Real World Data Across the Product Lifecycle: RWE from pre-clinical to launch

SVMPharma Ltd,Landmark House

Station Road, Hook, Hampshire, UK,

RG27 9HA

CONTACT US enquiry@svmpharma.com

+44(0) 1256 962 220www.svmpharma.com

Patient level data access in the

NHS: Generating positive outcomes

through Real World Evidence

Real world evidence (RWE) is becomingcommonplace in support of HTA submissionsand resubmissions in order to demonstrateefficacy and value for new drugs. Cost andtime efficient evidence gathering across theproduct lifecycle has never been moreimportant. Real world evidence opportunitiesacross all phases of drug development areincreasing through pre and post approvalstudies, providing opportunities for savingsand efficiencies through strategic planning,data collection, and re-purposing of studymaterials generated pre-launch as well as post-launch.

We examine the RWE opportunities across thedevelopment lifecycle, and assess howregardless of the phase, real world data isgenerated and available to drive RWE insights.

RWE: more than a post-launch toolReal world evidence collection currentlyprimarily occurs at a level to indicate costeffectiveness and efficacy in real world patientpopulations. Being generated in cohorts ofclinical patients, this data is therefore primarilycaptured retrospectively, post-launch to supportHTA submissions. However Real world data isavailable from across the product lifecycle, andmay be used to generate RWE to provideinsights and answer questions that informfuture research and development prospects.

Because data types, sources and availability aremore variable, proper study design is especiallyimportant in RWE. The choice of study dependson why the evidence is wanted, for example, tostudy long-term safety, study effectiveness inmultiple care settings, conduct economicanalyses, investigate unmet needs andinterventional pathways. The research questionto be answered will dictate the appropriatestudy type and origin of RWD. [2]

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Real World Data across the

product lifecycle: Generating RWE

from pre-clinical to launch

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Evidence gathering across the product lifecycleSix areas throughout the product lifecycle thatgenerate revenue growth and cost orproductivity improvements:

Pre launch, RWE enables us to understandcurrent treatment and the factors influencingtheir effectiveness. In response to this,pharmaceutical companies can betterdemonstrate where their brand has advantagesover current management choices in areas suchas impact on disease burden, standard of care,budget impact, unmet needs improvedadherence, increased quality of life, reducedhealthcare resource use, and patientpreference.

Typically companies have focused RWE ondemonstrating value, market access andclinical development. The approach is shiftingtoward a systematic strategy across all aboveareas, bringing about a change in RWE value.By connecting different datasets, companiescan deepen their understanding of patientjourneys and market influences.

Clinical trialsHealth economics and outcomes research hasbecome a more integral part of traditionalresearch and development, impacting clinicaltrial design and the management of economicendpoints within these trials. With major globalshifts in healthcare services to control costs, agrowing number of Phase III trials now includeeconomic endpoints and analyses, alongsidethe clinical outcome measures, to demonstratethe cost-efficacy of new products.[1]

RWE is regarded as having the ability to improveclinical development via enabling translationalresearch, better understanding of drugpathways, higher value population and productprofiles, and trial simulation and recruitment.Suggestions include trial simulation using RWEto ensure that trials likely to fail are not initiated(over 30% of trials fail for strategic rather thanoperational reasons).

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Improving clinical development

Improves trial enrolment, reduction in strategic design flaws, and better product profile design.

Increasing productivity and cost savings

Reduces duplicate primary research spending and doubles impact factor of publications

Better initial pricing and market accessAccelerates market access submissions, payment by use/indication for more effective price negotiations, and conditional access via coverage with evidence

development

Improving launch planning and tracking

Accurate patient pool segmentation and rapid adjustment of messaging/resource allocation at launch

Demonstrating ongoing safety and valueSupports continued access and reimbursement

through responding to queries and publications, as well avoiding label changes and protecting brand

from generic entry

Optimising commercial spend effectivenessBrand growth via RWE-enabled marketing, improved promotion via physician-patient

segments, and better forecasting via disease progression models

Named patient programmesDespite only being available on limited access,it is a myth that data cannot be captured duringa named patient program. There is nolegislation prohibiting data capture, althoughsome territories have specific guidance overwhat data can be provided. In fact, mostmanaged access programs contain clinical datacapture in some form as standard. Data capturein these incidents are incidental and does notmean the program is intended for research;patient cohort treatment remains priority. In amanaged access program observational data isprovided at the physician’s and patient’sdiscretion meaning there is no requirement foradditional protocol or ethics review to collectdata than already required for a managedaccess program. The intended use of data willdictate the context in which the data should becollected and may affect the mechanism used.

Pharmaceutical companies are embeddingnamed patient programs within the timeline ofproduct development, and these largelyuntapped opportunities should be leveragedwhere possible to generate ancillary RWD thatmay inform future development prospects orsupport RWE.

EU initiatives to increase use ofRWEThere are already many national and EUinitiatives ongoing to strengthen RWE. Throughgood coordination and a cross-stakeholdercollaborative approach we can address thechallenges and realise the full potential of RWE

in supporting product development, monitoringand decision-making at all stages of the productlifecycle.

EU level initiatives to increase the utility of RWEinclude:• Initiatives on patient registries (such as

PARENT Joint Action, ENCR - EuropeanNetwork of Cancer Registries, and the EMAInitiative on Patient Registries)

• Initiatives on electronic health records (suchas EH4CR, EMIF, EU-ADR Alliance, RD-Connect, epSOS, EuroRec)

• Initiatives aimed at establishing methods andplatforms to enable and facilitate dataaccess, analysis and collaboration (such asIMI GetREAL, IMI PROTECT, IMI ADAPTSMART, ENCePP; the European Network ofCentres for Pharmacoepidemiology andPharmacovigilance

• Initiatives on HTA (EUnetHTA JA3 aims toconduct pilots on post-launch evidencegeneration and to develop a tool to supportpermanent collaboration on post-launchevidence

• Approaches aimed at the use of social media

Through better data sharing initiatives such asthese, RWE is better placed to support accessto novel products by providing informationacross the life cycle of a medicine fromdevelopment through to HTA.

For example RWE could support productdevelopment by providing information on thenatural history of the disease and the unmetneed, an understanding of resource utilisationand current standards of care, patientrecruitment and potentially differential benefit-risk profiles in targeted subpopulations andbetter forecasting via disease progressionmodels that would all benefit pre-launchapplications.

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Managed access programs provideopportunities for data capture, justthe same as post-launch, as long ascollection doesn’t impact the patientor overburden the physician.

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1. Brooks, K. CRO Outlooks & Market Trends. 2016. Available from http://www.contractpharma.com/issues/2013-06/view_features/cro-outlook-market-trends Accessed July 2016

2. Page, M. J. Demonstrating effectiveness with Real World Evidence. 2015. Available from http://www.medpace.com/PDF/Articles/Get-Real-Demonstrating-Effectiveness-RWE.pdf Accessed July 2016

3. Hughes, B., Kessler, M., & McDonell, A. Breaking new ground with RWE: How some pharmacos are poised to realize a $1 billion opportunity. 2014. Available from https://www.imshealth.com/files/web/Global/Services/Services%20TL/rwes_breaking_new_ground_d10.pdf

Real world evidence opportunities across all phases of drug development are increasing throughpre and post approval studies, meaning opportunities for savings and efficiencies can berealised through strategic planning, data collection, and re-purposing of study materialsgenerated pre-launch as well as post-launch.

SVMPharma is a RWE agency with experience of working on successful HTA resubmissions for NICE and SMC that can help gather the right RWE data and ease the RWE process to ensure your best chance of gaining positive HTA submissions.

SVMPharma is an innovative strategic consultancy, specialising in Real World Evidence (RWE) for thepharmaceutical industry. SVMPharma generates RWE within UK and Europe through bespoke online RealWorld Treatment Evaluators, leading to successful health technology appraisal (HTA) submissions. Clinicaltrial programmes do not reflect real-world clinical practice and outcomes, RWE supplements and enhancesclinical datasets. SVMPharma’s specialist teams focus on delivering the outcomes that matter to your brand.SVMPharma also provides Global RWE, Patient Real World Outcomes, and Big Data Analytics.

To find out more call +44 (0) 1256 962 220

RWE Preclinical Case study

Recently a company who we will term X was looking to understand which molecule entities itwanted to carry forward to preclinical studies within disease areas it had no historical knowledge.Traditionally market insights would have been gained through a classical approach with the use ofdisease registries, market research and Key Opinion Leader insights.

RWE through patient chart reviews is a new approach to gaining insight into preclinical studies andscoping. This example of patient chart reviews involving 25 patients provided key insights into:• Patient clinical pathway from diagnosis to death• Patient treatment and diagnostic pathway use of drug interventions and its outcomes• Market sizing and potential

CONTACT SVMPHARMA

Vaneet NayarDirector SVMPharma

enquiry@svmpharma.com

Tel: +44 (0) 1256 962 220

SVMPharma LtdLandmark HouseStation RoadHook, HampshireRG27 9HA

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