proposed pharmacoeconomics guideline to saudi food & drug ...€¦ · proposed...

Proposed Pharmacoeconomics GuidelineProposed Pharmacoeconomics Guidelineppto Saudi Food & Drug Authority (FSDA) to Saudi Food & Drug Authority (FSDA)

Dr. Mohammed S. Al-SultanDr. Sinaa Al-Ageel

Pharmacoeconomics Research Unit Department of Clinical PharmacyDepartment of Clinical Pharmacy

King Saud University College of Pharmacy

SFDA Pricing Workshop (May 20-21, 2008)

Presentation Outline Presentation Outline ese tat o Out eese tat o Out e

Pharmacoeconomics guidelines: international experience

To present the proposed SFDA pharmacoeconomics guidelines

Situations where economic evaluations can Situations where economic evaluations can assist decision makers:assist decision makers:

Decisions by various levels of government or administrative bodies (e.g., different health care sectors, hospitals, health insurance companies) to fund a program service or technologycompanies) to fund a program, service or technology.

Pricing decisions by government regulators and technology manufacturers.

Clinical practice guidelines.

Post-marketing surveillance and updates of economic information based on the use of the technology in the “real world” (which can then be used to inform one of the other types of decisions).

Regulate cost effectiveness claims by pharmaceutical Companies

Pharmacoeconomics guidelines: International experiencesexperiences

International ExperienceInternational Experiencee o pe e cee o pe e ce

Several jurisdictions have imposed a ‘Fourth Hurdle’ or requirement for economic data as partHurdle’ or requirement for economic data as part of pricing/reimbursement decisions for drugs.

The new requirements are usually accompanied q y pby a set of guidelines for company submissions.

International ExperienceInternational ExperienceInternational ExperienceInternational Experience

In 1992, Australia became the first country to formalise and issue mandatory guidelines for health economics evaluations of pharmaceutical products as a grequirement prior to reimbursement

Currently more than 32 guidelines around the world

Most experience relates to pharmaceuticals, although some countries have central programmes for evaluating (e.g. The Netherlands, UK).

The scope of requirements differs from place to place:The scope of requirements differs from place to place:

All new drugs (Australia, Canada, Scotland), Drugs where a premium price is requested (the Netherlands )D ith j i t (E l d d W l P t l )Drugs with a major impact (England and Wales, Portugal )

International ExperienceInternational Experience

Purpose

Reimbursement/ Listing Methodological Standardsg

g

Government/Payers

Belgium Canada (2 provinces)

LDI Task Force (USA) Rovira et al(Spain)

FinlandHungary Ireland The Netherlands

Hannover (Germany)BESPE (Belgium) BMJ Working Party (UK) Garattini et al(Italy)e

The NetherlandsNew Zealand Norway Portugal

Garattini et al(Italy)College of Economists (France) So

urce

PortugalSweden United Kingdom

Academia Langley et al (USA) PHrMA (USA)Academia

Langley et al (USA)Alban et al (DK)

PHrMA (USA)

Industry PHrMA (USA)Industry

PHrMA (USA)

I t ti l E iI t ti l E iInternational ExperienceInternational Experience

Hjelmgren et al report agreement on 75% of methodological i i lprinciples

The main differences are in guidance on:The main differences are in guidance on:– study perspective (e.g. inclusion of non-healthcare

costs)– economic outcomes (e.g. QALYs, WTP)– handling uncertainty

Value in Health 2001,4:225-250

h d id li diffh d id li diffWhy do guidelines differ?Why do guidelines differ?

Some important differences in national perspective (e gSome important differences in national perspective (e.g. views on equity in healthcare)

Methods develop over timeMethods develop over time

Guidelines reflect the views of those who develop them, which may or may not reflect the wider consensus

Some guidelines may contain methodological errorsSome guidelines may contain methodological errors

Proposed SFDA Pharmacoeconomics Guidelines

Primary AudiencePrimary Audience

Saudi decision and policy makers who are responsible for:

registering, pricing, funding decision making regardingfunding decision making regarding health technologies.

Secondary AudienceSecondary Audience

A secondary audience for evaluations yincludes:

– Academics, – Health care providers, – Manufacturers.

Standard Reporting FormatStandard Reporting Format

A structured reporting format for the preparation of reports of economic valuations ensures that studies are thoroughly presented, and organizedstudies are thoroughly presented, and organized consistently to facilitate review and comparison by decision makers.

The standard reporting format will be available to download from the SFDA web sitedownload from the SFDA web site (http://www.sfda.gov.sa) once the guidelines get final approval.

Standard Reporting FormatStandard Reporting FormatPreface Executive summary yTable of ContentsAbbreviationsGlGlossary1. Objectives 2 Background2. Background 3. Literature Review4. Methods 5. Results 6. Discussion R fReferences Appendices

PrefacePrefaceList of authors, affiliations, and a description of contributionsdescription of contributions.

AcknowledgementsAcknowledgements.

Disclosure of funding and reportingDisclosure of funding and reporting relationships, study sponsor, contractual arrangements, autonomy of analysts, and

bli ti i ht d l ti f fli tpublication rights; declaration of conflicts of interest.

Executive SummaryExecutive SummaryThe Executive Summary should be one to two pages long and written in non-technical language.

Issue: a statement about the economic issue or reason for evaluating the technology

Obj tiObjectives

Methods

Results: a numerical and narrative summary of the findings

Discussion: study limitations, relevance of findings, health services impactimpact

Conclusions: state the bottom-line findings of the evaluation, uncertainty about the results, and caveats

1.1. OBJECTIVESOBJECTIVES

Description of issue(s) addressed in theDescription of issue(s) addressed in the report

Statement of study question

Define the study question, state it in an answerable form, make it relevant for the target audience.

Define the patients and population(s), intervention, and comparators.

Objectives of the studyObjectives of the study

22.. BACKGROUNDBACKGROUNDGeneral comments on condition

List the etiology pathology diagnosis risk factors prognosis (if

Technology description

List the etiology, pathology, diagnosis, risk factors, prognosis (if relevant).Describe the epidemiology (i.e., incidence or prevalence), burden

f th diti i S di A biF d b d d i d f f

Regulatory status

of the condition in Saudi Arabia.Describe the economic impact and burden of the condition in Saudi Arabia

For drugs, state brand and generic names, dosage form, route of administration, recommended dosage, duration of treatment, therapeutic classification, mechanism of action.

Describe the current clinical practice in Saudi Arabia. Refer to clinical practice guidelines (if relevant). Include a description or comparison of alternatives for the indication.

For non-drug technologies, state basic features, underlying theory or concept.List advantages and disadvantages (e.g., relating to clinical use).

List the approved indication(s) in Saudi Arabis that is the topic of the study, including applicable population and subgroups, and date of approval.p g g ( g , g )State adverse events, contraindications, cautions, warnings.Describe setting for the technology if relevant (e.g., hospital-based)

subgroups, and date of approval.Give additional approved indication(s) in Canada.Include the regulatory status and approved indications in other countriesbased)Give unit cost of the intervention and comparators.other countries

33.. LITERATURE REVIEWLITERATURE REVIEW

Discuss existing economic studies that address the same technology and similar studythe same technology, and similar study question(s).

Comment on the relevance and generalizability of g ythe results of the reviewed studies to the target audience.

44.. METHODSMETHODS4.1 Perspective

– State and justify the perspective(s) used in the analysis (e.g., MoH, insurance company or society)insurance company, or society).

4.2 Target population– Describe target population(s) and the care setting for the intervention g p p ( ) g

or expected use.

4.3 ComparatorsDescribe and justify selected comparators; relate choice of– Describe and justify selected comparators; relate choice of comparators to the study population, and the local context or practice.

4.4 Types of economic evaluationy– The type of economic evaluation conducted (e.g. CUA, CEA, CMA, or

CBA ) should be clearly stated and justified with respect to the study question

44.. METHODS METHODS cont.cont.

4.5 Effectiveness

– Indicate sources of information (e.g., trials, a meta-analysis of individual trials, literature, expert opinion).Give details about the quality of evidence on efficacy– Give details about the quality of evidence on efficacy and effectiveness used in the analysis.

– Identify factors that are likely to have an impact on effectiveness (e g adherence diagnostic accuracy)effectiveness (e.g., adherence, diagnostic accuracy), and describe how these were factored into the analysis.

– When short-term outcomes are extrapolated to final t l i l l ti hi d t h ioutcomes, explain casual relationships and techniques

used for the extrapolation. – Describe the strength of the evidence for the

l ti hi d li krelationships and links.

44.. METHODS METHODS cont.cont.

4.6 Outcome measurement and valuation

– Identify, measure, and value all relevant outcomes, including important adverse events, for each alternative.

– Give the sources of information and data, assumptions, and justificationand justification

– Give the HRQL measurement approach used, with the justification (a copy of the instrument may be included in an appendix). Describe the methods of eliciting preferences

– and the population measured– and the population measured. – • Include other outcomes that were considered but

rejected (with rationale).

44.. METHODS METHODS cont.cont.

4.7 Resource use measurement and valuation

– Identify, measure, and value all resources included in the analysis.Report the costing methods used (e g gross or micro– Report the costing methods used (e.g., gross or micro-costing).

– Report resource quantities and unit costs separately.f– Distinguish additional costs from averted costs.

– Report the method used for costing lost time, including productivity losses. Provide justification when time costs are not considered.

– Report all sources of information and data and assumptions.p

44.. METHODS METHODS cont.cont.

4.8 Modelling

– Economic evaluations typically involve building and then using models to synthesize evidence and assumptions from multiple sources to estimate the long-term incremental costs and outcomes of new therapiesterm incremental costs and outcomes of new therapies.

– Describe the model structure: description of the scope, structure, and assumptions made with justification; inclusion of a model diagram or decision tree isinclusion of a model diagram or decision tree is recommended.

– Describe how the model was validated. – List other data or assumptions with sources andList other data or assumptions with sources and

justification. This may include details about epidemiological factors, such as prevalence or incidence of the condition.

44.. METHODS METHODS cont.cont.

4.9 Variability and uncertainty

a) Handling variability

– State whether there are a priori identifiable subgroups for which differential results might be expected (e.g., based on effectiveness, preferences and utilities, costs).

– Describe how other types of variability (e.g., variation in costs or practice patterns) were analyzed, and provide theor practice patterns) were analyzed, and provide the justification.

b) Handling uncertainty) g y

– Identify sources of uncertainty in the analysis.– Describe methods used to analyze uncertainty .y y

44.. METHODS METHODS cont.cont.

4.10 Time horizon– Indicate the time horizon(s) used in the analysis, and its

justificationjustification.

4.11 Discount rateIndicate the discount rates used for costs and outcomes and– Indicate the discount rates used for costs and outcomes, and the justification.

4.12 Equityq y– State equity assumptions (e.g., a QALY is equal for all).– Identify equity-relevant characteristics of the main subgroups

that may benefit, or be adversely affected by, the technology, d d ib h th l dand describe how they were analyzed.

55. RESULTS. RESULTS

5.1 Main results

– Present all analyses in a step-by-step fashion, so the calculations can be replicated if desired.

– For both the technology and its comparator(s), the total costs and total benefits arising from their use should be presented

– Final results should be based on increments (i.e., differences ( ,between the intervention and alternatives) of expected costs and expected outcomes. The results for a CEA or CUA should be reported as ICERs.

– Provide tables of results in appendices; a visual display of results is encouraged.

55. RESULTS . RESULTS cont cont

5.2 Results of variability analysis

– Give the results for all subgroups analyzed.– Indicate the distribution impacts (i.e., benefits, harms,

costs) and ICER results for any subgroups that arecosts) and ICER results for any subgroups that are relevant for equity purposes.

5 3 Res lts of ncertaint anal sis5.3 Results of uncertainty analysis

– State the results of sensitivity analysis.y y– Identify the greatest sources of uncertainty (i.e., key

drivers).

66. DISCUSSION. DISCUSSION

6.1 Summary of results6.2 Study limitationsy6.3 Compare with other economic studies6 4 Generalizability6.4 Generalizability6.5 Equity considerations6.6 Health services impact

77. CONCLUSIONS. CONCLUSIONS

Add th h bj ti ( ) dAddress the research objective(s) and question(s).Summarize the bottom line findings of the studySummarize the bottom-line findings of the study, aggregate impact, uncertainty about the results, appropriate uses for the intervention (e.g.,appropriate uses for the intervention (e.g., population subgroups), and any caveats.

Thank YouThank You