Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017

1. Zaragoza, 27-28 April 2017
How can we build into the system the ability to review decisions
made on the basis of results? Monitoring Technologies for their
inclusion in the Spanish Health System Benefits Basket
Jesús González Enríquez
AETS-ISCIII
PUBLIC HEALTH CONFERENCE
10th anniversary of the Spanish Network of Health Technology Assessment (HTA) Agencies:
Insights for collaborative networking
Towards Patient and public engagement in HTA

2. Coverage with evidence generation. CEG
Heath authority decisions on coverage/funding conditional on the
collection of data on effectiveness and cost-effectiveness (evidence
generation/development), an option when there is uncertainty of net
benefits of a technology.
 Experimental or under research within the framework of CCT or
registers.
 Research in treatments with few or no alternatives.
 Potential benefits still not clearly demonstrated against
alternatives.
 Without coverage, it is not possible to generate new relevant
evidence.

3. Coverage with evidence generation. CEG
Assumption of risks making decisions under uncertainty:
Type I error : Covering technologies that are clinically ineffective or
harmful.
Type II error: Denying access to potentially beneficial or cost-
effective technologies.
 Specific coverage only in research (for patients involved in the
research).
 Temporary post-introduction covering with research (all patients
are given access).
 Covering with risk sharing (payment linked to results or
performance, number of patients).

5. Coverage with evidence generation
Technology selection criteria:
 Due to diferencies on regulatory approval and access to the
market: Medical devices, diagnostic tecnologies, surgical
procedures and surgical implants.
 Expected improvement in health benefits but still not clearly
shown comparing with rutine effective alternatives.
 Potential impact for widespread and risks with little evidence.

6. Coverage with evidence generation
Technology selection criteria:
 Required regulatory approval.
 Insufficient evidence to answer key questions.
 In the absence of CEG evidence would not become available.
 Feasibility/usefulness of the study design (value of new evidence)
 The benefits of generating additional evidence justify costs,
resources and time.
 Time framework for decision adapted to technology life- cycle.

7. Potential Advantages and Disadvantages associated with CEG
Advantages Disadvantages
Decision makers Managed entry of
promising tech. Evidence
generation.
Investment in tech. that
prove not to be CE.
Additional costs of
monitoring, difficulty in
withdrawing tech.
Healthcare Providers Earlier access to promising
tech.
Risks using tech. not fully
evaluated.
Manufacturers Initial adoption of tech.
with equivocal evidence.
Delays to market access for
effective tech. Additional
burden of monitoring.
Patients Access to promising tech.
otherwise not available.
Access to tech. that may
prove to be ineffective or
unsafe.

9. *Prior pilots of CEG (2000-2015)
*Positron Emission Tomography (PET) in Oncology
*Surgery for epilepsy
*Chondrocyte transplantation
*Stent for Abdominal Aortic Aneurysm
*Artificial Anal Sphincter in Severe Fecal Incontinence
*Neurosurgical treatment for Parkinson
*Intracoronary brachitherapy
Pre - introduction coverage:
*Surgical treatment for HIV-related facial lipoatrophy

10. Monitoring studies
Concept (MoH order 1356 / 2015, 2 July)
Registers for the assessment of health interventions and
techniques (implants) in phase of post-introduction in
benefits basket when:
 There is uncertainty about its effectiveness or its
efficiency in clinical practice.
 Unknown effects in specific population groups.
 Expected high economic or organizational impact.

11. *Monitoring studies
Features
 Technical coordination by an agency of HTA (Spanish Network of
HTA Agencies).
 Application only in health centers designated by the autonomous
communities on the basis of the Protocol.
 Access to all users of the NHS that comply with the Protocol.
 Final report to Health Authority to inform on the inclusion,
exclusion or modification of use in the benefits basket.

12. *Monitoring studies
Objectives
To collect and analyze data that allow you to provide
relevant new information to:
Inform on the inclusion of the technologies in the
benefits basket of services under the current
conditions of use or
modify its conditions of use, or their exclusion
Order by the MoH

13. Data registry
13
Monitoring studies
MS Specific
requirements
CCAA
DECISION
A
G
E
N
C
Y
H
T
A
Health
centers
Protocol
•Inclusion
•Exclusion
•Modify
Final
report
CPAF
Benefits
Basket
Commission
MoH order
Decision of
MoH
CISNS

14. * The agencies of HTA are responsible for
Technical coordination
Coordinating the protocol consensus process
Defining the duration of the study
Defining the criteria for inclusion and exclusion of patients
and the variables and outcomes in the MS
Collecting and analyzing the data
Monitoring and quality control of data
Reporting serious adverse effects occurred during the study
Drawing up the final report

15. *MS AGENCIES
Biodegradable stents for the treatment of
refractory or recurrent benign esophageal
stenosis
AETS-ISCIII
Reduction of persistent air leak with
endobronchial valve implants
SECS
Percutaneous mitral valve repair with
MitraClip for severe symptomatic
mitral regurgitation and heart failure
OSTEBA
Percutaneous left atrial appendage
closure device in patients with atrial
fibrillation
AETS-ISCIII

16. * Monitoring Studies main characteristics
Objectives Size Follow-up Study
duration
Device Company
Esophageal
stent
Safety
Effectiveness
130 1 year 36 m SX-ELLA BiOMED SA
Endobronquial
valve
Safety
Effectiveness
Costs
40 1 year 24 m Zephyr
IBV
Assut Europe
Olimpus
Mitra Clip Safety and
Efectiveness
Cost-
effectiveness
140 1 year 26 m MitraClip ABBOT
Left Atrial
Appendage
closure
Safety
Effectiveness
150 2 years 48 m Watchman
Amplatzer
LAA Ultraseal
Lambre
Boston Sc
St Jude Med
Mercé V
Evomed SL

17. *MS protocol elaboration
Elaboration of the first part of the protocols (tech
description, evidence, requirements, objectives).
Consensus process meetings (clinical experts, MoH,
Spanish Agency of Medicinal Products and Medical
Devices, agencies).
Protocol draft (inclusion/exclusion criteria, variables,
outcomes, follow-up periods, sample size, analysis,
model for informed consent).

18. *MS protocol elaboration
Other characteristics of the study methodology.
Characteristics and requirements of the centers to
participate in the study (expertise, clinical training,
resources).
Reviewed by other designated clinical experts,
companies. involved, and members of the Benefits
Basket Commission (CPAF).
Protocol final approval.

19. *SIEM
The MoH has developed a web based tool to record information
from each monitoring study (Information System of Monitoring
Studies (SIEM).
User manuals for the coordinating agency and for other users have
been developed (secure access, working with the tool, designing
formularies, statistics).
The agencies design the specific protocol database using SIEM.
Authorized clinician at each participating center load the data of
the intervened patients.
The information is finally analyzed by the agency.

20. *Current situation
All final MS protocols have been approved.
Designation by the autonomous regions of the centers
participating in the MS (requirements, authorizations,
signature for adhesion, self-assessment questionnaire,
conflict of interest). 27 Hospital centers in 12
autonomous regions.
Information System of Monitoring Studies (SIEM) is
already operational.
Specific protocol databases design using the tool (SIEM)
completed.
MS in the phase of inclusion of patients.

21. *Discussion
MS is an option for decisions on coverage/funding
conditional on evidence generation when there is
uncertainty of net benefits of a technology.
We need a clear procedure of identification, selection
and prioritisation of technologies suitable for MS.
We have made progress in standardization of
procedures (protocol consensus, handling conflicts of
interest, process of designation of centers).
We need to define the role of companies with interest
in the process.
Adaptation of long technology cycle and the decision
of coverage with additional evidence.

22. *Thank you