Health Technology Assessment The Regional Administration Role · Health Technology Assessment The...
Transcript of Health Technology Assessment The Regional Administration Role · Health Technology Assessment The...
16 april 2010Reggio Emilia
Health Technology AssessmentThe Regional Administration Role
Luciana BalliniOsservatorio Regionale per l’Innovazione
Summary
ORI and the governance of innovations
ORI and researchResearch gaps and research questions
Research Design support/service
The example of Image Guided Radiation Treatment
• Understand potentials of innovation
• Evaluate state of knowledge
• Identify organizational, professional, economic prerequisites
• Propose plans for adoption
• Develop programs for further evaluation
Support to the governance of innovations
Regional Health AuthorityRegional Health Authority
Health Trusts (AUSL e AOSP)
Health Trusts (AUSL e AOSP)
Emerging technologiesClinical-organizational innovations
Emerging technologiesClinical-organizational innovations
Innovative technologies: special features
In this situation of uncertainty it isnevertheless necessary to make a
decision
“…when there are reasonable grounds for believing that a technology will offer significant benefits but there is uncertainty around the clinical or cost‐effectiveness of the technology. “
Uncertainty is the main source of doubt and indecision
The decision affects the development of further
evidences
Governance of Innovations
Cost of waiting for betterinformation
Cost of premature diffusion
Delay / denial of effective care
Spread of ineffective / harmful care
Withdrawing a service is more difficult than witholding it
Some (cautious) decisions
• Risk‐sharing agreement
• Dose capping
• Price‐volume agreement
• Outcomes‐based reimbursement schemes
Uncertainty used as a mechanism to “adjust” expenditure
No direct actions aimed at reducing uncertainty
Conditional Coverage Policy
A powerful tool forEvidence‐Based Decision Making
Coverage with Evidence Development (CED)Only in Research (OIR) Option
Coverage with Evidence Development (CED)Only in Research (OIR) Option
Protects the evidence base of emerging technologies
Allows to give coverage for an innovation provided that eligible patients take part in clinical trials aimed at providing further and robust results on clinical
benefits and risks.
Cost of waiting for betterinformation
Cost of premature diffusion
HTA PROCESS
RETROSPECTIVE HTASystematic review of evidence
Economic Analysis
Organisational, legal, ethical implications
Recommendations for adoption / use
PROSPECTIVE HTAClinical Trials
Observational studies
Clinical audit
Cost‐effectiveness
Recommendations for adoption / use
ORIConceptual Knowledge
(the rationale)
Instrumental knowledge
(review of evidence)
Research pathway
(research gaps)
Recommendations for research
HTA process: from the policy question to the research question
Policy questions
“If research is the answer, what is the question ?”Jonathan Lomas
1. Should we acquire it and how ?
2. Should we use it and how ?
3. Should we diffuse it and to whom ?
4. Should we pay for it and how much ?
Do we need it now ?
HTA question:The rationale for the innovation
The reasonable grounds for believing that a technology will offer significant benefits
IGRT‐IMRT
Radiation treatment
Dose‐tolerance threshold
Treatment intent
Proximity to vital organs
Dose‐targeting
Patient set‐up
Organ motion
Acute/low toxicity
Treatment response
Imaging
Dose intensity Alfa/beta
ratio
Fractionation
“A better correction for set-up errors and organs’ motion and a consequent more accurate dose targeting can decrease toxicity and/or increase clinical
effectiveness of radiation treatments with radical intent of tumours in proximity of vital organs
Defines the potential clinical useand the target population
The rationale
IGRT\IMRT
The clinical scenarios
Tumour Clinical scenarioProstate • Radical radiation treatment for patients with
low or intermediate risk prostate
Lung • Radical radiation treatment for patients withT1 e T2 / in stage IIA, IIIA e IIIB
•Radiation treatment for metastatic cancer
Head & Neck
• Radiation treatment with radical intent with hypofractionation – exclusive or associated with chemotherapy – in patients with any type of head and neck cancer, excluding those of the larynx,)
Brain • Radical radiation treatment for primary tumour• Radiation treatment for metastatic tumour
Pancreas • Pre-operative radiation treatment• Post-operative radiation treatment• Radiation treatment for inoperable pancreatictumour
metodi
The evidence profile: the outcomesDimension OutcomeTechnical Performance Set-up error
Organ motion
Feasibility Patient’s complianceLearning curveCosts
Safety Acute toxicity / adverse effectsLate toxicity / adverse effects
Clinical efficacy Surrogate outcomesTreatment’s responseLocal controlLoco-regional controlSecondary outcomesDisease free survivalProgression free survivalQuality of LifePrimary outcomesDisease specific survivalOverall survival
Technical
Performance
Feasibility Safety Clinical
Efficacy
Cost‐effectiveness
“Evidence profile” and Review of Literature
The Evidence Gaps
Simulations +
planningUncontrolled case series
Controlled case series
CCT
RCT
RETROSPECTIVE HTA
The role of experts
Evaluation of immature technologies :
definition of the potential clinicalbenefit of a technology
Experts decide clinical use and population target
Agree on the relevant clinical outcomes
Define the evidence profile
Define the research questions
Prioritize the research needs
AVAILABLE DATA AND INFORMATION
Estimated annual regional target population (all types of treatment)
78 (18% prevalence)
Estimated cost of IGRT/IMRT treatment € 7 400 – 8 700 [23‐30 fractions]
Estimated cost of 3D‐CRT treatment € 4 488 [30 fractions] VOTES
Outcome Estimate 3D‐CRT ExpectedIGRT/IMRT
Clinical relevance of outcome *
Outcome’s clinical relevance*
Outcome’s relevance in a clinical trial*
Acute toxicity
Enteritis G2: 60%G3‐4: 15‐20%
< No studies
Late toxicity
Duodenal stenosis 2‐3% < No studies
Clinical efficacy
Disease specific survival at 2 yrs
50‐60% (neg. margins)9% (pos. margins)
> No studies
Overall survival at 2 yrs 50‐60% (neg. margins)9% (pos. margins)
> No studies
Severity of disease: mortality*
morbidity*
Expected clinical impact of the tecnology: mortality*
morbidity*
Feasibility of a regional clinical trial (Number of patients, of participating centres, resources availability, etc.)*
low moderate high
1 2 3 4 5 6 7 8 9
Pancreas
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VOTE ON PRIORITY
RECOMANDATIONS FOR RESEARCH
Innovative Radiation therapy:
IGRT‐IMRT
ORIentamenti 2
Robotic assisted surgery:
Da Vinci Robot
ORIentamenti 1
Prostate cancer
Colo‐rectal cancer2 CCTs
3 RCTsProstate cancer
Head & Neck cancer
Lung cancer
ORI:Research Design support/service
ORI & Innovative technologies: limits
THE CONCLUSIONS
• Not based on comparative evaluations (competing investments ?)
• Not focused on clinical problem (therapeutic options ?)
THE PROGRAMME
• No formal process of priority setting
• Defined by request (Region, Health Trust, Industry)
• Informal link with decision making