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Bridging the gap between clinical development and patient access with real-world evidence

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In a world where manufacturers must not only prove their product’s safety and efficacy prior to launch, but also ensure commercial success, real-world evidence (RWE) is crucial. Here, Dr Chitra Lele, chief scientific officer at Sciformix Corporation discusses the RWE’s continuum and how it could bridge the gap between clinical development and patient access.

Real World Evidence (RWE) is defined by the FDA as ‘clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD)’.1RWD is the data collected from sources other than traditional clinical trials. These data are being obtained from electronic tracking systems used to capture patient experiences during care.

RWE is increasingly crucial for life science companies to ensure patient access and commercial success. Generating and leveraging RWE requires technical expertise along with strategic insights for informed healthcare decisions and optimal outcomes. However, as the sources of RWD are disparate and the quality can vary greatly, accessing, analysing and drawing conclusions from the data is more challenging than ever. Going forward, RWE will become increasingly more patient centric, as highlighted through the growing emphasis by payers and regulators for advances in this area.

RWE in clinical development and post approval

RWE has a role in both pre- and post-approval. In the clinical development stage RWE can enhance the effectiveness of randomised clinical trials, for example in the initial phases (Phase I and Phase II) it can aid in the identification of patient sub-populations and in understanding patient pathway, disease progression, treatment preferences and patient related outcomes (PRO’s). RWE facilitates operational aspects of drug development by optimising patient recruitment and patient access.

Post-approval RWE can assist in generating further insights to improve safety and effectiveness of products, prescribing patterns and adherence, along with real world health outcomes.

A value-based, patient outcomes approach is driving the role of RWE in formulating a product’s evidence requirements across the development continuum. RWE may potentially explain facets like head-to-head comparative effectiveness, differentiation in sub-population and effects of switching. For example, data from an observational study among insulin-naïve patients with uncontrolled Type 2 diabetes was used to assess health outcomes of Toujeo (insulin glargine) compared to basal insulins.2RWD pooled from US insurance claims databases was used to demonstrate effectiveness of Eliquis (apixaban) among non-valvular atrial fibrillation (NVAF) patients.3

RWE in patient access

RWE can play a positive role in improving patient access by providing payers with information on the effectiveness in the real world and the cost-effectiveness in comparison to other drugs on the market. RWD can improve health outcomes for patients and help pharmaceutical companies to develop a more efficient and streamlined commercialisation process.

The scope of RWE in market access includes achieving optimal price and full reimbursement, favourable recommendation from health technology assessment (HTA) authority and inclusion in various formularies at hospital, regional and national levels. As an example, RWE from US insurance claims helped Zytiga (albiraterone acetate) receive recommendation by National Institute for Health and Care Excellence (NICE) for prostate cancer patients.4

Performance-based risk-sharing arrangements (PBRSA) involve conditional reimbursement by payers based on health outcomes and help improve market access for manufacturers. RWE provides insight into the impact of new intervention in the ‘real’ patient population; focusing on the efficacy to effectiveness translation. Smart contracts can be used to trigger drug reimbursement from insurers only for responders, thus enabling risk sharing between the stakeholders, based on RWD sharing via blockchain technology. RWD is also important for high-priced therapies; wherein ‘micro-pricing’ agreements are fulfilled based on attaining continuous outcomes and quality of life targets. There is an example of a pay for performance agreement in the cardiovascular area; wherein reimbursement will depend on reduction in heart failure hospitalisations.5

Clinical development to patient access: The RWE continuum

Previously, there was a difference in the evidence requirements for clinical development and patient access. The continuum of RWE looks to bridge this gap with value-development plans. In the now saturated pharmaceutical market, manufacturers must not only prove their product’s safety and efficacy prior to launch, but also present evidence-based recommendations on the way the product’s value is determined, demonstrated, communicated and realised in the market.7The plan should be formulated during the proof-of-concept phase, and later include new information over the product lifecycle as it evolves. RWE allows for more accurate conclusion about medications to be drawn and provides new insights.

Evolving RWE

Going forward, drug pricing will be based on health outcomes rather than payment per pill. The current constraints of using and leveraging RWE will be mitigated by the use of technologies like big data, machine learning and blockchain technology. The evolution of the Internet of Things (IoT) means that the future sources of RWE are likely to be much more diverse. Furthermore, blockchain technology will enable more secure storage of patient health data and the direct transmission of anonymised data to pharma companies, helping to incentivise the right health outcomes in the system.

Also, precision health economics outcomes research (HEOR) through big data can aid healthcare decision-making tailored to specific clusters, so as to provide the most optimum and cost-effective individualised treatment. RWD obtained from wearable technology can be used by regulatory and HTA authorities to determine drug effectiveness. Perhaps the biggest impact of these future trends will be felt in the emerging markets by ‘leapfrogging’ the aforementioned innovations.

Summary

With increasing emphasis on using RWE seamlessly across the entire product lifecycle, from development to patient access and back to discovery and development, there’s a distinct advantage to having cross-skilled teams. Solid experience in clinical trials with a strong orientation to the needs of real world studies and the peculiarities of RWD is the need of the hour.

Since the volume of work to be done in RWE, HEOR and MA is growing, largely due to vast amounts of data being available and the advances in data analytics, partnering with service providers with the required domain understanding and robust analytical and scientific writing skills is becoming more prominent. Partners who have the right blend of clinical development and patient access experience will be well-poised to provide value-added support to their clients.

References:

  1. FDA. Real World Evidence. 2018. Available online: https://www.fda.gov/ScienceResearch/SpecialTopics/RealWorldEvidence/default.html[accessed 27/02/2018].
  2. Sanofi, 2017. Switching to Toujeo in a real-world setting produced a similar number of severe low blood sugar events as insulin Degludec. (Internet) http://www.news.sanofi.us/2017-11-30-Switching-to-Toujeo-R-in-a-real-world-setting-produced-a-similar-number-of-severe-low-blood-sugar-events-as-insulin-degludec[accessed 27/02/2018]
  3. Bristol-Myers Squibb Press Release, 2017. Bristol-Myers Squibb and Pfizer Present Observational Real-World Data Analysis on the Effectiveness and Safety of Eliquis (apixaban)
  4. Ramaekers, B.L.T., et al, 2017. Abiraterone Acetate for the Treatment of Chemotherapy-Naïve Metastatic Castration-Resistant Prostate Cancer: An Evidence Review Group Perspective of an NICE Single Technology Appraisal PharmacoEconomics (2017) 35: 191. (Internet) https://doi.org/10.1007/s40273-016-0445-5[accessed 27/02/2018]
  5. Omar Ishrak. Medtronic. 2018, Jan 8. 36th Annual J.P. Morgan Healthcare Conference, San Francisco. (Internet)file:///C:/Users/ppal/Downloads/Release0463fc6e-d125-459e-a1e0-4f83a78b9ad9_2324263.pdf[accessed 28/02/2018]
  6. Business wire, 201 Cigna Implements Value-Based Contract with Novartis for Heart Drug Entresto (Internet) https://www.businesswire.com/news/home/20160208005778/en/Cigna-Implements-Value-Based-Contract-Novartis-Heart-Drug[accessed 28/02/2018]
  7. Richard Borrelli, 2008. Health Economics, Outcomes Research and Brand Strategy-Canadian Pharmaceutical Marketing, An IMS Viewpoint. (Internet) http://www.stacommunications.com/journals/cpm/2008/04-April%202008/023-IMS.pdf[accessed 22/01/2018]
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