Download Pharmacoeconomics

Pharmacoeconomics: future ahead
Presented bySonu Bhardwaj
Sharma
Flow of presentation
• Changing healthcare environment
• Use of pharmacoeconomics and methodologies
• Transition from EBM to VBM concept
• Evolving drivers
• Increasing demand of PE studies
I. Healthcare technology assessment( HTA)
II. Comparative effectiveness
• Future of PE
I.
Risk sharing agreement
II. PE and regulatory appraisals
• PE Case Study : Lucentis
Changing healthcare environment
20th
century
Clinical
development
Marketing
authorization
Marketing & sales
Market access
21st
century
Clinical
development
Marketing
authorization
Marketing & sales
Criteria for market access are different from those for marketing authorization
Market access
Market
authorization
• Efficacy
• Safety
• Quality
Regulatory
authorities
Pricing and
reimbursement
Formulary listing/
coverage
• Unmet medical &
economic need
Relative effectiveness
• Costeffectiveness
• Budget Impact
(national level)
• Budget Impact
(local level)
• Rebates and
discounts
• Additional
services/studies
National Pricing &
Reimbursement
authorities
HTA organizations
Regional and hospital
Purchasers
USE OF PHARMACOECONOMIC RESEARCH
• Pharmaceutical reimbursement
• Selecting formulary products
• Price Negotiations
• Clinical Practice Development guidelines
• Communication to prescribing physicians
COMPARING PHARMACOECONOMIC METHODOLOGIES
METHODOLOGY
•
Cost-Minimization
•
Cost-Effectiveness
•
Cost-Benefit
•
Cost-Utility
•
Cost-Consequence
CONSEQUENCES
•
Quality-Adjusted Life Years (QALYs)
•
long-term cost–benefit assessments
•
treatment convenience and
compliance for patients
•
long-term risk-benefit analysis
Evolution from “EVIDENCE” based medicine to “VALUE” based
Clinical Evidence Focus
to
Clinical Value Focus
VBM (value based medicine)
PRE EBM
focus on novel MOA
Focus on effectiveness and
“value” to stakeholders
VBM
GUIDELINES
AND
COMPENDIAS
EBM
EBM (evidence based medicine)
Focus on efficacy only
TRIAL BASED RESEARCH
Value pyramid
The evolving drivers for assessing the “value” of a drug
Existing drivers
Evolving drivers
Features – what it is
Molecule
Mechanism of action
Features – what it is
Molecule
Mechanism of action
Benefits – what it does
Efficacy
Safety
Benefits – what it does
Efficacy
Safety
Value – why it matters
Cost effectiveness
Quality of life
Convenience
The increasing demand for comparative clinical effectiveness
and cost effectiveness (pharmacoeconomics)
NICE
Expansion of HTA
& evidence-based
reimbursement
Comparative
effectiveness
research
• Economic evaluation introduced into pricing & reimbursement
process in Ontario in 1991 and Australia in 1992.
• “Opportunity cost” concept as justification for “rationing”: have
to compare new technology to the next best alternative in terms
of benefits and costs: comparative clinical effectiveness and cost
effectiveness
•
•
•
Full consideration of clinical, economic, legal and ethical aspects
Applied in some shape or form in every major market ex-U.S.
Increasingly also applied at the regional and hospital level
• CMS has always provided coverage guidance based on clinical
effectiveness for devices and procedures
• Private payers are requesting clinical and economic data based
on the AMCP format for formulary submissions
Health Technology Assessments
Definition
HTA is a broad
concept
referring to
evaluation
of both clinical and
economic
performance
for many types of
medical technologies:
• surgeries
• diagnostics
• pharmaceuticals
Effects of HTA
dissemination
Affect corporate investment decisions
• Modify R&D priorities and spending
levels
• Change regulatory policy
• Modify marketing of a technology
• Change third-party payment policy
• Affect acquisition or adoption of a new
technology
• Change the rate of use of a new
technology
• Change clinician behaviour
• Change patient behaviour
• Reallocate national or regional health
care resources
• In UK, the National Institute for Clinical Excellence (NICE): uses HTAs to assess cost–benefit
of newly licensed agents and has traditionally fixed a bar of £30,000 incremental cost per QALY for
treatments to prove cost effectiveness
• Centres for Medicare and Medicaid Services (CMS), for evidence of value demonstration.
• In U.S, HTAs and comparative-effectiveness research (CER) used to compare cost to benefit comparisons
Recent example : Provenge. The assessment of Provenge by CMS marks a landmark event in US reimbursement
history.
Comparative effectiveness
Cost per quality-adjusted life-year (QALY) gained from selected clinical strategies
Drug comparison
cost per QALY gained
Switch to am aromatase inhibitor for
early-stage breast cancer vs. continued
tamoxifen
$22,900
Implant a cardioverter-defibrillator
(primary prevention)
vs. continued medical management
$37,400 to $77,200
Perform fusion surgery for degenerative
spondylolisthesis with spinal stenosis vs.
conservative management
$120,000
Prescribe trastuzumab for metastatic
breast cancer vs. standard chemotherapy
$150,000
Prescribe erlotinib for advanced
pancreatic cancer vs. gemcitabine
alone
$370,000 to $500,000
*Values are given in 2008 U.S. dollars, with adjustment for inflation according to the Consumer Price Index.
Numbers are the ratios of the added cost per person to the gain in QALYs per person
Risk-sharing agreements (RSAs) will evolve
What is an RSA?
Who is
utilizing RSA?
Why use a RSA?
RSAs typically serve to hedge a payer’s financial risk and to
ensure value where it might not be clear
• RSAs can be performance and/or financially based
• Risk-sharing contracts may be pushed for by patient-advocacy
groups where patient access might be limited
A number of European countries, including the UK, Germany
and France; Australia; Canada; and the US
• Specifically, RSAs are gaining momentum in the UK where
guidance decisions by NICE determine whether drugs will be
covered and reimbursed by the NHS
A shift in the clinical trials’ process to bring drugs to market
more quickly, the increasing cost of drugs, and a focus on
value demonstration is driving the popularity
• NICE delivered the preliminary assessment of a multi-technology appraisal of
treatments for metastatic renal cell carcinoma (RCC)
• Example: declaring that Sutent® (sunitinib), Avastin®
(bevacizumab) (+ interferon), Nexavar® (sorafenib), and Torisel® (temsirolimus) were not costeffective
Options and should corrected before making available on NHS .
PE studies and regulatory appraisals
• At present , bodies like NICE perform PE studies but occur after regulatory approvals,
• FDA and EMA continue to use EBM
• But in future, As VBM begins to permeate and shape clinical decision making, other factors
such as QoL, which are already computed with HTAs, might begin to influence regulatory
approvals.
• Capture data that can be used to support value propositions for products in clinical
development. like:
 Health-related Quality of Life
 Target Patient Subgroups
 Value from the Stakeholders’ Perspective
 Communicating Value Propositions
PE Case evaluation
• Dose modelling to support Lucentis market access in Europe, Canada and Australia
Situation
Outcome
Complication
• Lucentis (ranibizumab) licensed for patients with wet
age-related macular degeneration
• Pivotal trials used monthly dosing regimen
• Smaller scale trials and drug simulation indicated that
flexible dosing would achieve similar outcomes
Label specifies 3 initial monthly injections followed by flexible
OCT guided dosing scheme
• Economic assessment driven by annual treatment
cost
• Payers concerned about the annual cost of monthly
dosing
Modelling cost-effectiveness of Lucentis
o Efficacy data from registration trials
•Translating vision benefits into patient utilities & QALYs
o Resource use and cost data from drug modelling
• Costs driven by the number of vials used and the associated costs of injection
o Resulting cost-effectiveness found highly acceptable, subject to uncertainty about realworld dosing (and resulting efficacy)
o “Risk-sharing” offered by manufacturer
• can bear this risk given the additional information manufacturer has about product
characteristics
Drug Costs are high and on the rise
• U.S. costs roughly double those rest of world
Overall Rising Costs–
Due to 3 components:
• 1. Increased per capita drug use
• Some related to aging population
• But some pure increase in drug #s/use
• 2. Increased prices of individual drugs
• Rising significantly higher than inflation rate
• 3. Shift to newer more expensive drugs
Needs to monitor value of pharmaceuticals
• Increased cost.
• Increased number of alternatives
available to treat illness and
disease.
• Growing demand for
pharmaceuticals.
• Introduction of high cost
biotechnology products.
Indexes of Average U&C Prices for 50 Brand and 46 Generic Drugs
Frequently Used by BCBS FEP Enrollees, by Month, 2000 through 2004
PE studies are becoming a part of clinical studies
• Clinical trials of future oncology products need to consider overall survival and other
clinical endpoints deemed meaningful for a given cancer, safety endpoints, and measures
of generic and disease specific quality of life.
• These outcome “channels” should be prioritized based on indication characteristics such
as:
(For cancer patients)
• tumour type (stage/grade)
• epidemiology
• severity of disease (morbidity)
• age of onset
• unmet medical need
LUCENTIS CASE
LUCENTIS CASE
LUCENTIS CASE
LUCENTIS CASE