Tom Drake, MA, CMPP, Global Outcomes Group, Reston, VA
Publication professionals work in an industry experiencing a shift in required evidence related to a product’s value, as well as its safety and efficacy. They manage the release of clinical and economic data that can improve the lives of billions of people globally. That is the easy part. The challenge today is to navigate the changing needs of stakeholders, both inside and frankly, more importantly, those external stakeholders who control the levers of product approval, product utilization, and product reimbursement.
This article will cover one segment of the transformative health care industry, notably communicating a product’s value in a competitive global marketplace. Three key trends in health economics and outcomes research (HEOR) are expected to have a significant impact on publication planning:
- Role of Real-World Evidence (RWE) and Patient-Reported Outcomes (PRO)
- ICER (Institute for Clinical and Economic Review)
- Earlier Use of HEOR in Publication Planning
Historical Review of Economic Examples
First, let’s consider the title – Salt, Louis IV, and HEOR and their connection to publication planners. Governments have struggled over millennia about how best to manage and distribute finite resources.
- Table salt was once as prized as gold; North African traders made fortunes moving salt from Timbuktu to Europe. (Salt: A World History, Kurlasky M., 2002). Due to its scarcity and value, European kingdoms taxed salt for needed revenue; in France, it was termed “gabelle” and was in place until 1946. Gandhi produced salt in India against British imperial law.
- King Louis IV of France hired a cost accountant in the 17th century to determine the actual price of his warships and calculate how many ships could be built before bankrupting his court (The Information Master, Soll J. 2009).
- As for health care, it is one of the costliest segments of present-day government expenditures. In 1948, the United Kingdom set up the National Health Service (NHS) to manage health care as a government single-payer system. In the United States around that time, private sector innovators like Henry Kaiser set up comprehensive health clinics for workers (ie, Kaiser Permanente). Now 70 years later, governments remain under pressure to better design funding and delivery systems that provide effective health care to its citizens.
This brief historical review illustrates that governments and industry are not embarking on a new quest but rather integrating innovation, technology, and information in ways not previously possible to support health care and, more specifically, pharmaceutical/medical device expenditures and utilization. The collaboration of publication professionals with HEOR benefits governments, industries, and patients by coalescing clinical and economic data and managing its timely dissemination to relevant stakeholders.
RWE/PRO & ICER & Earlier Use of HEOR Information
Three key Health Care Economic Information (HCEI) trends – RWE/PRO, ICER, and Earlier Use of HEOR Information – provide both a challenge and opportunity for publication professionals. How do publication professionals become a competent partner to HEOR colleagues, thereby providing effective communication/publication planning strategy and tactical roll-out? For those who are not health economists, there is some good news: numerous resources exist to become more fluent and comfortable in the HEOR sector. Several ISMPP resources can be found in the Appendix at the end of this article.
Trend #1 – RWE/PRO
Growing demand exists across health systems globally for the inclusion of RWE and PRO analyses in clinical trials to support reimbursement and market access decisions. Currently, only about one-third of product launches achieve revenue expectations. (The Secret of Successful Drug Launches, McKinsey, 2014) Earlier involvement of RWE in clinical trials, starting in Phase 2, as part of an evidence plan has the likelihood to deliver more formulary success earlier in a product’s launch. Stakeholders, such as the US NIH (National Institute of Health) and PCORI (Patient-Centered Outcomes Research Institute), acknowledge that PRO and subsequent PRO-performance measures (PRO-PM) are important sources of medical data that can be shared and collected into reliable, valid measures of product and patient performance. PCORI has funded more than $2 billion in PRO research since its founding in 2010 as part of the US Affordable Care Act. Expedited product approvals using RWE and PRO data are also on the rise. An integrated publication plan incorporating RWE and PRO data, which addresses the hurdles to formulary uptake, would aid in enhancing product acceptance and usage.
The Food and Drug Administration (FDA) acknowledged that RWE/PRO data can “effectively complement the knowledge gained from ‘traditional’ clinical trials, whose well-known limitations make it difficult to generalize findings to larger, more inclusive populations of patients, providers, and healthcare delivery systems… In the face of increasing treatment costs, payers are increasingly asking for evidence of clinical value before providing coverage.” (McKinsey RWE Report, May 2018)
Trend #2 – ICER
“ICER was founded in 2006 as an independent, non-governmental, non-profit, non-partisan research organization to objectively evaluate the clinical and economic value of prescription drugs, medical tests, and other health care and health care delivery innovations” (ICER 2018).
ICER is becoming increasingly relevant as a credible source for product evaluation reports, which include a long-term perspective on value, evidence on comparative clinical effectiveness, and information on potential budget impact. These reports cover many disease segments and products, including Atrial Fibrillation and Opioid Use Disorder, with more than a dozen new reports presented each year. Importantly, 26 pharmaceutical manufacturers and payers have joined ICER – including AstraZeneca, GlaxoSmithKline, Merck, Novartis, and Sanofi, along with Aetna, Anthem, and Blue Cross Blue Shield – signifying the increasing importance of independent evaluation of both clinical and economic evidence.
In a recent example, ICER created a report last year on rare (orphan) disease treatments and hosted a multi-stakeholder policy meeting in which methods for assessing the value of new drugs for rare conditions was discussed. The meeting included a review of how these methods can be applied to recommend prices that reflect the value of orphan drugs to patients and the health system, to allow for broader insurance coverage for innovative new treatments. A full report is available on the ICER website https://icer-review.org/topic/orphan-drug-summit/.
On April 10, 2018, ICER published two white papers on RWE in collaboration with the UK Office of Health Economics (OHE), bringing their joint perspective to this important segment of the health care industry. The white papers provide guidance on the development of RWE for drug coverage and formulary decisions.
- The first paper, Real World Evidence for Coverage Decisions: Opportunities and Challenges, “highlights the challenges associated with wider use of real world evidence, including increased potential for bias, the risks of incomplete data, and the lack of universally accepted methodological standards.”
- A companion paper, Understanding the Context, Selecting the Standards: A Framework to Guide the Optimal Development and Use of Real World Evidence for Coverage and Formulary Decisions, “sets out a conceptual framework to guide the generation and adoption of real world evidence in light of a shared understanding of key contextual issues, such as whether the evidence is intended to support an argument of superiority of a drug versus similar comparators, or whether the evidence will be used to argue for increased drug spending in hopes of lowering overall health system costs.”
Publication professionals can track the release of ICER reports pertaining to their disease or therapy areas of interest. ICER publishes its calendar for disease/therapy area summit meetings and subsequent release of reports on its website: https://icer-review.org.
Trend #3 – Secure Your Piece of the PIE: HCEI/HEOR in Pre-Approval Publication Planning
In June 2018, the FDA released a final guidance covering the dissemination of both HCEI and Pre-Approval Information Exchange (PIE). This FDA ruling, in addition to the 21st Century Cures Act in the US, has been applauded by payers and manufacturers for providing clearer guidance on what economic information can be communicated among stakeholders ahead of product approval. The FDA guidelines provide examples of HCEI that do, and do not, relate to an FDA-approved indication. Importantly, these guidelines highlight information that should be disclosed to audiences receiving HCEI; they also clarify communication of material not explicitly included in FDA-required drug labels, but that “may be consistent” with FDA-required drug labels.
The FDA guidelines also explain that supporting the exchange of health care information pre-approval is allowable and emphasize the importance of maintaining communication channels between payers and manufacturers. Finally, the guidelines explicitly state that PIE must be “unbiased, factual, accurate, and non-misleading.” Traditionally, the timeframe that companies focused on economic issues was at or near launch. Due to congressional actions with both the 21st Century Cures Act (December 2016) and, more recently, the FDA guidance of HCEI and PIE (May 2018), the timelines for building HEOR into publication plans has been accelerated, potentially by several years.
In July 2018, ISMPP University presented a webinar on this topic titled, “Improving HCEI Communication: Publication Planning Professionals Are Key Partners in Building Successful HCEI Exchange.” Figures 1 and 2 below from the ISMPP U webinar summarize the FDA guidance and illustrate possible solutions for earlier dissemination of PIE within the industry environment, respectively.
Figure 1. Summary of FDA Final Guidance
(Improving Health Care Economic Information (HCEI) Communication, Peter Fendt, PharmD, MBA, RPh, Manager, Global Market Insights, Celgene; Brian Ung, PharmD, RPh, MS, Manager, US Health Economics & Outcomes Research, Celgene, July 2018 ISMPP U webinar, slide 28)
Figure 2. Possible Solutions for Earlier Dissemination of PIE
(Improving Health Care Economic Information (HCEI) Communication, Peter Fendt, PharmD, MBA, RPh, Manager, Global Market Insights, Celgene; Brian Ung, PharmD, RPh, MS, Manager, US Health Economics & Outcomes Research, Celgene, July 2018 ISMPP U webinar, slide 27)
In addition, several poll questions were asked of the audience during the ISMPP U webinar. The results indicated the need for publication professionals to become familiar with this FDA guidance and embrace this expanding segment of industry.
- Nearly 50% of webinar respondents were directly involved in communication with payers.
- 30% of respondents routinely interacted with their HEOR/RWE colleagues in both strategic and tactical collaborations.
- Importantly, 70% of respondents sometimes and rarely interacted with HEOR/RWE colleagues.
Publication professionals would benefit from working more closely with HEOR/RWE colleagues throughout publication planning and implementation. Embracing the three trends above would positively impact your organization’s competitiveness, as well as your own professional development. Utilize the wealth of educational content made available to all ISMPP members to build your knowledge of the latest information on these important trends.
Strive to become more comfortable in the HEOR/HCEI segment of the industry. As Dr. David Nash, who was named to Modern Healthcare’s list of Most Powerful Persons in Healthcare, proposed in a 2010 presentation, “No Outcome, No Income” is becoming a sign of the times for publication professionals.
“No Outcome, No Income” – Dr. David Nash
Appendix. HEOR/HCEI Resources in ISMPP Archives and The MAP Newsletter
The ISMPP Archives is available to Society members on ISMPP’s website and contains multi-media resources with HEOR/HCEI content. ISMPP’s The MAP newsletter also contains informative articles on HEOR and RWE. These ISMPP resources provide timely information to better understand HEOR and RWE, and how this trend is impacting the publication planning environment. ISMPP’s European Meeting and Annual Meeting also provide opportunities to continue to gain knowledge about these trends.
2018 European Meeting Session – The Growing Importance of RWE: What Does It Mean for Publication Professionals?
2018 European Meeting Poster – Has Publication Policy Adapted to the Growing Importance of Real-World Data?
14th Annual Meeting Session – The Role of RWE in a Value-Based World: Who’s Listening?
14th Annual Meeting Posters – Evaluation of Data Reporting in Real-World Publications in Clinical Versus Health Economic Journals; Real-World Evidence (RWE) Publications Are An Integral Part of Company-Evidence Submissions for Payer Discussions
The MAP Article, April 2017 – RWE: A Brave New World for the Medical Publications Professional, Richard White, MA, PhD, Oxford PharmaGenesis Ltd, Oxford, UK; Gemma Carter, PhD, Oxford PharmaGenesis Ltd, Oxford, UK; Jacob Willett, MPH, Oxford PharmaGenesis Inc., Newtown, PA, USA
The MAP Article, March 2017 – HEOR: Why It Is Changing the World of Medical Publications, 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