Rethinking Unmet Patient Medical Needs Through the Lens of the Patient Journey

Biopharma companies are acutely aware of the importance of understanding unmet patient medical needs and how to address them with novel treatments. Today, they focus on developing novel therapeutics for diseases where the standard of care is sub-optimal or no treatments exist. According to regulators, "unmet medical needs" mean a condition for which no satisfactory method of diagnosis, prevention, or treatment exists or, even if such a method exists, to which the medicinal product concerned will be a significant therapeutic advantage to those affected. This broad definition accounts for therapeutic, quality-of-life, and societal needs, noticeable across many well-known diseases, as highlighted here:

Subbarao Jayanthi, Managing Partner, RxC International

• Better management of chronic diseases. Chronic diseases, such as diabetes, heart disease, and asthma, are the leading causes of death and disability worldwide.
• Improved therapies for neurodegenerative diseases. According to WHO, approximately 50 million people worldwide have dementia.¹ Many neurodegenerative diseases have no cure and rely on symptom management treatments.
• Personalized cancer treatments. Cancer is a leading cause of death worldwide, with an estimated 10 million deaths in 2020.² While chemotherapy and radiation therapy are effective in many cases, they can also cause significant side effects and don't work for everyone.
• Improved prevention and treatment of infectious diseases. Infectious diseases are a leading cause of death worldwide. However, resistance is a growing problem, making treating infections more difficult with existing antibiotics.
• Better management of mental health conditions. Mental health conditions are a leading cause of disability worldwide, affecting an estimated 1 in 4 people at some point.³ However, existing therapies, such as antidepressants and antipsychotics, may not work for everyone or have serious side effects.
• Better treatments for rare diseases: Rare diseases affect an estimated 400 million people worldwide, but many have no cure and limited treatment options.⁴
• Better treatments for pain and chronic pain: Chronic pain affects an estimated 20% of adults worldwide, which impacts the quality of life, mental health, and overall functioning.⁵ However, existing treatments, such as opioids, can have serious side effects associated with addiction and overdose.

A high disease burden leads to poor health, disability, mortality, or economic burden. Understanding these unmet medical needs is imperative to develop appropriate medical and non-medical interventions. Potential solutions include diagnostics, drugs, devices, monitoring technologies, and patient engagement solutions. Life sciences companies are uniquely positioned to address medical and non-medical unmet needs to impact patients' health and quality of life.

Even with the continued advancements in science and technology, the unmet needs persist across diseases due to various factors. The primary reason is that we still don't understand the root causes of many diseases. The unmet needs present in different forms: (a) treatments do not exist; (b) treatments exist but are inadequate or ineffective; (c) treatments exist, but delivery mechanisms or formulations are inadequate; and (d) treatments alone may not be sufficient to produce optimal health outcomes. More recently, some essential medications have been in either short supply (e.g., insulin) or underutilized (e.g., opioid addiction treatments) for several reasons, even though the impact on public health is significant.

Acute vs. chronic diseases

LizAnn Sung, Management Consultant, RxC International

In acute conditions, the goal is to find an optimal medical intervention to get the patient to their original state. However, treatment resistance is common, which is the case in many infectious diseases, and novel treatments may be needed to address this resistance. Acute conditions may also recur and often require preventive treatment. For instance, a serious, acute bacterial infection may begin with a strong intravenous antibiotic. If the patient begins to improve, they may be "stepped down" to an oral formulation of an antibiotic. Conversely, if the patient shows resistance to the antibiotic, there is a need for a novel antibiotic to address the infection instead.

In chronic conditions, once the disease is controlled with appropriate medications, the treatment focus shifts to day-to-day issues, such as managing side effects, co-morbidities, and quality of life. This is the case for human immunodeficiency virus (HIV), for which clear diagnostic tools and many efficacious drug regimens are available to patients. As such, the greater concern for HIV patients today has shifted to managing co-morbidities and quality of life. Prophylactic treatments are also available for HIV to prevent the onset of the disease. Patients’ range of disease management issues tends to be relatively consistent across chronic diseases. These issues often are multi-factorial issues in which medication alone may not be sufficient to produce optimal health outcomes.

Today, many diseases have been turned into chronic diseases. In addition to medical interventions, patient engagement and connectivity with social groups have delivered compelling health outcomes. An example of this would be chronic myeloid leukemia (CML). While diagnosis is difficult, multiple efficacious therapies manage the disease well. However, disease maintenance in the “new normal” involves drug compliance, close monitoring, and peer-to-peer engagement.

Copyright 2022 RxC International LLC.

Copyright 2022 RxC International LLC.

Uncovering unmet patient needs

Biopharma companies can uncover unmet patient needs across diseases by closely monitoring each stage of the patient journey. This process should account for patient characteristics and socio-economic factors to understand the patient's unmet needs. The following considerations must be taken into account when assessing each phase of the patient journey, spanning from disease prevention and diagnosis to the initiation, optimization, and maintenance of treatment. Those considerations are:

• Disease prevention. Though prevention is the best cure for any disease, preventing many known diseases is impractical. The primary reason is a lack of understanding of disease biology and pathophysiology. However, every attempt should be made to address the prevention or delay of the onset of diseases.
• Disease diagnosis. Some of the most common diseases can be diagnosed with accurate biomarkers (e.g., A1C levels for diabetes patients). Advanced genomic tests allow companies to understand various mutations causing cancers. However, many diseases remain undiagnosed or underdiagnosed, and the lack of biomarkers makes the goal of offering personalized treatments more challenging.
• Treatment initiation and optimization. General treatment guidelines exist for common diseases but often don't account for patient characteristics and co-morbidities. Treatment optimization is challenging in many common conditions where objective biomarkers don't exist.
• Treatment maintenance. In this phase, effective disease management entails ensuring patient adherence to treatment and conducting regular screenings and monitoring of outcomes. Additionally, it is essential to consider other factors that could impact the patient's physical and psychological well-being, societal support, and financial burden.

Stakeholder collaboration

Biopharma companies must collaborate with various healthcare system stakeholders to address patient needs meaningfully. Although the stakeholders broadly agree on addressing unmet medical needs and minimizing patient burden, they often don't align, given the varied economic incentives. At a simplistic level, patients seek optimal solutions to reduce disease burden and disruption to daily activities. Providers address patients' medical needs while striving for the best health outcomes. However, they face challenges ranging from the inability to use the proper medications to engaging patients to improve health outcomes.

The policymakers often account for the disease burden, the size of the impacted patient population, and competing public health priorities in their investment decisions. Payers focus on managing their patient pools cost-effectively and prioritize treatment coverage based on potential benefits vs. health economics. The so-called "fair distribution of resources" paradigm often stalls due to the complex interplay between political and economic priorities. Inertia also plays a role. For example, payers and clinical personnel may not feel compelled to opt for solutions that, in their opinion, can only move the needle incrementally over the existing standard of care.

Patient-centricity, differentiation, and access

Biopharma companies should gain a deeper understanding of patient unmet needs to drive innovation. A patient-centric approach is critical to optimize R&D programs while accounting for stakeholder preferences to lay the pathway for better access to solutions. For example, payers and clinical assessors may not wish to pay for a minor, incremental enhancement in outcomes over existing standards of care. As the standard of care continues to evolve rapidly across many diseases driven by innovations, one of the most challenging issues to confront is product differentiation across all stages of development.

Diseases characterized by high mortality rates, substantial disease burden (including frequency and cost of hospitalization), and widespread impact on large populations will persistently garner increased attention due to their economically attractive nature. Implementing the Orphan Drug Designation Act has facilitated the efforts of biopharmaceutical companies to address numerous rare diseases with significant unmet medical needs. Biopharma companies are uniquely positioned to tackle many of these unmet needs while collaborating with stakeholders to ensure access.

References

1. WHO, “Dementia,” https://www.who.int/health-topics/dementia#tab=tab_1 (accessed March 2023).
2. WHO, “Cancer,” https://www.who.int/news-room/fact-sheets/detail/cancer (accessed March 2023).
3. Children’s Hope Chest, “Global Mental Health Statistics,” https://www.hopechest.org/global-mental-health-statistics/ (accessed March 2023).
4. Khoury, M. J. Rare Disease Day 2022: The Evolving Impact of Genomics and Precision Health. CDC Blog. 2022. https://blogs.cdc.gov/genomics/2022/02/28/rare-disease-day-2022/ (accessed March 2023).
5. Goldberg, D. S.; McGee, S. J. Pain as a Global Public health priority. BMC Public Health. 2011, 11, 770. DOI: 10.1186/1471-2458-11-770 (accessed March 2023).

About the authors

Subbarao Jayanthi is a managing partner for RxC International.

LizAnn Sung is a management consultant for RxC International.