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Pre-conference workshop - Tuesday 9 December 2008
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| Instrument development and evaluation for Patient Reported Outcomes Assessment |
Objectives
Patient reported outcomes are widely used to evaluate the impact of health technologies, practice innovations or changes in health policy from the patients’ perspective. This course is designed to familiarise people with the range and scope of what PROs are used for; how they are developed and evaluated, and how PRO data can be used to support licensing and reimbursement applications.
The course content will describe the scope and range of what patient reported outcomes measure. This includes generic and disease specific measures of health related quality of life (HRQL), as well as measures of patient preference, utility, treatment satisfaction and measures of work productivity.
We will describe the steps that researchers generally go through in order to develop and test a new PRO. This will include qualitative work, item development and testing and then validation studies.
Finally in the last hour we will frame this in terms of what the FDA and EMEA expect to see when PROs form an important part of a licensing submission. In addition we will describe the approach of bodies such as NICE and how they review PRO data and use it to guide reimbursement decisions.
This is an entry level course which assumes only a passing familiarity with patient reported outcomes.
Course Leaders:
Scott Doyle, Outcomes Researcher, Oxford Outcomes Ltd
Diane Wild, MSc, Founding Director, Oxford Outcomes Ltd
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9.30am | Registration & coffee
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10am | Introductions
• What is health-related quality of life (HRQL)?
• Why are HRQL/ patient reported outcomes (PROs) useful?
• How is PRO data gathered?
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11.15am | Break out task & coffee
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11.45am | Developing PRO measures
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12.15pm | Break out task 2
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12.45pm | Lunch
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1.45pm | Measurement challenges in PRO research
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2.30pm | Regulatory issues
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3pm | Q&A and coffee
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3.30pm | End of workshop
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Day One - Wednesday 10 December 2008
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8.30am | Registration & coffee
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9am | Opening remarks from the chair
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| STRATEGIC BACKDROP |
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9.15am | Towards International standards for assessing cost-effectiveness
• How diverse are current international requirements for economic evaluation?
• What are the key areas of controversy in methods?
• What could be done to harmonise requirements?
• What are the implications for those conducting and using studies?
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9.45am | The use of outcomes research in healthcare policy making
• HO Research can inform the variety of health policy decisions from priority setting to insurance design and coverage decisions
• To ensure objectivity and fairness, HO research should be separate from the process of decision making although it should be designed in response to the questions and priorities of decision makers
• There is a need for HO research to be done on the outcomes of health policy decisions
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| | Dr Marc Berger, Vice President, Global Health Outcomes, Eli Lilly & Co
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10.15am | Morning tea
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| REVIEW OF HTA IN EUROPE |
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10.45am | HTA in the UK
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11.15am | Early Health Technology Assessment of new drugs in Scotland
• Scotland has adopted a rapid review process to assess clinical- and cost-effectiveness of all new drugs coming to the clinical arena
• The review, which take only 16 weeks, involves stakeholders from the NHS, patients and the pharmaceutical industry
• The process aims to shape and form clinical practice as it evolves, rather than changing existing practice patterns
• The process has to be able to undertake 80-100 assessments per year, but experience shows this to be possible within reasonable resource constraints.
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11.45am | HTA in France: centralisation and separation of powers
• The stakeholders in a Bismarckian-centralised system
• HTA and maximisation strategies
• The new challenges introduced by the 2008 Social Security finance law (economic evaluation)
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12.15pm | HTA in Spain
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12.45pm | Lunch
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1.45pm | The role of the cost-effectiveness criterium in the Netherlands
• Overview of the reimbursement system - prescription drugs and hospital based drugs
• The assessment of the cost-effectiveness and the appraisal; how do we value cost/QALY?
• Conditional reimbursement of hospital based drugs: need for additional data so-called outcomes research;
• Guidance document for outcomes research and practical experiences
Gepke Delwel, Senior Policy Advisor, CVZ, The Netherlands
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2.15pm | Speed networking
Fun, high speed networking
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3pm | HTA in Germany
• German health system
• Target group of HTA in Germany
• HTA applicants and agents
• Role of DIMDI and other German HTA institutions
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3.30pm | Afternoon tea
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4pm | HTA in Sweden: what problems do we encounter?
• Therapeutic choice: how can we provide room for it?
• Uncertainty: how should we handle it?
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4.30pm | HTA in Denmark
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5pm | Panel discussion: comparing and contrasting international guidelines
Why are there differences and are they justified?
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5.30pm | Closing remarks from the chair
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5.45pm | Drinks reception
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6.30pm | End of day one
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Day Two - Thursday 11 December 2008
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8.45am | Registration and coffee
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9.15am | Opening remarks from the chair
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| REVIEW OF HTA IN AMERICAS |
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9.30am | HTA in the US
• Perspective and process
• Assessment of new drugs
• Cost effectiveness and affordability
• Bridging the evidence gap
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| | Dr Naomi Aronson, Executive Director, Technology Evaluation Centre, BlueCross Blueshield
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10am | HTA in Canada
• Healthcare in Canada
• Health technology trends
• Impact on health decision makers
• CADTH’s role and programmes
• Thoughts about the future
• Conclusions
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| | Dr Jill M Sanders, President & CEO, Canadian Agency for Drugs and Technologies in Health (CADTH)
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10.30am | Industry perspective: how do we develop drugs that are valued by payers, providers and patients?
• What are the key pressures facing providers of healthcare?
• How is the industry responding to these challenges?
• How should the industry payers and patients be working together?
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11am | Morning tea
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11.30am | Panel discussion: developing better communication between industry and agencies to enable faster patient access to new medicinal products
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12.30pm | Lunch
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| BEST PRACTICE IN REPORTING AND UNDERTAKING HTA |
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1.30pm | Towards transparency in decision analytic models
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2pm | The Future of the QALY: experience from cancer treatments
• How well does the QALY pick up patient benefit
• How can QALYs be put in the context of societal preferences
• Does can QALY based HTA need to adapt?
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2.30pm | Cost effectiveness of pharmacotherapies across the regions: convergence or divergence?
• Introduction and brief summary of cost-effectiveness requirements across major markets (including US)
• Discussion related to timing of evaluations relative to phases of development, including a brief overview of types of cost-effectiveness evaluations
• Examples of when and in which circumstances different analyses apply and examples of successful cost- effectiveness evaluations
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| | Dr Josephine Sollano, Director, Global Therapeutic Head-GHO, Cardiovascular & Metabolics, GlaxoSmithKline
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3pm | Afternoon tea
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3.30pm | HTA submissions for orphan products and therapies for chronic diseases: case study examples
Drawing on anonymised example submissions a number of issues will be discussed:
• Generating health economic models when clinical data is limited
• Developing health economic arguments for chronic diseases with short term clinical data
• The importance of cost effectiveness arguments in the context of broader issues of indirect medical costs, societal costs and equity
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| | Dr Jim Furniss, Director, Market Access Solutions, Bridgehead International
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4pm | Case study: preparing an HTA submission for new medicinal product
• What information was gathered?
• What modeling techniques were used
• What feedback was given
• Lessons learnt
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4.30pm | Closing remarks from the chair
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4.45pm | End of conference
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