Richard White, MA, PhD, Oxford PharmaGenesis Ltd, Oxford, UK; Jacob Willett, MPH, Oxford PharmaGenesis Inc., Newtown, PA, USA; Polly Field, MSc, DPhil, Oxford PharmaGenesis Ltd, Oxford, UK
This article is part of the Medical Publications 101 article series, which serves as an introduction to various topics that are relevant to medical publication professionals.
How Is HEOR Going to Change the World of Medical Publications?
Health economics and outcomes research (HEOR) has been the fastest-growing function in the pharmaceutical industry for a decade or more. Medical publication professionals are seeing publication plans increasingly populated by HEOR studies – particularly post-launch. While medical publication professionals are accustomed to working with randomized controlled trials (RCTs) and literature review publications, HEOR studies require engagement with unfamiliar methodologies, technical jargon, and new collaborators. Yet, communicating the evidence provided by HEOR studies is now essential in order to succeed in bringing new health interventions to patients who need them. The role of medical publication professionals has never been more important!
What Are the Fundamentals of HEOR?
HEOR studies are designed primarily to provide evidence for a payer audience (eg, Health Technology Assessment bodies, Pricing and Reimbursement/Formulary Committees, Pharmacy Benefit Managers) to support the value proposition for a healthcare intervention – which could be a drug, device, diagnostic tool, or procedure – and to answer the following two key questions.
- What is the impact of the intervention on health outcomes? (Benefits)
- What are the economic consequences of implementing the intervention? (Costs)
A schematic approach to developing a value proposition is shown here.
To assess benefits and costs fully, an HEOR plan must provide comprehensive evidence to:
- Characterize the disease area and the unmet need in different geographies
- Who are the patients? How many are there? What is the burden of the disease (costs and impact on patients)? How are patients treated in different countries? What measures can be used to assess patient-reported outcomes (PROs)? How effective are current treatments?
- Demonstrate the benefits of the intervention required to achieve reimbursement
- What are the comparative clinical benefits and safety profile in terms of hard outcomes (eg, hospitalization, stroke, myocardial infarction, death)? How does treatment improve patient (and caregiver) function, quality of life, and healthcare resource utilization?
Health economic (or pharmacoeconomic) models can then quantify the cost-effectiveness of the new intervention by assessing the long-term incremental benefits of an intervention (compared with the current standard of care), relative to the incremental costs (new interventions generally cost more than the standard of care).
Estimates of the number of patients in the approved indication and the expected uptake of a new intervention in a particular region or country can be used to assess the short-term budget impact of introducing that intervention (ie, how much extra would it cost a health system per year compared with the existing standard of care?).
That’s the high-level overview – but the devil is in the details. HEOR studies provide unique insights that are of interest to clinicians and patients because HEOR studies are designed to incorporate and complement the data provided by RCTs (which are still designed primarily to demonstrate efficacy and safety for regulatory approval).
Efficacy – the effect of an intervention in controlled conditions, such as an RCT (ie, what can the intervention do) [click this video link for information about the difference between efficacy and effectiveness]
Effectiveness – the effect of an intervention in routine practice (ie, what does the intervention do) [click this video link for information about the difference between efficacy and effectiveness]
HEOR (health economics and outcomes research) – depending on the company, may also be called HEORE (HEOR and epidemiology), HOPE (health outcomes and pharmacoeconomics), HOMA (health outcomes and market access), etc.
PRO (patient-reported outcome) – a measure of the patient’s experience, obtained directly from the patient (ie, in some form of questionnaire)
RWE (real-world evidence) – studies of patient outcomes in routine clinical practice that are generally observational and non-interventional (ie, those conducting the study observe outcomes but do not influence the clinical management of the patient)
What Do HEOR Colleagues Do, and How Do They Do It?
HEOR groups typically begin their work early in the clinical development program for a new intervention – ideally, after clinical proof of concept is established – and support a product throughout its life cycle. HEOR professionals often commission studies from external vendors to develop the required evidence, with the approach depending on the disease, the potential indication in question, and the phase of the product life cycle.
Systematic literature reviews are a common first step to determine the current level of evidence, and to inform a gap analysis of the need for new research. When the existing literature is outdated or insufficient, common methodologies employed by HEOR groups to gather the required evidence include:
- clinical/claims database analyses and chart reviews (real-world evidence [RWE] studies)
- clinician and payer research, including interviews
- patient interviews, and PRO and utility studies
- modeling studies (eg, health economic models, indirect treatment comparisons)
A major change over the past decade has been the increasing demand by payers and other decision-makers for evidence of product value throughout the life cycle, rather than the previous focus on information to support launch. HEOR studies have, therefore, become the major source of new data post-launch of a product in a new indication. HEOR groups are highly active post-launch, as the balance of evidence shifts from indication-seeking RCTs towards database/RWE studies of effectiveness/safety/drug use and modeling of comparative effectiveness.
What Insights Can HEOR Provide Beyond Traditional RCTs?
The breadth of HEOR studies reflects the fact that key health stakeholders have different value drivers and, hence, different evidence requirements, which may not be met by RCTs alone.
Examples of the ways in which HEOR studies can complement and go beyond the clinical efficacy and safety data provided by RCTs include:
- Translating RCT endpoints into long-term health outcomes – using surrogate endpoints/short-term RCT data to model the impact of interventions on hard outcomes over a longer time horizon, often a lifetime.
- Providing comparative evidence – indirect treatment comparisons assess the relative efficacy and safety of different interventions, even when a head-to-head RCT has not been or cannot be done.
- Quantifying disease burden – clinical/claims database and other RWE studies assess burden of disease in terms of monetary costs (eg, costs of hospitalizations, surgical procedures, drug treatments) and humanistic impact (eg, effect on quality of life, daily functioning, caregiver requirements).
- Delivering locally relevant evidence – HEOR studies are often designed to build on RCTs by collecting data from a particular country or region, and providing local evidence not usually obtainable from a multinational RCT.
Publications on all these aspects are of value to clinicians and patients – and that is where the medical publication professional has a key role to play.
What Are the Challenges with HEOR Study Publications for Medical Publication Professionals?
HEOR groups may not necessarily consider publications (and particularly peer-reviewed journal articles) as a priority communications channel for their target audience. Payer stakeholders may be better engaged directly through formal dossier submissions or face-to-face communication tools (eg, payer value presentations), and will be accustomed to citations to conference abstracts/posters or simply unpublished ‘data on file.’
The lack of familiarity with approaches to routine publications for RCTs brings further challenges.
- Expert clinical input – HEOR study vendors often conduct studies themselves, without input from clinicians; however, without a sound clinical perspective on the design, analysis, and interpretation of results, the studies can have limited value.
- Timelines – HEOR studies (eg, database analyses, chart reviews, economic models) often require iterative analysis and, therefore, have less clear-cut timelines and completion dates than RCTs.
- Writing style – HEOR study vendors tend to focus on presentations at specialist conferences, and often write in a technical style that is not suitable for mainstream clinical conferences and journals.
- Compliance – HEOR groups may not be familiar with guidelines on good publication practices (eg, GPP3) and related issues, such as author engagement, review processes, and disclosures.
How Do Medical Publication Professionals Overcome Challenges with HEOR Study Publications?
It is often said that the barriers to success are internal, and this is certainly true when it comes to maximizing the value of HEOR study publications. Establishing a collaborative working relationship between HEOR and medical publications groups is essential, and a joint workshop involving all relevant parties can be an important first step.
Key success factors include the following actions:
- Share and understand each other’s perspectives, objectives, and audiences.
- Help HEOR groups understand the importance of clinical publications, journal selection, good publication practices, and compliance.
- Help medical publication professionals understand the evidence provided by different types of HEOR studies, and the challenges of HEOR study planning and analysis.
- Define common goals, and plan publications together.
- Co-create an integrated publication plan.
- Agree on standard procedures for document review and approval.
- Engage medical writers with an understanding of HEOR who can achieve the following:
- Gain the respect of the HEOR team, and work effectively with them.
- Communicate HEOR data to a clinical audience.
- Agree on processes for ongoing communication of publication plan updates.
With effective collaboration and communication, medical publication professionals and HEOR professionals can help unlock the power of HEOR evidence in demonstrating the value of new interventions. Ultimately, it is the patients who will benefit!