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


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



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




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


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


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


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 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


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)


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

http://www.rarebestpractices.eu/

http://www.rarebestpractices.eu/

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

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