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  • Advancing Health Equity through Health Economics and Outcomes Research

    Advancing Health Equity through Health Economics and Outcomes Research

    In a world where access to healthcare should be a universal right, the persistent disparities in health outcomes starkly remind us that health equity remains an elusive goal. Health equity is defined as the absence of unfair and avoidable differences in health among population groups defined socially, economically, demographically, or geographically. This concept underscores the need to ensure that every individual has a fair opportunity to achieve their highest possible level of health, unimpeded by systemic barriers or socioeconomic disadvantages.[1]

    Health Economics and Outcomes Research (HEOR) examines the cost-effectiveness, value, and real-world outcomes of healthcare interventions, enabling data-driven resource allocation. HEOR is pivotal in advancing health equity by analyzing the economic and clinical outcomes of healthcare interventions. HEOR evaluates the value of medical treatments and services, providing data-driven insights that inform decisions about the allocation of healthcare resources. By intertwining health equity with health economics, HEOR helps identify which interventions deliver the most significant benefits across diverse populations, guiding efforts to reduce disparities and improve overall health outcomes.[2]

    The COVID-19 pandemic showed how social determinants of health, such as income, occupation, and access to technology, impact health equity. Higher-income individuals often had the advantage of working from home, reducing their exposure to the virus and benefiting from better internet connectivity, which enabled access to virtual healthcare. In contrast, essential workers in lower-paying jobs faced greater exposure and limited healthcare access, leading to worse outcomes. These disparities highlight the urgent need to reevaluate healthcare practices and structures to address inequities effectively.[3-5]

    HEOR plays a critical role in this re-evaluation by incorporating cost-effectiveness analysis (CEA) to determine which healthcare interventions provide the most value. CEA can also include equity-weighted analyses that prioritize interventions benefitting disadvantaged populations. CEA compares the costs and outcomes of various strategies, enabling policymakers to allocate resources efficiently, especially in settings with limited healthcare budgets. For example, analyzing cancer screening programs’ cost-effectiveness can identify the most beneficial approach for underserved communities, ensuring that resources are directed where they can achieve the greatest impact.[6]

    Furthermore, HEOR’s focus on real-world evidence (RWE) extends beyond controlled clinical trials to understand how treatments perform in everyday practice. This evidence is essential for addressing health disparities, as it reflects the diverse experiences of different patient populations. For instance, RWE during the pandemic showed how vaccine hesitancy and limited access impacted vaccination rates in underserved communities, guiding targeted outreach campaigns. Studies might show that a particular medication is less effective in certain ethnic groups due to genetic variations or disparities in healthcare access, prompting the development of tailored strategies to improve outcomes for these groups.[7]

    Patient-reported outcomes (PROs) and electronic PROs (ePROs) are vital components of HEOR that enhance health equity by capturing patients’ perspectives on their health status, quality of life, and treatment satisfaction. Incorporating PROs and ePROs ensures that the voices of all patients, including those from marginalized groups, are considered in healthcare decision-making. This approach helps uncover specific challenges faced by different populations and informs the design of more inclusive and effective interventions.[8]

    HEOR also influences policy decisions by providing evidence on the broader social determinants of health, such as education, housing, and employment. Highlighting the impact of these factors on health outcomes allows HEOR to advocate for integrated policies that address these root causes of inequity. For instance, research showing that stable housing reduces emergency room visits has prompted healthcare systems to invest in housing assistance programs. In addition, research might demonstrate that improving access to quality education and stable housing can lead to better health outcomes and reduced healthcare costs, supporting the case for holistic approaches that combine social and health policies.[5]

    The pharmaceutical industry benefits significantly from integrating health equity into HEOR. By including diverse populations in clinical trials and subsequent research, companies can develop treatments that are effective across different demographic groups. Failing to include diverse populations can lead to treatments that are less effective or even harmful for certain groups, underscoring the importance of inclusive research practices. This approach not only improves health outcomes but also enhances market access and ensures compliance with regulatory requirements focused on diversity and inclusion.[7]

    In conclusion, advancing health equity through HEOR is both a moral imperative and a strategic necessity for optimizing healthcare delivery and outcomes. By embedding equity considerations into economic evaluations, RWE, PROs, and policy research, HEOR can guide the development of more inclusive healthcare strategies. This comprehensive approach ensures that all individuals, irrespective of their background, have access to the care they need to lead healthy and fulfilling lives. Moving forward, it is essential for all healthcare stakeholders to prioritize health equity in their research and decision-making processes, paving the way for a fairer and healthier future for all.

    Become A Certified HEOR Professional – Enrol yourself here!

    References:

    1. Health equity. World Health Organisation. Available from: https://www.who.int/health-topics/health-equity#tab=tab_1
    2. Fautrel B. SP0124 Health economics and health equity: two complementary disciplines.2017;76:31.
    3. Closing the Gap in a Generation: Health Equity Through Action on the Social Determinants of Health. Final Report of the Commission on Social Determinants of Health. World Health Organization; 2008:1 -256. Available from: https://apps.who.int/iris/bitstream/handle/10665/43943/9789241563703_eng.pdf;jsessionid=365271ACE2052888542881700EEDCA8B?sequence=1.
    4. Burström B, Tao W. Social determinants of health and inequalities in COVID-19. European journal of public health. 2020 Aug 1;30(4):617-8.
    5. Thomas R, Chalkidou K. Cost–effectiveness analysis. InHealth system efficiency: How to make measurement matter for policy and management. 2016. European Observatory on Health Systems and Policies.
    6. Fendrick AM, ISPOR. Real-World Evidence: Additional Tool to Support Clinical Decision Making. Available from: https://www.ispor.org/docs/default-source/strategic-initiatives/ispor-rwe-byline-article_10-25-21.pdf?sfvrsn=687e4bc8_0
    7. Rosenberg SS, Carson BB, Kang A, Lee TH, Pandey R, Rizzo EJ. The Impact of Digital Health Technologies on Health Equity: Designing Research to Capture Patient-Reported Outcomes. ISPOR value & outcomes spotlight. Available from: https://www.ispor.org/publications/journals/value-outcomes-spotlight/vos-archives/issue/view/addressing-assessment-and-access-issues-for-rare-diseases/the-impact-of-digital-health-technologies-on-health-equity-designing-research-to-capture-patient-reported-outcomes.

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  • Patient Preference Information (PPI) in Improving Patient-centricity of Healthcare

    Patient Preference Information (PPI) in Improving Patient-centricity of Healthcare

    Patient Preference Information (PPI) has emerged as a powerful tool, gaining recognition in the healthcare industry for its multifaceted ability to enhance patient-centered care, refine clinical trial design, and inform crucial treatment decisions. The drive towards patient-centered care has become a hallmark of modern healthcare, emphasizing the need to tailor healthcare decisions and practices according to patients’ values and preferences. In this context, PPI assumes a pivotal role by providing invaluable insights into patients’ authentic desires and requirements. Furthermore, PPI has the potential to revolutionize clinical research and treatment strategies by actively incorporating the patient’s perspective into the decision-making process.[1]

    The advantages of incorporating PPI into clinical research are manifold, including enhanced patient recruitment and retention, refined trial design, and elevated patient satisfaction. Clinical trials stand as the bedrock of advancing medical knowledge and ushering in new treatments. However, traditional RCTs are often criticized as being physician-centric, research-oriented, and being executed in a ‘controlled setting’ without much importance to patient preferences.[2] The integration of PPI into clinical trial design can potentially improve the patient-centricity of the research [3]. For example, in a clinical trial, patients may be given the choice between two treatment regimens with differing efficacy and side effect profiles: hypothetically, patients can be asked to select either an option with a lower efficacy but better safety, or an alternative with a higher efficacy but not-yet-completely-known safety profile. Based on these preferences, randomization can be carried out: a design sometimes referred to as ‘preference-based randomization’. This ensures that the selected treatment aligns closely with the patient’s preferences and lifestyle.[4] Such preference-based randomization allows patients to articulate their treatment preferences, enabling researchers to allocate treatments accordingly. This approach not only enhances patient engagement but also holds the potential to improve treatment adherence, thereby yielding more accurate outcomes.[1-3]

    PPI extends its influence beyond the confines of clinical trials, exerting a substantial impact on the daily landscape of patient care. Central to the concept of patient-centered care is the notion of treating the patient as an active and engaged participant in their healthcare journey. In this context, PPI serves as a vital connective bridge that facilitates seamless communication and collaboration between healthcare providers and patients, bridging the gap between medical expertise and individual values and choices.[1] PPI equips healthcare providers with profound insights into patients’ values, preferences, and goals, facilitating the creation of personalized care plans that harmonize with the patient’s desires. For instance, an older patient facing a terminal illness may prioritize the quality of life over aggressive treatments: accordingly, PPI can steer the development of a care plan that honors this preference.[1,2]

    On the other end of the table, PPIs can also empower patients. The PPI gathering process can make acquaint the patient with comprehensive information regarding their treatment options and possible outcomes, making them more inclined to actively partake in decisions regarding their care, thus championing shared decision-making and fostering enhanced collaboration between patients and healthcare providers to make well-informed choices. For instance, a patient diagnosed with breast cancer may harbor strong preferences concerning the timing of surgery. Here, PPI can guide discussions regarding the appropriate treatment timeline. This collaborative approach often translates into enhanced treatment adherence and heightened patient satisfaction.[1,2]

    Regulatory agencies have come to recognize the pivotal role of PPI in healthcare decision-making. These agencies are increasingly incorporating PPI into their evaluations of novel drugs and medical devices. For instance, the USFDA developed a set of guidance in 2020 regarding the collection and use of PPI in regulatory decision-making, underscoring its significance in benefit-risk assessments. Similarly, the European Medicines Agency (EMA) is actively exploring the incorporation of patient preferences in benefit-risk assessments. Additionally, Health Canada has initiated the Patient and Public Engagement Initiative, involving patients in drug approval processes. The Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK values patient input and engagement in its regulatory activities, while the Therapeutic Goods Administration (TGA) in Australia is incorporating patients’ perspectives, particularly in medical device approvals. This information informs regulatory decisions and ensures that healthcare interventions align with patient preferences and expectations.[5-11]

    All said, incorporating PPI into clinical research presents several significant challenges. First and foremost is the need for standardization in the collection and analysis of PPI, as patient preferences can vary widely across different contexts. Ensuring that the preferences of trial participants accurately represent the broader patient population is another challenge, as volunteer biases can skew data. Moreover, using PPI for randomization in RCTs may introduce bias, as it could lead to a non-representative sample if patients with strong preferences self-select into certain treatment arms. Ethical considerations, resource intensiveness, integration with regulatory requirements, interpreting and applying PPI data, patient education, and engagement are all practical challenges that must be addressed. Finally, achieving regulatory acceptance and standardization of PPI methods and findings across regions and healthcare systems remains a critical hurdle.[12]

    The use of PPIs in routine clinical practice is associated with a distinct set of challenges. These include the seamless integration of PPI data into electronic health records and clinical workflows, the importance of upholding patient privacy and securing informed consent for its use. Other complexities include the need for proper interpretation and application of PPI data in individual patient care decisions and the requirement for training healthcare providers to communicate and utilize PPI effectively. Additionally, allocating adequate time and resources in busy clinical settings, fostering patient engagement in sharing their preferences, and developing standardized methods for collecting also pose complications to the use of PPIs in healthcare. Overcoming these challenges necessitates collaboration among stakeholders to harness the potential benefits of PPI in enhancing the relevance and patient-centeredness of clinical research and healthcare decision-making.[12]

    In conclusion, PPI is an indispensable and versatile tool in healthcare, both in research and routine healthcare delivery, as indicated by the growing acknowledgment of PPIs by regulatory agencies. Its manifold applications encompass the enhancement of clinical trial design, the augmentation of patient-centered care, and the facilitation of treatment selection, all fostering an enhancement of patient-centered care and medical research. PPI empowers patients to occupy a central role in their healthcare decisions, ultimately culminating in improved patient outcomes and heightened patient satisfaction.

    Become A Certified HEOR Professional – Enrol yourself here!

    References

    1. McPherson K, Chalmers I. Incorporating patient preferences into clinical trials. Information about patients’ preference must be obtained first. BMJ. 1998 Jul 4;317(7150):78; author reply 78-9.
    2. Wasmann KA, Wijsman P, van Dieren S, Bemelman W, Buskens C. Partially randomised patient preference trials as an alternative design to randomised controlled trials: systematic review and meta-analyses. BMJ Open. 2019 Oct 16;9(10):e031151.
    3. Lambert MF, Wood J. Incorporating patient preferences into randomized trials. Journal of clinical epidemiology. 2000 Feb 1;53(2):163-6.
    4. Kowalski CJ, Mrdjenovich AJ. Patient preference clinical trials: why and when they will sometimes be preferred. Perspectives in biology and medicine. 2013;56(1):18-35.
    5. Sharma NS. Patient centric approach for clinical trials: Current trend and new opportunities. Perspect Clin Res. 2015 Jul-Sep;6(3):134-8.
    6. Marshall, Deborah A. “Brief Overview of Patient Preference Information (PPI).” FDA Committee. PowerPoint Presentation. March 2021. https://www.fda.gov/media/146925/download.
    7. Irony T, Ho M, Christopher S, Levitan B. Incorporating patient preferences into medical device benefit-risk assessments. Statistics in Biopharmaceutical Research. 2016 Jul 2;8(3):230-6.
    8. Mühlbacher AC, Juhnke C, Beyer AR, Garner S. Patient-focused benefit-risk analysis to inform regulatory decisions: the European Union perspective. Value in Health. 2016 Sep 1;19(6):734-40.
    9. Weeks L, Polisena J, Scott AM, Holtorf AP, Staniszewska S, Facey K. Evaluation of patient and public involvement initiatives in health technology assessment: a survey of international agencies. International journal of technology assessment in health care. 2017;33(6):715-23.
    10. Aiyegbusi OL, Cruz Rivera S, Oliver K, Manna E, Collis P, King-Kallimanis BL, Bhatnagar V, Herold R, Hopkins J, Campbell L, Croker A. The opportunity for greater patient and public involvement and engagement in drug development and regulation. Nature Reviews Drug Discovery. 2023 May;22(5):337-8.
    11. Russell TG, Jones AF. Implications of regulatory requirements for smartphones, gaming consoles and other devices. Journal of physiotherapy. 2011 Jan 1;57(1):5-7.
    12. Selman LE, Clement C, Douglas M, et al. Patient and public involvement in randomised clinical trials: a mixed-methods study of a clinical trials unit to identify good practice, barriers and facilitators. Trials. 2021 Dec;22:1-4.

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  • Patient Preference Information (PPI) in Healthcare:  What is it, and How is it Collected?

    Patient Preference Information (PPI) in Healthcare:  What is it, and How is it Collected?

    In recent years, healthcare has undergone a profound transformation, shifting its focus from traditional, clinician-centric models toward patient-centered care. This shift recognizes that healthcare decisions should not solely revolve around clinical outcomes but should also consider the patient’s values, preferences, and goals. At the heart of this transformation lies the concept of Patient Preference Information (PPI), a critical component that encompasses a wide range of factors extending beyond clinical outcomes.[1]

    The factors usually considered under PPI include treatment modalities, potential side effects, the impact on a patient’s quality of life, and the collaborative process of shared decision-making with healthcare providers. Recognizing and effectively integrating patient preferences into the decision-making process is instrumental in empowering individuals to actively engage in their healthcare journey. This, in turn, enhances overall patient satisfaction and adherence to treatment plans, leading to significant improvements in treatment outcomes.[1]

    PPI is related to, but is distinct from another familiar concept in patient-centric healthcare, namely the Patient-Reported Outcomes (PROs). On the one hand, PROs focus on assessing a patient’s subjective health status and quality of life after treatment administration; they are reported directly by the patient, and are thus crucial in evaluating the effectiveness of the administered treatment from the patient’s viewpoint. On the other hand, PPI delves into the patient’s preferences and values regarding various treatment options available at his/ her disposal. It aims to identify the attributes or characteristics of healthcare interventions that matter most to patients, ranging from route of administration, frequency of administration, place of treatment delivery, treatment convenience, cost, measurement of outcomes, and so on. To put things in context, a treatment plan aligned with a patient’s preferences (that is, following PPI) is more likely to be followed by patients diligently, leading to improved overall well-being and improved treatment outcomes, that is, PROs as well as physician-reported objective outcomes.[1-3]

    For instance, patients with osteoporosis might have a distinct set of preferences revolving around attributes such as route of administration, cost, dosing frequency, and risk-benefit ratio. Many patients tend to favor treatments like 6-month subcutaneous injections or monthly oral tablets while actively avoiding gastrointestinal side effects. This wealth of preference data forms the foundation of PPI. In this context, PROs focus on evaluating treatment outcomes from a patient’s viewpoint, in terms of overall quality of life, symptom relief, pain-free days, treatment satisfaction, and medication adherence.[4]

    PPIs can be measured as a part of traditional RCTs, or as a dedicated study with the sole objective of measuring PPIs; the latter study designs are often termed ‘PPI trials’. These PPI trials often involve open-ended interviews that allow patients to express their experiences and articulate the aspects of treatment that hold the most meaning for them.[1,5]

    PPI estimation can be qualitative (what do you prefer?) or quantitative (how much do you prefer?). While qualitative methods for PPI estimation often involve ranking of options based on preferences, quantitative methods involve detailed interviews to quantify patient preferences in difficult situations using different evolving techniques. Some of these techniques are conjoint analysis (which allows patients to rank or rate hypothetical scenarios, deriving utility values and determining attribute importance), Best-Worst Scaling (that enables patients to identify attribute preferences by choosing between the best and worst aspects of treatment options), Time Trade-Off (TTO) and Standard Gamble techniques (that involve estimating trade-offs between treatment effectiveness and uncertainty), and so on. These quantitative approaches collectively enhance the evidence base for patient-centered healthcare decisions, resulting in more tailored and effective interventions.[1,5]

    Collecting PPI data demands ethical considerations, informed consent, and strict privacy protection. Diverse patient populations must be included to accurately represent patient views. Advances in digital health technology, including wearables and health-centric apps, facilitate real-time PPI data collection. This data guides treatment decisions and addresses health disparities by considering underserved populations’ preferences, leading to culturally sensitive interventions.[5]

    The applications of PPI collected through diverse methodologies can include facilitating treatment choices, improving clinical trial design, informing regulatory decisions, and enhancing shared decision-making. By valuing and integrating patient preferences, healthcare revolves around individual needs, fostering a patient-centric future.

    Become A Certified HEOR Professional – Enrol yourself here!

    References

    1. Deborah A Marshall. Brief Overview of Patient Preference Information (PPI). FDA Committee. Power Point Presentation. 2021 Mar.
    2. Rincon-Gonzalez L, Selig WK, et al. Leveraging Patient Preference Information in Medical Device Clinical Trial Design. Therapeutic Innovation & Regulatory Science. 2023 Jan;57(1):152-9.
    3. Chachoua L, Dabbous M, François C, et al. Use of Patient Preference Information in Benefit-Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives. Front Med (Lausanne). 2020 Oct 26;7:543046.
    4. Reaney M, Rodriguez AM. Patient Experience Information: Streamlining and Harmonizing the Collection of Patient Preference and Patient-Reported Outcomes Data. Pharmaceut Med. 2020 Oct;34(5):309-314.
    5. Medical Device Innovation Consortium. Medical device innovation consortium (MDIC) patient centered benefit-risk project report: a framework for incorporating information on patient preferences regarding benefit and risk into regulatory assessments of new medical technology. Med Dev Innov Consortium. 2015 Dec 27;4:e34.

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