Revolutionising the Lifecycle of Pharmaceuticals

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Revolutionising the Lifecycle of Pharmaceuticals

Eyeforpharma RWD conferenceJune 5, 2013

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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

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=Centre for the Advancement of Sustainable Medical Innovation

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www.casmi.org.uk

The Innovation Gap

time

progressBioscience – understanding human biology and disease= potential patient benefit

Actual patient benefit

The Innovation Gap

The danger of doing nothing

progressBioscience – 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

What’s 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

A Global Solution requires a Global Network

EFPIAIMIOther European centres

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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

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Topics

Introduction to CASMI Current lifecycle and its issues Forces shaping the future Adaptive licensing and its consequences Speculations about where we are headed

Current development path

I II IIIa Review HTA

IIIb IV

PoC Ph 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

FIM P&R

Access

PV & RM

External activities

Sponsor activities

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Forces shaping the future of biopharma

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Potential New Flexible Blueprint

Regulatory Review Access Reviews

Patient Use

PhV & B/RPreclinical Phase I Phase 2 Phase 3

Phase IV

Reference: Athenaeum Group

Exploratory R&D Confirmatory trials

Basic division between exploratory and confirmatory trials, rather than Phases I-IV


Regulatory ReviewExploratory R&D Confirmatory trials

Collaborative design step before the most expensive confirmatory trials are

commissioned

Review & design

Access Reviews

Patient Use

PhV & B/R


Exploratory R&D Confirmatory trials

Ability/need to customise the model for different benefit/risk/uncertainty profiles

Review & design

Access Reviews

Patient Use

Regulatory Review

PhV & B/RSubmit &Confirm approval

Initial access


Exploratory R&D Confirmatory trialsReview & 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 oncondition of

data collection


Exploratory R&D Confirmatory trialsReview & design

PhV & B/RSubmit &Confirm approval

Initial access

Early access oncondition 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

Key elements of the concept

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

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 mechanisms6. Could be either a new NCE/NBE or one approved in another

indication

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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

Hierarchies of evidence should be replaced by accepting—indeed embracing—a 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.

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

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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 Salford’s e-Health records infrastructure – a clinical information system providing a single, integrated electronic patient record across both primary and secondary care.

http://www.srft.nhs.uk/EasysiteWeb/getresource.axd?AssetID=13809&type=full&servicetype=inline













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Consequences of these developments ….?

Increasing pressure on budgets and prices

CPRD and related e-health developments

Increasing focus on outcomes

Sharper definition of patient populations

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Lessons for today

Ensure you’re 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 GSK’s Salford project and

IMI’s 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 product’s outcomes

Revolutionising the Lifecycle of Pharmaceuticals

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