Moving the Needle on Health Policy: Focus on Outcomes-Based Care
The healthcare system paradigm has slowly been shifting towards real-world outcomes-centered care, and rightfully so—in 2019, it was estimated that overtreatment, low-value care, and failure to price medical services and products in the United States alone cost anywhere between $306-$342 billion.1
Wasteful spending, however, is not unique to the United States and is a common occurrence across the world in high-, middle-, and low-income countries alike. It bears not only financial but also clinical burden when a treatment provided to the patients doesn’t yield the expected results.
The constantly increasing demand for healthcare resources and emergence of innovative therapies that enter the market at incredibly high price points challenge traditional policy making and reimbursement processes. This forces decision makers to reassess ways in which these resources are distributed. Using traditional clinical trial safety, efficacy, and quality of care data alone to drive healthcare decisions is no longer seen as best practice and reimbursement agencies around the world are looking for ways to complement them with population-specific insights on treatment effectiveness using real-world outcomes data.
From Plan to Action
Both clinical and nonclinical patient outcomes should be at the forefront of every treatment-related decision. In the United States and other high-income countries, most pharmaceutical manufacturers invest heavily in health economics and outcomes research (HEOR) studies to support their product by providing clinical, humanistic, and economic real-world data that they collect throughout the lifecycle of the product.2 Additionally, countries such as the United Kingdom, Spain, South Korea, and Thailand have a formal way of requesting HEOR information during the health technology assessment (HTA) process allowing them to consider comparative cost-effectiveness and health outcomes data when creating health policies.
“In the Philippines, emphasis should be put on the research to have more data available, but it is mostly a budgetary issue and doing HEOR research is not a priority.” — Chris Muñoz
However, in other parts of the world, it is not always as straight-forward. As Don Husereau, MS, Adjunct Professor, University of Ottawa, School of Epidemiology and Public Health, Ottawa, Canada, points out, “administrative data is collected differently according to how health systems are structured and without any consistent data standard,” and emphasizes that despite the efforts of making consistent decisions, there are various challenges related to mechanisms to use real-world evidence, particularly in disease areas with low patient-population prevalence.
For example, Chris Muñoz, Board Vice President, Philippine Alliance of Patient Organizations (PAPO), Manila, The Philippines, explains that the HTA council within the Philippines Department of Health was created only 2 years ago and this year, patients will finally be able to submit their first topic nominations (referrals of topics for technology appraisals that can then be used for dissemination and health policy decision making.)3 “Generally, everyone, including pharmaceutical companies and the government, can submit this form,” explains Muñoz, “but the patient form is very different—it mostly focuses on patient wellbeing after receiving the treatment, because we added a quality-of-life survey as an attachment to this form.” He adds that PAPO’s goal is to not only show the direct effect of treatment on the disease outcome, but to also provide the government with information about the effect it has on patients’ families and their everyday lives.
“We have to make institutions stronger. They need to have a framework and improve their capacity to build data tools, collect data, and collect outcomes that we need.” —Eva Maria Ruiz de Castilla, PhD
Similarly, in most Latin American countries using health outcomes and cost-effectiveness data to support health policy decisions is not always a common practice. Eva Maria Ruiz de Castilla, PhD, Regional Director for Latin America in the Global Alliance for Patient Access (GAFPA), confirms that while some policy-related decisions in countries such as Brazil and Colombia can be driven by HEOR data, it often comes from other countries that don’t necessarily have equivalent infrastructure. Ruiz de Castilla adds that “It’s complicated—some countries are implementing HTA systems and establishing HTA institutes. They are trying to collect outcomes data, but I don’t see them use this data to decide on policies. This is more political than evidence based.” And of course, these are not the only examples. Many middle- and low-income countries are still in the process of either establishing their HTA bodies, developing region-specific frameworks or working on officially incorporating cost-effectiveness and patient outcomes data into the policy decision-making process.
Barriers Are Hard to Break Down
Muñoz points out that in the Philippines, funding is a major barrier to obtaining real-world outcomes data. “In the Philippines, emphasis should be put on the research to have more data available, but it is mostly a budgetary issue and doing HEOR research is not a priority. Studies here are financed by the Department of Science and Technology and before funding HEOR studies they would still have to fund the creation of clinical and practical guidelines. If these guidelines are not available, the program will not proceed.” He also adds that a constrained healthcare budget makes it difficult to justify long-term outcomes studies and cost-effectiveness data collection for therapeutics that soon might have more effective alternatives entering the marketplace.
The Philippines, of course, is not alone in this situation. In fact, during the 2022 ISPOR HTA Roundtable in Middle East and Africa region most participants agreed that while varying levels of progress have been made towards creating a structure and guidelines for implementing HTA assessments and real-world evidence data gathering into the health policy decision making, budget remains a significant barrier.
Lack of transparency and subsequently, trust is another obstacle holding back progress. Ruiz de Castilla explained that political instability can often hinder any advances. She explains that “We have to make institutions stronger. They need to have a framework and improve their capacity to build data tools, collect data, and collect outcomes that we need.” Ruiz de Castilla adds that if the institutions are not regulated there is always lack of trust, clarity, and as a result ineffective implementation of policies.
“Payers often have similar background training. As a result, these payers may be less familiar with advanced epidemiological and data science methods such as real-world evidence.”— Don Husereau, MS
Despite the existing difficulties, it is apparent that HEOR data collection and HTAs are seen as invaluable tools and are gaining traction among decision makers. Husereau explains that from the Canadian observations, “Payers often have similar background training. As a result, these payers may be less familiar with advanced epidemiological and data science methods such as real-world evidence,” which aligns with the low- and middle-country expert observations that lack of professionals and access to training is frequently one of the major barriers to real-world data implementation. Husereau adds that 1 requirement to motivate stakeholders to push for outcomes-related data inclusion in health policy decision making includes, “requiring stakeholders to have an increased trust in new methods—to work with trusted experts who have better education and awareness. This can help stakeholders be more trustful of unfamiliar approaches.”
While competing priorities among various stakeholders impede the progress of implementing HEOR data in traditional health policy decision making, both Ruiz de Castilla and Muñoz agree that focusing on patient experience is paramount and they expect to see more public involvement in decision making in the future through patient advocacy groups. However, the work doesn’t stop at the data collection level. Even though equivalents to such programs as Surveillance, Epidemiology, and End Results Program exist in many countries, making the economic and patient outcomes assessments available in theory, there is no policy that requires to take them into account when making reimbursement and access-related decisions.
ISPOR Helps to Build HEOR Capacity
ISPOR has recognized the need for HEOR-related education and support and has created an HTA Council that works towards improving cost-effectiveness and outcomes research education access all over the world. It provides regional platforms for trainings, guidance, annual roundtables, and recommendations for countries interested in advancing their health policy decision-making capacity. With such collaborative effort, ISPOR hopes to provide a widely available platform for global knowledge sharing among various stakeholders.
About the Author
Ilze Abersone, BS, MS, is a research consultant for Vital Statistics Consulting, Hoboken, NJ, USA.
References:
1. Shrank WH, Rogstad TL, Parekh N. Waste in the United States health care system. Estimated costs and potentials for savings. JAMA. 2019;322(15):1501-1509.
2. Holtorf AP, Briner D, Bellows B, et al. Current and future use of HEOR data in healthcare decision making in the United States and in emerging markets. Am Health Drug Benefits. 2012;5(7):428-438.
3. Department of Health. The Philippines. Philippine HTA Process Guide. First Edition. Published 2020. Accessed on March 17, 2022.
