health technology assessment (HTA) Flashcards
how does WHO define health technology
a health technology is the application of organised knowledge and skills in the form of devices, medicines, vaccines, procedures and systems developed to solve a health problem and improve QoL
what are some ways to describe health technology
- physical nature
- drugs
- biologics (vaccines, blood products, CTGTP)
- devices, equipments, supplies (cardiac pacemaker, MRI scanner, diagnostic test kits)
- medical and surgical procedures (nutritional counselling, psychotherapy, acupuncture, coronary angiography, gallbladder removal, bariatric surgery)
- public health programmes (immunisation programme, smoking prevention)
- support systems (clinic laboratory, blood bank, electronic health record system, telemedicine system, drug formulary)
- organisational and managerial systems (medication adherence programmes, prospective payment, alternative healthcare delivery configurations) - healthcare purpose
- prevention
- screening
- diagnostic
- treatment
- rehabilitation
- palliation - stage of diffusion or maturity
- future (conceptual stage, anticipated or earliest stages)
- experimental (undergoing bench or animal lab testing)
- investigational (undergoing initial clinical evaluation)
- established (considered by clinicians to be a standard approach)
- obsolete, outmoded, abandoned (superseded by other technologies or demonstrated to be ineffective or harmful)
how does ACE define HTA
HTA is an established scientific research methodology to inform policy and clinical decision making on relatively new health technologies such as drugs, devices and medical services, compared to existing standard of care.
It is conducted using analytical frameworks drawing on clinical epidemiology and health economic information to determine how best to allocate healthcare resources.
what are the dimensions that can be considered in determining value
through examining the intended and unintended consequences of using a health technology compared with existing alternatives
depending on the perspective taken, dimensions to examine may include
(i) clinical effectiveness
(ii) safety
(iii) costs
(iv) economic implication
(v) social, ethical, cultural, legal, organisational, environmental
in which point of the product lifecycle could HTA be applied
HTA can be applied throughout product lifecycle
- innovation stage for horizon screening or early assessment
- regulatory approval decisions
- coverage and reimbursement
- healthcare provider adoption decisions
- assessing if ‘optimal’ use to determine need for disinvestment
what are the two main questions and how are they used to classify the health technology
- how well does the new technology work compared to the established practice?
- how much does this technology cost?
quadrant A (top left): higher cost, worse outcome = DOMINATED
quadrant B (top right): higher cost, improved outcome = ??
quadrant C (bottom left): lower cost, worse outcome = ??
quadrant D (bottom right): lower cost, improved outcome = DOMINANT
how is HTA done and what are the key characteristics to have
no standard approach but accepted characteristics are
- clear formulation of policy qn
- explicit methodology
- wide scope on the technology
- transparency of process and reporting
- globalise the evidence
- clinical assessment of the safety and effectiveness through systematic literature review
i) comprehensive searches for research to answer policy qn
ii) pre-specified rules for inclusion and exclusion of studies
iii) critical appraisal of design and conduct of studies
iv) data extraction
v) synthesis of result - localise the decision
- selection of relevant comparator using PICO
- cost effectiveness in local context or health system
- ethical issues
- access issues
- consumer preferences
- workforce planning
- training or credentialing users of technology
what is PICO
P atient/population, condition of interest
I ntervention (technology under evaluation)
C omparator (alternative treatment options to the intervention used in routine clinical practice
O utcome (clinically meaningful outcomes of interest)
what are some basic HTA orientations
- technology-oriented assessment (intended to undermine characteristics or impacts of a particular technology)
- problem-oriented assessment (focus on solution or strategies for managing a particular disease or other problem for which other alternative or complementary technologies might be used)
- project-oriented assessment (focus on a local placement or use of a technology in a particular institution, programme or other designated projects)
what are the various areas to be assessed using HTA
- technical properties
- performance characteristics and conformity with specifications for design, composition, manufacturing, tolerances, reliability, ease of use, maintenance etc - safety
- a measure of probability of an adverse outcome and its severity - efficacy and/or effectiveness
- economic attributes or impacts
- cost effectiveness, cost utility, cost benefit - social, ethical, legal and/or political impacts
what are some reasons for HTA
- need a tool to guide decision making in healthcare, HTA bridges the research evidence-policy making gap
- increasing demand within limited resoures
- healthcare costs are increasing rapidly at an unsustainable pace
- many new drugs entering the market have no added benefit
- regulatory approval ≠ clinically proven in HTA (using surrogate outcomes to provide indirect measurements of clinical effects where direct measurements are not feasible or practical (eg. cancer drugs) vs patient-relevant or clinically relevant outcomes are still preferred endpoints in clinical trials)
what is the ECHO model
a conceptual model relating
1. clinical outcomes (medical events that occur as a a result of disease or treatment)
- efficacy (response rate, duration of response, disease-free survival, overall survival)
- safety (toxicity and s/e, tolerability)
- humanistic outcomes (functional status or QoL)
- physical, social, general health and wellbeing, life satisfaction - economic outcomes (total costs of medical care associated with health technology and treatment alternatives balanced against clinical or humanistic outcomes)
- direct, indirect and intangible costs
others components of the ECHO model
- clinical indicators (used to infer degree of disease)
- treatment modifiers (factors that alter outcome associated with treatment alternatives)
- external controls (non clinical factors that affect availability or use of treatment alternatives)
- costs (medical, non-medical and indirect costs)
- humanistic intermediaries (effects of disease or treatment on humanistic outcomes)
what are some types of cost and who pays
- direct medical costs (medications, supplies, lab tests, HCP time, hospitalisation)
- patient y/n
- payer y
- hospital y
- society y - direct non-medical costs (transportation, food, family care, home aids)
- patient y
- payer n
- hospital n
- society y - indirect costs (lost wages due to morbidity, lost income due to mortality)
- patient y/n
- payer n
- hospital n
- society y - intangible costs (pain, suffering, inconvenience, grief)
- patient y
- payer n
- hospital n
- society y/n - opportunity costs (lost opportunity, revenue forgone)
- patient ?
- payer ?
- hospital ?
- society ?
how is singapore’s healthcare system financed
through public and private expenditure
- copayment to avoid overservicing and exceeding budget
- public healthcare financed through a system of subsidies by government direct funding through block grants and case-mix funding
what are block grants vs case-mix funding
case-mix funding pays hospitals a set amount for each inpatient episode and day surgery cases according to the DRG assigned
diagnostic related group (DRG) is a system used to group patients with similar clinical characteristics and resource utilisation patterns
what are current drug subsidies and schemes in singapore
- standard drug list
- MAF/MAF+
- Medisave, Medishield Life, Medifund
- CHAS
- acute inpatient care at PHI
- implants
- vaccination and childhood developmental screening
- specialist outpatient clinic care at PHI
- careshield life
- day surgery and day operation at PHI
- eldershield
- community hospital sub acute and rehabilitative srevices
- residential LTC services
- non-residential LTC services
- enhanced screen for life
- community dialysis services
- interim disability assistance programme for the elderly (IDAPE)
- foreign domestic worker (FDW) levy concession
- senior’s mobility and enabling fund (SMF)
- caregivers training grant (CTG)
- elderfund
- home caregiving grant (HCG)
- pioneer generation package
- merdeka generation package
what is the subsidy scheme for SDL and MAF/MAF+
[SDL]
all PRs get 25%
if SC, look at PCHI
PCHI = 0: AV≤21k gets 75%, AV >21k gets 50%,
0<PCHI≤2000: 75%
PCHI>2000: 50%
[MAF/MAF+]
all PRs get 20% unless PCHI >6500 then get 0%
if SC, look at PCHI
0<PCHI≤2000: 75%
2000<PCHI≤3300: 50%
3300<PCHI≤6500: 40%
PCHI>6500: 0%
what are the fundamental principles regarding cost
due to limited resources, need to avoid harsh rationing and ultimately refusal of care thus need to allocate scarce healthcare resources wisely, fairly and efficiently
- categorise patients into similar population characteristics
i) not possible for every patient to get maximum effectiveness
ii) can monitor and adjust accordingly after but initial or empiric treatment based on standardised care as part of cost minimisation technique
what is the purpose of having a drug formulary
- gives clarity and reliability in terms of supply (usually ~3-6m worth of stocks in case of disruption)
- manage storage space and inventory
- helps in clinical decision making and limits choices to what has been proven to be clinically and cost effective (reflects the quality and standard of care)
what does the formulary comprise of vs non-formulary drugs
- STD (approved for use based on safety efficacy and cost effectiveness)
- NSTD (may have restrictions or limitations on use compared to STD, for specific populations, conditions or treatment protocol)
- PAP (available through special programmes to assist those with difficulty affording them)
- exemption
non-formulary drugs are retail, sample (for trial or promotional purposes), clinical trial being investigated, pending approval), ad-hoc named patient (requires special approval for a use by a specific patient), exemption
what considerations does MOH have for new medications
- efficacy, comparative efficacy
- safety, comparative safety
- quality
- cost
what is the role and goal of the hospital P&T committee
pharmacy and therapeutics (P&T) committee’s goal is to ensure patients are provided with the best possible cost-effective and quality of care through determining what medicines will be available, at what cost and how they will be used
responsible for developing, managing, updating and administering the drug formulary within the organisation or institution
what are the principles of a sound drug formuary
- decisions are based on scientific and economic considerations that achieve appropriate, safe and cost effective drug therapy
- encompasses drug selection, drug utilisation review and other tools to foster best practices in prescribing, dispensing, administering and monitoring of outcomes
- P&T committee or equivalent body comprises of actively practicing physicians, pharmacists and other HCPs (multi professional)
- formulary system must have its own policies or adhere to other organisational policies that addresses conflict of interest or disclosure by P&T committee members
- should include educational programmes for payers, practitioners and payers concerning their roles and responsibilities (coverage decisions, drug selection etc)
- well-defined process if medically indicated to use a non-formulary drug by a physician or other prescriber
what is a drug formulary and outline the process that goes into forming the drug formulary
- product of P&T committee upon review and evaluation processes to guide physician prescribing through the inclusion of only selected products from various drug classes
- drugs chosen for the formulary are included based upon an evaluation of published literature
- a pricing contract is then negotiated with product manufacturer and is factored into the decision
what is the hierarchy of evidence from weakest to strongest
case reports, opinion papers, letters
–> animal trials, in vitro studies
–> cross sectional studies (observe at a single point in time the prevalence of exposure and outcome, causality CANNOT be determined)
–> case control studies (compare those with outcomes and those w/o outcomes to assess potential risk factors or exposures)
–> cohort studies (follow a group of individuals for a time period to assess association between exposure to a risk factor or intervention and the development of a specific outocme)
–> RCT
–> meta analyses and systematic review
what are the concerns regarding availability of relevant RCTs
- comparator in trial are not always the comparator in local practice
- placebo-controlled trials for regulatory approval of new drugs
- time lag from design of RCT to published result to conduct of HTA = comparator in trial may no longer be relevant
- near parallel development of new drugs by different companies = RCTs will be compared to SOC
- single arm studies are increasingly common in selected disease areas like cancer, rare disease
- even when active comparisons are made, evidence is often limited eg. lack of long term results or outcomes assessed
how to overcome lack of availability of RCTs
through indirect comparisons
indirect treatment comparisons (ITCs) uses relative effect of the treatments vs a common comparator to assess the H2H comparison of interest
- trial quality should be subjected to usual assessments eg. Cochrane ROB
- transitivity assumption: assumes no systematic differences between available comparisons (eg. control groups are sufficiently similar)
what are other types of indirect comparisons if there is a lack of available RCTs
- network meta analysis (NMA) = simultaneously compare multiple interventions
- matching adjusted indirect comparisons (MAIC) = propensity score matching by matching population of a single arm study to another trial to compare results
why is there a need to assess clinical significance
what may be a statistically significant difference does not mean that it is a clinically important difference
what is the MCID
minimally clinically important difference (MCID) is defined as the smallest difference in score in the domain of interest which patients perceive as beneficial and would mandate, in the absence of troublesome s/e and excessive cost, a change in the patient’s management
what is the usual hypothesis testing hypotheses
test for
(i) non-inferiority
f/b (ii) superiority
what are the considerations for clinical claims regarding non-inferiority and superiority
- timepoints (single or multiple reported?)
- outcomes assessed (same outcomes, multiple outcomes?)
what is pharmacoeconomics
pharmacoeconomics is a tool designed to provide users and decision makers with information about cost-effectiveness of different pharmacotherapies
what are the micro to macro applications of pharmacoeconomics
from micro to macro
- clinical decision
- justify clinical service
- drug use guideline
- formulary management
- resource allocation
