svmpharma real world evidence – real world evidence as a driver of hta approval: can you afford to...
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SVMPharma Ltd,Landmark House
Station Road, Hook, Hampshire, UK,
RG27 9HA
CONTACT US [email protected]
+44(0) 1256 962 220www.svmpharma.com
Real World Evidence as a driver of HTA approval: Can you afford to miss out?
Patient level data access in the
NHS: Generating positive outcomes
through Real World Evidence
Health economies use technology appraisals(HTAs) to assess the value of new technologies.Many positive HTA submissions require afollow up review 2-4 years later, so planningsources of new data has to come almost assoon as the drug gets out the gate. Generationof new data in support of such HTA submissionre-reviews is vital to maintaining access to adrug. Further RCTs can be beneficial, but costlyand hard to launch on a reduced timescale.Real World Evidence offers an exceptionalalternative; after all what could be moresupportive than real clinical outcomes,generated from within the patient population?
We examine the benefits of RWE in support ofHTA submissions and re-submissions, and askwhether RWE is something you can afford topass up.
1. HTA: the path to market accessHealth Technology organisations such as theUK’s NICE, Scotland’s SMC, Canada’s CADTH,Australia’s PBAC, France’s HAS and Germany’sAMNOG assess and give guidance on healthtechnologies to evaluate their clinical and costeffectiveness.
In addition to the promotion of good healthindicated by HTA approval, fundingopportunities are also available to drugs withreceiving positive submissions. Since January2002, it has been compulsory for NHSorganisations in England and Wales to providefunding for medicines and treatmentrecommended by NICE.
Overall, HTA submissions have become anessential part of European market access andthe established norm in the pharmaceuticalindustry, with positive submissions generallyleading to successful uptake of new drugs.
2. Re-reviews: the new standardfor HTA submissions?Increasingly, positive HTA submissions aresubject to re-review, and often within just a fewyears. Since January 93% of new and positiveNICE submissions had assigned re-reviews, andall of those were within three years (at time ofwriting). Of all published technology appraisals(TAs; n=19), three were themselves updates onpreviously published guidance, one was unableto be reviewed due to a lack of evidence, andonly one was approved with no re-review datestipulated. However, in this case a re-reviewwas recommended should there be any changesresulting from a review of another appraisal. Ofthe remaining 14 with mandated review dates,three were recommended within just two yearswith the 11 remaining within three years [1].
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Real World Evidence as a driver
of HTA approval: Can you afford to
miss out?
www.svmpharma.com
©2016 SVMPharma Ltd. All rights reserved
16%
5%
5%
16%58%74%
Published NICE TAs in 2016
Updates to previously published guidance
New & negative
New & no review*
New & positive - Review in 2 years
New & positive - Review in 3 years
RCT data enable many new drugs to gain HTAapproval but are not without their ownlimitations; in particular, whilst good atillustrating clinical outcomes, RCTs areconstrained by their cohort and are difficult toextrapolate economic data from. NICE haspublished guidelines on how it selectsinterventions for review [2]:
At the time of writing, of the ten most recentlypublished positive NICE submissions withupcoming re-reviews, only one has an RCTplanned at the time of writing [1,3]. What arethese companies doing to resubmit? Aseparate evaluation of global HTA submissionslooked at 1,840 HTA decisions across multipleagencies (NICE, SMC, CADTH, PBAC, HAS and G-BA). Only 6% incorporated observational data,however of these submissions, 77% resulted inpositive decisions, compared with 67% in theformer group [4].
When it comes to generating new data insupport of a review, and within such anaccelerated timeframe, RWE typically holds anumber of advantages relative to RCTs:
1. Lower cost2. Speed to set up and run3. Ease to plan and carry out4. Numerous data types available for review5. See how the drug is actually performing in
the patient population
So it is easy to see how RWE offers an attractive alternative to running another RCT, but how do the results stack up? A recent evaluation of NICE submissions that utilised RWE in support of their application revealed that:
Can you afford not to consider RWE for yourHTA submissions and resubmissions?
3. Real World Evidence: the futureof HTA approval?According to the International Society forPharmaeconomics Outcomes Research (ISPOR)Real-World Data Task Force, “data used fordecision-making that are not collected inconventional randomised controlled trials(RCTs)… are essential for sound coverage,payment, and reimbursement decisions” [5].
HTA success is driven by multiple factors andRCTs may not be reflective of the full picture.
For example, if no new clinical outcomesare presented by RCT, how do you showvalue to the healthcare system? RWE
employs a wealth of data already existing inpatient notes that can be utilised to support arange of criteria assessed by HTA bodies.
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©2016 SVMPharma Ltd. All rights reserved
Can you demonstrate the following for your re-submission?
• Is the technology likely to result in asignificant health benefit, taken acrossthe NHS as a whole, if given to allpatients for whom it is indicated?
• Is the technology likely to result in asignificant impact on other healthrelated government policies (e.g.reduction in health inequalities)?
• Is the technology likely to have asignificant impact on NHS resources(financial or other) if given to allpatients for whom it is indicated?
• Is the Institute likely to be able to addvalue by issuing national guidance?
• Is there significant inappropriatevariation in the use of the technologyacross the country?
REAL WORLD EVIDENCE DRIVES
HTA APPROVAL IN 86% OF
SUBMISSIONS!
4 ©2016 SVMPharma Ltd. All rights reserved
www.svmpharma.com
SVMPharma re-submission case study[6]
With an SMC resubmission looming and no new RCTs, the only route was patient access scheme(PAS) or PAS+RWE. The study aimed not to replicate the original RCT, and so primary outcomeslooked at hospitalisations, exacerbations and IV antibiotics. Secondary outcomes collected werethe primary outcomes of the RCT. By judicious selection of these parameters from patient casenotes and Hospital Episodes Statistics (HES) data, the drug was approved for a positiveresubmission using data from just 113 patients across 14 sites in the UK and Ireland. Proof thatgood quality real world data, even in small cohorts, can drive positive HTA outcomes.
NICE rare disease case study[7]
NICE has announced that it can approve BioMarin’s rare drug treatment Vimizin, but hasproposed RWE collection and a price cap to overcome doubts about the drug’s value. The moveshows NICE is developing a new model for rare disease drugs which can transform the health ofpatients, but carry a very high price tag.
HTA success is driven by multiple factors andRCTs may not be reflective of the full picture.
For example, if no new clinical outcomesare presented by RCT, how do you showvalue to the healthcare system? RWEemploys a wealth of data already existing inpatient notes that can be utilised to support arange of criteria assessed by HTA bodies.
RWE needn’t be a complex process involvinghundreds of patients, innumerable recordsacross a myriad of data sources. See theSVMPharma re-submission case study belowfor details on how a small number of patientsreflecting good quality data is sufficient tostrengthen a submission and gain HTA approval.
The feasibility of large-scale trials within apipeline that is ever more focused on niche andpersonalised medicines is decreasing,particularly in the fields of rare diseases andoncology. Within the changing landscape ofdrug development, payers continue to demandrobust evidence to assess the value andeffectiveness of innovative treatments for bothsmaller patient populations and increasinglyexpensive medicines. In these situations, RWEhas proven to be a strong alternative to RCTs.See the NICE rare disease case study below formore information on how HTA bodies such asNICE are actively encouraging RWE to widenaccessibility for limited access drugs.
REAL WORLD EVIDENCE DRIVES
HTA APPROVAL IN 86% OF
SUBMISSIONS!
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1. National Institute for Health and Care Excellence Guidance and advice list. 2016. Available from https://www.nice.org.uk/guidance/published?type=taAccessed June 2016
2. National Institute for Health and Care Excellence NICE technology appraisal guidance. 2016. Available from https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance Accessed June 2016
3. UK Clinical Trials Gateway Clinical Trial search. Available from https://www.ukctg.nihr.ac.uk/home/ Accessed June 2016
4. Van Engen, A. Achieving HTA success with real world evidence. 2016. Available from http://www.quintiles.com/blog/achieving-hta-success-with-real-world-evidence#sthash.3a7oLy76.dpuf Accessed June 2016
5. International Society for Pharmacoeconomics ISPOR global healthcare systems roadmap. 2008. Available from http://www.ispor.org/htaroadmaps/uk.asp Accessed June 2016
6. SVMPharma & Scottish Medicines ConsortiumSVMPharma’s RWE behind a successful re-submission to the SMC. 2015. Available from http://www.svmpharma.com/svmpharma-smc-cayston/Accessed June 2016
7. National Institute for Health and Care Excellence NICE draft guidance conditionally recommends elosulfase alfa (Vimizim) for treatment of very rare life-threatening genetic disorder. 2015. Available from https://www.nice.org.uk/Contents/Item/Display/27268Accessed June 2016
Health technology assessment organisations want robust effectiveness and cost-effective data for more definitive proof that a product provides added value over existing care. When pharmaceutical industry can’t provide the outcomes data to validate these claims, they are facing lower prices and more restricted access than they would like [4].
HTA success is driven by multiple factors and RCTs may not be reflective of the full picture. Demonstration of clinical effectiveness isn’t the only string to the RWE bow, so even if an RCT is commissioned in support of an HTA review, RWE can complement the clinical data generated in powerful ways that RCTs may not. A small number of patients reflecting good quality RWE data can be sufficient to gain approval in HTA submissions and resubmissions. Positive HTA submissions ensure continued access and funding, and are vital for modern European market access. Can you afford not to consider RWE for your HTA submissions and resubmissions?
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) forthe pharmaceutical industry. SVMPharma generates RWE within UK and Europe through bespokeonline Real World Treatment Evaluators, leading to successful health technology appraisal (HTA)submissions. Clinical trial programmes do not reflect real-world clinical practice and outcomes,RWE supplements and enhances clinical datasets. SVMPharma’s specialist teams focus ondelivering the outcomes that matter to your brand. SVMPharma also provides Global RWE, PatientReal World Outcomes, and Big Data Analytics.
To find out more call +44 (0) 1256 962 220
www.svmpharma.com
Written by Dr Harrison Davis
©2016 SVMPharma Ltd. All rights reserved
CONTACT SVMPHARMA
Vaneet NayarDirector SVMPharma
Tel: +44 (0) 1256 962 220
SVMPharma LtdLandmark HouseStation RoadHook, HampshireRG27 9HA
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