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

FROM RESEARCH QUESTIONS TO RESEARCH NEEDS

PROSPECTIVE 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

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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

“INNOVATIVE” EVALUATION

Comparative Effectiveness Research

Prostate cancer – low risk

ROBOTIC ASSISTED

PROSTATECTOMY

RADICAL  TREATMENT

TOMOTHERAPYvs

GRAZIE