Revolutionising the Lifecycle of Pharmaceuticals Eyeforpharma
RWD conference June 5, 2013 1 Slide 2 Topics Introduction to CASMI
Current lifecycle and its issues Forces shaping the future Adaptive
licensing and its consequences Real world data and its role
Speculations about where we are headed Lessons for today 2 Slide 3
Topics Introduction to CASMI Current lifecycle and its issues
Forces shaping the future Adaptive licensing and its consequences
Real world data and its role Speculations about where we are headed
Lessons for today 3 Slide 4 4 = Centre for the Advancement of
Sustainable Medical Innovation = www.casmi.org.uk Slide 5 The
Innovation Gap time progress Bioscience understanding human biology
and disease = potential patient benefit Actual patient benefit The
Innovation Gap Slide 6 The danger of doing nothing progress
Bioscience understanding human biology and disease = potential
patient benefit Actual patient benefit time - First ever industry
reduction in R&D spend - $4bn drop in overall US spending in
2011 Slide 7 Whats distinctive about CASMI? Centre for the
Advancement of Sustainable Medical Innovation Providing independent
thought leadership, convening power - and so acting as a catalyst
for change Embedded in UCL and Oxford, but networked with other
European and international centres Interdisciplinary Medical,
Bioscience, Law, Ethics, Business, Economics, Statistics etc
Convening all stakeholders: academia, industry, patient groups,
regulators, policymakers, investors Slide 8 A Global Solution
requires a Global Network EFPIA IMI Other European centres Slide 9
Basic bioscience Translation to clinical candidates Innovations in
clinical trials Regulatory & reimbursement approval Approved
products Uptake by health systems Innovations used by patients
Patient Benefit 9 Gap 2: Failure to gain approval after costly
development Gap 3: Failure to achieve widespread use and patient
adherence to treatment Three core gaps in translation persist
across the value chain Gap 1: Failure to turn early stage research
into potential products Slide 10 Topics Introduction to CASMI
Current lifecycle and its issues Forces shaping the future Adaptive
licensing and its consequences Speculations about where we are
headed 10 Slide 11 Current development path IIIIIIaReviewHTA IIIbIV
PoCPh III entry Regul. Subm. & approval Launch Key
characteristics of current model Inflexible processes and method
Expensive and increasing data demands Lack of early alignment
between key parties: Segmented input & decision making Access
needs not designed in Patient perspective not fully addressed
FIMP&R Access PV & RM External activities Sponsor
activities Slide 12 Topics Introduction to CASMI Current lifecycle
and its issues Forces shaping the future Adaptive licensing and its
consequences Real world data and its role Speculations about where
we are headed Lessons for today 12 Slide 13 Forces shaping the
future of biopharma Future of Biopharma Emergence of regulatory
science and new technology Stratified medicine -> molecular
definition of disease More flexible regulatory processes for better
targeted drugs Pressure to make drugs more affordable Demand for
real world (comparative) effectiveness evidence Emergence of tools
to collect clinical and patient-reported outcomes Aspiration to
collect and analyse Big Data 13 Slide 14 Topics Introduction to
CASMI Current lifecycle and its issues Forces shaping the future
Adaptive licensing and its consequences Real world data and its
role Speculations about where we are headed Lessons for today 14
Slide 15 Potential New Flexible Blueprint Regulatory Review Access
Reviews Patient Use PhV & B/R Preclinical Phase IPhase 2Phase 3
Phase IV Reference: Athenaeum Group Exploratory R&DConfirmatory
trials Basic division between exploratory and confirmatory trials,
rather than Phases I-IV Slide 16 Potential New Flexible Blueprint
Regulatory Review Exploratory R&DConfirmatory trials
Collaborative design step before the most expensive confirmatory
trials are commissioned Review & design Access Reviews Patient
Use PhV & B/R Slide 17 Potential New Flexible Blueprint
Exploratory R&DConfirmatory trials Ability/need to customise
the model for different benefit/risk/uncertainty profiles Review
& design Access Reviews Patient Use Regulatory Review PhV &
B/R Submit & Confirm approval Initial access Slide 18 Potential
New Flexible Blueprint Exploratory R&DConfirmatory trials
Review & design Access Reviews Patient Use PhV & B/R
Ability to allow early, controlled patient access, if justified by
interim findings in confirmatory trials Submit & Confirm
approval Initial access Early access on condition of data
collection Slide 19 Potential New Flexible Blueprint Exploratory
R&DConfirmatory trials Review & design PhV & B/R Submit
& Confirm approval Initial access Early access on condition of
data collection Subject to requirements for pharmacovigilance and
pharmaceconomic analyses before full green light for wide access
and longer term reimbursement policy Effectiveness/comparative
studies Slide 20 20 Slide 21 21 Slide 22 Key elements of the
concept Adaptive Licensing = MAPPs* Conditional Approval (or
approval on conditions) Managed Market Entry Real world
effectiveness tracking Co-design of confirmatory trials
Stakeholders: -Sponsor -Regulator -HTA -Patient org EMA, based on
rapporteur evaluation of submission Patients treated; sponsor
reimbursed Outcome, safety data collection via reliable network *
MAPPs = Medicines Adaptive Pathways for Patients Slide 23 Slide 24
Do you have a potential pilot? - draft pilot selection criteria 1.A
robust case for accelerated process including unmet clinical need.
2.Strong efficacy signal from initial trials; positive benefit/risk
profile likely 3.Sufficient numbers of patients that can be
recruited. 4.Appropriate data collection mechanisms: robust trial
networks in the specific disease area with clinicians that are
interested in participating in research 5.Existing patient support
mechanisms 6.Could be either a new NCE/NBE or one approved in
another indication Slide 25 Topics Introduction to CASMI Current
lifecycle and its issues Forces shaping the future Adaptive
licensing and its consequences Real world data and its role
Speculations about where we are headed Lessons for today 25 Slide
26 RCTs May be unnecessary, inappropriate, inadequate, or
impractical Efficacy versus effectiveness Population: may not be
available for sub-populations and vulnerable populations
Interventions: may not be able to assign high-risk interventions
randomly Comparators: may not represent standard care Outcomes: may
report intermediate outcomes rather than main health outcomes of
interest Timing: may be too short in duration Setting: may not
represent typical practice Expense and bureaucracy Slide 27
Hierarchies of evidence should be replaced by acceptingindeed
embracinga diversity of approaches. This is not a plea to abandon
RCTs and replace them with observational studies. Nor is it a claim
that the bayesian approaches to the design and analysis of
experimental and non-experimental data should supplant all other
statistical methods. Rather, it is a plea to investigators to
continue to develop and improve their methods; to decision makers
to avoid adopting entrenched positions about the nature of
evidence; and for both to accept that the interpretation of
evidence requires judgment. Slide 28 Efficacy v effectiveness
Efficacy patient benefit and harm in experimental and closely
monitored research studies, normally RCTs. major advantages in
minimising bias generalisability questionable restricted entry
criteria unrepresentative settings Effectiveness patient benefit
and harm when the technology is actually applied in everyday
practice. pragmatic clinical trials adverse event reporting
clinical audit Slide 29 Slide 30 30 Groundbreaking RWD Study
Launched in Salford The Salford Lung Study is a unique
collaboration between GSK, North West e-Health (NWeH), The
University of Manchester, Salford Royal NHS Foundation Trust, NHS
Salford, local GPs and local community pharmacists. Around 4,000
patients with COPD and 5,000 patients with asthma from Salford,
Greater Manchester, will be enrolled in the year- long study. It is
the first time a large, real-world study has been performed on a
pre-licence medicine, across a large population within one
geographical setting. It will utilise Salfords e-Health records
infrastructure a clinical information system providing a single,
integrated electronic patient record across both primary and
secondary care. Slide 31 Topics Introduction to CASMI Current
lifecycle and its issues Forces shaping the future Adaptive
licensing and its consequences Real world data and its role
Speculations about where we are headed Lessons for today 31 Slide
32 Consequences of these developments .? Industry is in the data
business, not just the product business Big Data insights on
routine use of drugs Real world data to confirm/refine B/R and
value Adaptive approaches to pre-approval trials 32 Increasing
pressure on budgets and prices CPRD and related e-health develop-
ments Increasing focus on outcomes Sharper definition of patient
populations Slide 33 Topics Introduction to CASMI Current lifecycle
and its issues Forces shaping the future Adaptive licensing and its
consequences Real world data and its role Speculations about where
we are headed Lessons for today 33 Slide 34 Topics Introduction to
CASMI Current lifecycle and its issues Forces shaping the future
Adaptive licensing and its consequences Real world data and its
role Speculations about where we are headed Lessons for today 34
Slide 35 Lessons for today Ensure youre well networked into
industry bodies (like ABPI) Have a medical information lead on
board in your company Ensure cross-functional dialogue Consider
AL/MAPPs for early stage projects (Phase 1 onwards) Monitor RWD
developments (like GSKs Salford project and IMIs GetReal) and their
findings Find one or more centres (HERCs?) focused on relevant
therapeutic areas Consider creating a collaborative project to
monitor and evaluate your products outcomes 35
