By Rachel Tham, Senior Statistician
Real world evidence generation is playing an increasingly important role in enabling sponsors to gain better insights across a range of scenarios to improve the patient experience. At Veramed our Launch and Commercialisation business unit draws on our team’s extensive experience both internally and within sponsor organisations, supporting evidence generation beyond clinical trials.
In this blog, drawing on material from a recent webinar, I highlight some typical scenarios where statistical support comes into its own to underpin product launch, post marketing surveillance, and Health Technology Assessment (HTA) submissions.
How we understand the landscape
Both pre-launch and post approval, there are numerous occasions where sponsors want to understand the environment that their treatment will be operating in. Data obtained from real world settings provides a vital tool in this process of understanding. Our exploration often centres around these key questions:
- Who are the patients we are aiming to treat?
- How do physicians currently treat the disease? (how will my drug disrupt this current method?)
- What is the current treatment path that payers support?
- What are the treatment guidelines and how well are they adhered to?
- What are the challenges each PPP group faces?
In the context of these questions, I will walk through some different scenarios that may arise as part of a product‘s commercialisation journey, and how real world evidence can help to inform product positioning and communication with stakeholders to ensure the treatment is made available for the right patients.
Pre-launch/late phase development: Working with new or rare diseases
Developing a strong understanding of the landscape is particularly vital when working with therapies in new or rare indications where we don’t have a well-defined way to identify the patients of interest. In such a situation, if there are unknowns about the patient profile and typical treatment pathway, we can leverage real world studies to help identify the patient characteristics and better understand their treatment, comorbidities, and frequency of visits. This characterisation is vital where the indication has no defined medical code to inform pre-launch planning and give our product the best possible chance of reaching the right patients.
Pre-launch: Understanding the burden of care to better position your product
Another key area for the sponsor in preparing for launch of a new product is understanding the burden of care for both the patient and the healthcare system. Evaluating and communicating this burden is often an essential component in preparing for Health Technology Assessments and helps the payer to evaluate the cost benefit ratio of the product. Using real world data allows us to properly measure the quality of life experienced by the patients through either patient reported outcomes, their frequency of hospital visits, and the occurrence of invasive or painful methods of care. Importantly, we can also assess if the cost of the services may be mitigated by our new product, thus reducing the healthcare burden. Naturally, establishing this case helps to better commercialise and position our product to stakeholders. For example, in one project I have supported, we sought to compare the hospital duration and readmittance outcomes for the sponsor’s product (which relied on IV administration) versus an oral route of administration in patients with gastrointestinal complications. The study compared time to first hospitalisation and length of stay after administration of the drug and found that the IV treatment reflected better hospitalisation outcomes – which in turn gave the sponsor a competitive edge.
Post launch: Establishing how product use may differ from official guidelines
Another common scenario in post launch settings is establishing how a product is used in the real world versus official medical guidelines. This type of analysis may be required if, for example, a product is failing to meet its pre-launch expectations. By identifying patients of interest and quantitatively comparing the actual therapeutic pathway as compared with official guidelines we can gain a deeper understanding of any deviations and devise informed plans for our market access strategies and engagement with stakeholders.
These are just a few highlighted examples, and as our access to data improves along with our ability to harness increasingly powerful statistical methods, the use of real world evidence for commercial applications will surely expand. In turn, as Statisticians, we have an increasingly important role to play in supporting not only clinical development activities, but also helping sponsors to generate valuable evidence that informs communications with payers, patients and physicians and ensures that the right products are used at the right time by the right patients.
Get in touch to learn more about the strategic application of real world evidence in launch and commercialisation settings, and disover our recent webinars on the topic of RWE, HTA submission, more below. Check out our most recent webinar: Getting HTA submissions right in key regions: USA, UK & Germany
