How real-world evidence supports market access for high cost/low volume therapies 

Written by Mariam Bibi

Real-world evidence (RWE) can be leveraged by biopharmaceutical manufacturers to highlight the value of high-cost and low-volume therapies and highly targeted novel therapeutic approaches.

In this excerpt from her published article, Mariam Bibi, Senior Director, Global RWE, explains why manufacturers are developing their real-world evidence strategies earlier in the clinical development process, and makes recommendations for overcoming the challenges of using RWE in multiple markets and differing regulatory landscapes. 

Leveraging real-world evidence to highlight the value of high-cost and low-volume therapies 

The advent of precision medicine has driven innovations in therapy areas in which there are significant unmet patient needs, including oncology and rare diseases. These novel therapies harness the body’s cells and/or DNA to fight diseases, resulting in highly personalized treatments targeted to individual patients. 

RWE can be leveraged by biopharmaceutical companies to highlight the value of these high-cost and low-volume therapies and highly targeted novel therapeutic approaches such as CAR-Ts, which reprogram the body’s immune system to fight cancer. Often, these therapies are approved based on the results of Phase 2 studies, which is considered appropriate because of the high unmet clinical need. However, due to the rare nature and genetic foundations of some diseases, patient populations are small, and it can be difficult to identify and recruit patients for clinical trials, leading to gaps in the clinical trial evidence base. 

In addition, these single-dose cell and gene therapies can cost hundreds of thousands of dollars. The launch price is based on the initial data, which indicate that the one-time treatments could cure fatal diseases and reduce the burden on healthcare systems. However, more data are needed to prove these claims across the patient’s lifetime in routine clinical practice. As a result, payers and insurers are turning to outcomes-based agreements, which include regular reviews of RWE that demonstrate the effectiveness of the product in the market. 

Innovative biopharmaceutical companies are developing their real-world evidence strategies earlier in the clinical development process 

While randomized clinical trial data are still the gold standard to show that a treatment can work, lack of external validity means the evidence may not reflect what is happening in the real world. 

With RWE, you can make use of existing data sources, bespoke data collection, and different approaches for analyzing data. It is important to have a strategy for using RWE and to be very clear about your evidence gaps and study objective(s). You must also adopt the right data source for the stakeholders you are developing the evidence for. Having a clear protocol and statistical analysis plan is important. Understanding the breadth of RWD sources in a particular therapy area and market can also help you develop the appropriate RWE strategy. 

Historically, in the pharmaceutical sector, there has been a focus on gathering this evidence around the time of market approval.2 However, innovative biopharmaceutical manufacturers are now developing their RWE strategies as early in the clinical development process as possible, allowing time for the planning and implementation of RWE to support payer discussions and negotiations. 

Real-word evidence is increasingly being recognized as an important source of data by payers, providers, and healthcare professionals 

Historically, funders and regulators have been reluctant to use RWD and RWE to make decisions about the value of products, particularly at launch. Regulatory authorities have identified problems with poor quality and inconsistent data.3 A primary source of RWD is electronic health records, which may be inconsistent and unstructured. While patients are constantly monitored in randomized controlled trials (RCTs), in the real world they may not visit their doctor regularly, meaning that the electronic health record data may be incomplete. However, we are now seeing RWE increasingly being recognized as an important source of data by payers, providers, and healthcare professionals. 

As when developing RCTs, taking a global and consolidated approach is central for developing an RWE strategy that supports the value of the product. However, the difference in data sources across markets and countries presents a challenge. 

Each market has a different healthcare system and approach to data collection. In the West, for example, there are more established data structures, the data are more readily available, and there are processes and protocols in place to access existing data and collect bespoke data. This differs in other areas of the world – for example, in the Middle East, where there is limited availability of epidemiological data – more often than not, bespoke data collection is required. 

Overcoming the challenges of using real-word evidence in multiple markets and differing regulatory landscapes 

There is a lack of guidance on using RWE in multiple markets, and the regulatory landscape differs between countries. Manufactures can overcome these challenges and develop a consolidated approach by producing a global, nuanced strategy. When developing an RWE strategy, it is important to be very clear on the overarching objectives and then to identify the data sources and evidence-generation strategies to suit your markets of interest. The evidence requirements differ by market, so a pragmatic approach is important. 

When designing RWE studies, companies must consider stakeholder requirements, which can affect the approval of therapies by both regulators and payers. We are seeing more “outcomes-based funding” models, in which payment is approved based on the performance of therapies. 

The biopharmaceutical sector has been proactively engaging with regulators and payers. In Europe, the GETREAL initiative, part of the Innovative Medicines Initiative, was set up to drive the adoption of RWE, helping stakeholders to make healthcare decisions based on data. They have engaged with different stakeholders, such as biopharmaceutical companies, payers, and regulators to produce guidance and training. 

In addition, the European Medicines Agency (EMA) is developing a real-world evidence network called Darwin EU. The National Institute for Health and Care Excellence (NICE) recently published their RWE framework looking at how RWE can inform NICE guidance. Initiatives like this have been motivated by an appetite for RWE, which has been driven by novel therapies and advances in data curation and analysis. 

Maximize the value of your asset by leveraging real-world evidence 

This excerpt was taken from the free article Maximizing the value of your asset by leveraging real-world evidence. The full article explains how manufacturers can use RWE strategies can better leverage data on the effectiveness and safety of novel therapies over time, and develop iterative evidence generation strategies to meet payer requirements. 

Download the article now to discover: 

  • why global regulatory authorities are placing increasing importance on RWD and RWE. 
  • how manufacturers can leverage RWE to highlight the value of high-cost and low-volume therapies and highly targeted novel therapeutic approaches such as CAR-Ts 
  • why engagement with healthcare professionals is crucial for generating RWE, and some recommendations for effective partnerships. 

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