Archive for category Value Demonstration

Key Elements of a Real-World Evidence Generation Plan: Integration of Product Support Activities

Shelby Corman
Shelby Corman
Pharmerit International

Real-world evidence is a key component of value demonstration for pharmaceuticals, diagnostic agents, and medical devices throughout the product lifecycle. Planning for real-world evidence needs early in the pre-launch period can allow for seamless integration of health economics and outcomes research to support product value and market access.

  1. Start with the “aspirational” product value story and work backwards

It is necessary to plan your real-world evidence generation strategy early in the product development process, so that the correct and most impactful data can be collected and analyzed to support the product’s value story. A set of aspirational value messages can help guide evidence generation strategies, including those for real-world evidence. A literature-based “match and gap” analysis can be used to determine whether aspirational messages are supported by existing literature, and then evidence generation strategies can be tailored to fill key data gaps.

Choosing the appropriate study design and planning for the time required to execute are important. Secondary data sources (such as administrative claims data) are readily accessible, and analyses can be completed in 3 to 6 months, but essential elements such as disease staging and severity are often missing. Primary data collection is often required to obtain data elements for stratification of patients, such as biomarker results or rationale for treatment decisions. Retrospective chart reviews can generate results in 6 to 12 months and can be combined with a patient-reported outcome (PRO) survey in a hybrid design to provide a snapshot of quality of life or patient preference. Prospective study designs usually take 12 months or longer to generate results but create opportunities to collect longitudinal quality of life or other health outcomes data.

  1. Establish the disease burden

Demonstrating the clinical, economic, and humanistic burden of a disease is a key need early in the development process and sets the stage to inform stakeholders regarding the potential value of a product. For rare diseases, this can mean raising awareness of the disease, estimating incidence and prevalence, and characterizing the population with the disease. Data for more common illnesses may be available but out of date. Estimating the economic burden of disease is especially important to a payer audience, in order to justify resources required to adopt new technologies.

Real-world evidence on the epidemiology and burden of disease should be communicated in peer-reviewed publications, incorporated into global and payer dossiers, and used to populate economic models. An accurate estimate of disease incidence and prevalence is crucial to understand the budget impact of the adoption of new technologies.

  1. Identify current treatment patterns and unmetneeds

Understanding the current standard of care for the target patient population and, by extension, the unmet patient needs expected at the time of launch, is essential for an informed market access strategy. Retrospective studies to identify current market share are required to populate budget impact models and can be used to drive the selection of comparators for cost-effectiveness models.

Further exploration of patient characteristics and clinical outcomes associated with current treatments can support a value narrative centering on unmet needs. For example, real-world evidence of early treatment discontinuation due to adverse events with existing therapies can support value messages for products with improved adverse event profiles. Such a finding could also highlight the need for the integration of qualitative research or patient-reported outcomes assessments in your real-world evidence generation strategy to further characterize the burden of adverse events on patients.

  1. Determine the key economic drivers

A solid economic value story is crucial to reimbursement in some countries and plays a supporting role in others. As such, data needs for economic models are a key consideration for real-world evidence generation plans.

The scope of real-world evidence needed depends on the complexity of the disease process and its treatment, and in turn the complexity of the model. Real-world studies can be used to quantify the cost of treatment success or failure, stratify medical costs by disease severity, and identify the incremental cost of adverse events.

Early economic models, typically designed and executed during phase II development, can be powerful tools for ensuring a solid evidence base for subsequent payer-facing models. Gaps in data required to populate cost-effectiveness and budget impact models, such as treatment pathways and healthcare resource utilization, can be identified early and addressed using real-world studies.

  1. Support clinical effectiveness

From a product development perspective, the role of real-world studies has historically been limited to the post-marketing setting, in which they have been used to fulfill post-marketing commitments, confirm safety and effectiveness in a broader patient population than that studied in clinical trials, and assess comparative effectiveness of treatments. These studies are important components of an evolving value proposition for a product, which can be communicated to decision-makers in the form of publications and incorporation into value dossiers.

In the United States, the 21st Century Cures Act has the potential to significantly reshape how real-world evidence is used in the development of new health technologies. The Cures Act, signed in December 2016, gives the Food and Drug Administration (FDA) two years to develop a framework for using real-world evidence. Guidance has been developed for medical devices, noting that real-world data could be used for purposes such as generating hypotheses to be tested in prospective studies, as a historical or concurrent control group, and as evidence to support biomarker validity, among others. Similar guidance is in develop for drugs and biologics.


Real-world evidence can support product value directly, by raising awareness of the disease burden and unmet needs, and indirectly, by providing inputs for economic models and identifying opportunities for additional qualitative or patient-centric research. Understanding the role of real-world evidence in the overall product value strategy and coming up with a solid plan early in the product development process can help justify the need for resources to conduct these studies in the face of competing priorities.

Contact Pharmerit for more information on Real-World Evidence.

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Who Are the Real Change-Agents in Pharma Market Access? The Growing Role of Value Demonstration

Would you believe it if I said that in a few years, the term “HEOR” may go the way of man-buns and flip phones?

While the man-bun joke about the terminology may be a slight exaggeration, our field is indeed undergoing a notable change and we are leaving some old approaches behind. We will talk about these changes in HEOR in an interactive, live-streaming Fireside Chat with a Panel of leading industry executives from Pfizer, Teva, Oxford PharmaGenesis, and HealthEconomics.Com:

[WebinarThe Changing Face of Careers in HEOR & Value Demonstration: Fireside Chat with Pharma and Communications Agency Executives

February 22, 11am-12noon EST

Fortunately for you (and us), the science of HEOR (and its commercialization counterpart, Market Access [MA]) will likely remain highly relevant and continue to grow; however, the way we are applying it is evolving into “Value Demonstration”, and it is much more customer-centric.  This change is more than just semantics, or at least many of us think so.

Value Demonstration Tools


Value Demonstration refers to tailored evidence-gathering solutions that demonstrate the clinical and economic impact of products on patients and health systems, while linking outcomes to quality measures, all of which are strategically incorporated into product life cycle planning.

We are moving away from a brand, volume, and narrow clinical focus of pharmaceutical interventions, and broadening our scope to real world evidence that has a broader end-customer perspective based on their definition of value.

Are you and our industry adequately prepared for this new role?

Perhaps, but it will take Organizational Transformation.

The change to Value Demonstration and Real-World Evidence is playing out in all corners of the healthcare ecosystem. In a study of HEOR professionals conducted by HealthEconomics.Com, 90% of Biopharma and more than 80% of Consulting professionals say that market demand for RWE is increasing. Departmental siloeing is an albatross, and there are herculean efforts to work at an enterprise level and focus on the customer.

Simultaneously, many Pharma/Life Sciences, Consultancy/CROs, and Scientific Communication Agencies are showing growth in Medical Affairs and Payer Evidence/Patient Access commercialization departments. These groups are gaining clarity in their responsibility for RWE, Value Demonstration, Information Strategy & Data Analytics, and Value Translation.

Change brings opportunity (and challenges).

This change to Value Demonstration is an opportunity. An opportunity to positively impact the healthcare system and patients, an opportunity for innovative business models, and an opportunity for employees to bring new and needed skill sets to their organization.

According to the most recent Global Salary Survey for HEOR & Market Access of 500 industry professionals conducted by HealthEconomics.Com, the percentage of respondents who identify Value/Market Access as their main job grew by 20% in the past two years, while those that specifically identified HEOR as their main job function decreased by 24%!  Other job functions that grew in 2017 (vs 2015) were Pricing and Reimbursement (three-fold increase) and HEOR/Value-focused Medical Communication (two-fold growth).

Respondent-reported Job Function, 2017 vs 2015 Global Salary Survey


In addition to organization level changes, skill sets of employees are changing. In this same Global Salary Survey, we asked about the top 3 things to change about HEOR/MA. As shown in the figure below, involvement of HEOR in the early development phase and less separation of HEOR/Market Access from other departments were the main areas that needed the most change in our industry. Moreover, HEOR professionals expressed the need for additional cross-departmental/functional opportunities, more time with customers, and the need for more business experience (as opposed to technical) to establish better links between HEOR and other industry functions and customers.


Areas of Needed Change in HEOR/MA, Global Salary Survey, % responding


These issues related to strategic focus, organizational change, and skill sets for those involved in HEOR and Value Demonstration are pivotal. We, as an industry, need to develop strategies to address the pressing concerns in industry careers and job responsibilities of HEOR & Market Access professionals.  Register now for the collaborative February 22 webinar:


Led by Dr. Patti Peeples, CEO at HealthEconomics.Com and Dr. Richard White, Commercial Director at Oxford PharmaGenesis, along with industry expert Paenlists Dr. Riad Dirani, VP of Global HEOR, Teva, and Danielle Bargo, Director, HEOR (Gastroenterology), Pfizer, we will discuss:

  • Trends in modern Value Evidence Job functions
  • Staffing and training to develop employees to successfully manage new customer demands
  • Trends in Compensation (Salary, Benefits, Bonuses) for HEOR, Market Access, and Value Demonstration
  • A case study of new skill sets essential for Value Demonstration/Medical Affairs
  • Salary trends derived from the 2017 HEOR & Market Access Salary Survey
  • Substantial Q&A between Audience and Panelists

Drop us a note if you have issues you would like to see addressed in our webinar, or thoughts about this blog post.  See you on February 22nd!

Written by:

Ankit Shah, MS, BS Pharm, Contributing Writer

Patti Peeples, RPh, PhD, CEO of HealthEconomics.Com

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