option due to the severe course on the untreated disease.
Outcomes: a key step in creating evidence-based guidelines is determingcritical disease outcomes, factors important for patients, providers and healthcare systems. There are several challenges in defining relevant outcomesin RD (case reports, surrogate outcomes, patient reported outcomes).
The most reliable outcomes only be those related to morbidity and mortality
Menaka Pai et al. for the RARE-BestPractices Consortium, 2015
Specific challenges for guideline production in rare diseases:the PICO question
Conclusions (1/2)
The Directive 2011/24/EU is intended to provide a legal framework within the European Union to facilitate cross-border care
Art 12 (Directive 2011/24/EU) supports ERNs, which must have the capacity to produce good practices guidelines
Good quality guidelines, produced according to international standards, do improve health care
MS have a great opportunity, through the establisment of ERNs,
to foster a cooperative and coordinate approach
to share exiting expertise and resources
for the production and implementation of care guidelines
across countries
a cooperative and coordinate approachamong MS to : - optimize guideline production and ameliorate quality assurance practices
- identify and fill knowledge gaps
- formulate, prioritize and comunicate recommendations to researchers and research funders
ERNs can be an opportunityto devise
a new research governance
ERNs are paramount to foster
Conclusions (2/2)
Thank you !
www.rarebestpractices.eu