For many years, the randomized clinical trial has been at the core of pharmaceutical development, providing valuable insight into the efficacy of new treatments. However, clinical trials can’t provide a complete picture of the real-world effectiveness of therapies and more and more pharma companies are exploring alternative sources of real-world data (RWD) to fill these gaps. Despite its potential, RWD poses challenges when it comes to accessing and analyzing data effectively. How can these be overcome to help RWD reach its full potential?
While the gold standard of investigative analysis to determine the efficacy and safety of a new investigational drug remains the randomized clinical trial, events such as the COVID-19 pandemic have encouraged a new-found reliance on RWD. This is due to the need to collate rapid real-time data on the performance of promising therapies being used on patients hospitalized with Coronavirus in order to identify genuine life-saving treatments. This RWD can come from insurance company records, healthcare provider (HCP) databases, or from patients’ medical devices.
As statistical consultants have long argued, such data has the potential to create more efficient trial designs and provide additional data to inform clinical practice.
With this in mind, there has been a marked increase in the number of pharma companies using RWD. They have access to more data than ever before and more data points on a wider range of patient variables than can be obtained using clinical trial data alone.
Changing use of RWD
Pharma companies are increasingly looking to take advantage of this expanded variety of information, exploring ways of segmenting the data they receive from patients taking new treatments by genetic or biomarkers, or other markers. For many, it is crucial to find the most appropriate data sets possible to inform their understanding of the performance of their therapies.
They are also looking for more qualitative data, exploring not just measurable results of treatment performance, but also how treatments make patients feel, and the perceptions of treatment efficacy among HCPs.
RWD is also increasingly being fed into pricing plans. Pricing market access managers are exploring data on treatment performance, as well as on pricing for similar therapies worldwide to inform the price point for new treatments.
RWD has proven particularly valuable in the world of cell and gene therapy (C>). Traditionally, C>, has posed challenges for pharma companies when it comes to demonstrating value and return on investment due to its necessarily small pool of potential patients. More and more gene therapy specialists are leveraging RWD to showcase the value of their new treatments and the positive outcomes they can achieve.
Challenges with non-standard databases
The amount of data available to customers through RWD, the need to merge data sets, and to segment information as well, all adds a new level of complexity to the world of medical data analysis and clinical research.
RWD is still in its relatively early stages as a source of data for research use. The data itself – what and how much is collected, how and where it is stored, and how it is analyzed – is a long way from being standardized. Best practice in RWD – as well as guidelines from regulators, payers and academic bodies – is constantly evolving and remains highly fragmented.
Efforts are being made not just to turn fragmented recommendations into comprehensive guidance[1], and to harmonize records across different international databases. Nevertheless, many standardization attempts so far have sacrificed some database specific advantages, which either affect the amount of data that can be analyzed or the ease of analysis.
However, it does mean each database has its own structure, advantages and limitations. Some are clean and consistent, but contain relatively basic information, while others are disorganized and incomplete, but offer in-depth insight into a number of aspects relating to patient health and their response to treatment.
Integrating third party data with internal data is another challenge that pharma companies must wrestle with. When a patient is enrolled in a new therapy, privacy laws, such as the Health Insurance Portability and Accountability Act (HIPAA) in the US can make it difficult to access vital information about their health and wellbeing outside of a clinical trial. Patients’ medical data must be anonymized, which can make it hard to track participants in a study across all of their medical records, unless they give explicit consent.
Combatting challenges
These challenges may seem daunting for pharma companies looking to benefit from RWD. Specialist expertise is needed to analyze databases, overcome the complexity and diversity of the data in order to draw actionable conclusions.
However, companies like Ashfield Advisory are here to support in accessing the right data at the right time. We are also working to address the gaps in RWD to ensure it truly lives up to its potential.
As a business, we work to identify and service data sets on behalf of customers. We support clients to understand the latest best practice recommendations for RWD and to navigate the fragmented and changing regulatory and payer guidelines in order to identify and collate the highest quality data.
We are dedicated to exploring ways to combine datasets efficiently to enable companies to benefit from effective and insightful analysis, and to helping customers find best-in-class data and to merge them quickly to empower them to extract real value.
We’re also working to create a common understanding across customers’ businesses of the wide-ranging value of data to every aspect of their business. In addition to providing RWD on treatment performance, we’re able to provide insight into pricing across different markets, as well as into communications challenges and needs for both HCP and patient audiences. As a result, we are helping to provide actionable recommendations for the successful commercialization of new treatments.
The future of RWD
We can expect the demand for RWD to grow in the coming years. We can expect more and more companies to look for data from across the globe, instead of relying on datasets sourced from within a single geography. Companies like Ashfield Advisory are already exploring ways to source and compile truly global datasets for companies.
New solutions are also being found to use data effectively while ensuring compliance with HIPAA and similar data protection laws. Tokenization is an invaluable new tool that can allow us to follow patients through their records without revealing their identity, simply by ascribing them with an encrypted identifier.
In addition, we are seeing more and more companies exploring ways of using RWD to provide insight that can enable them to create new indications for existing drugs. In future, they hope to use this data to demonstrate the value of the drug in new indications to regulatory bodies and payers without the need for an additional clinical trial.
Thanks to all of this, we can expect RWD to play an even more important role in the pharma industry. With the support of analytical experts like Ashfield Advisory, pharma companies can ensure that they take advantage of the benefits of RWD to inform both the drug development and the commercialization process.
