Living is More Than Surviving: Balancing Clinical Outcomes with Patient Impact

Author: Awny Farajallah, M.D., Chief Medical Officer, Global Oncology Business Unit

Over the past several decades, the rapid pace of scientific advancement has catalyzed a revolution in the way we treat cancers. This has led to a stronger focus on survival outcomes, and, in some cancers, many patients are living longer on treatment than ever before thanks to therapeutic developments such as novel targeted therapies and immunotherapy. The improvement in survival rates has created an opportunity – and obligation – to consider factors beyond just survival rates when it comes to the management of the disease.

As we continue to advance treatment options, there has been a growing movement towards prioritizing patient-centered care – considering the individual health status, preferences, values and goals of patients when making treatment decisions. Balancing clinical outcomes with the real-world experiences of patients can be complex but is necessary to provide the best possible care.

Let’s dive a little deeper into one of the reasons we’ve observed such notable improvements in the survival rates for certain cancer types – targeted therapies. Targeted therapies have revolutionized the way some cancers are treated by enabling us to target a genetic mutation that may be driving the cancer. This scientific achievement has dramatically changed the prognosis for certain types of cancers, such as breast and lung.

For example, breast cancer is the second-most common and lethal cancer type in women, and treatment options were previously limited to chemotherapies, radiation, and surgical procedures. For HER2-positive breast cancers specifically, which account for about 20% of all breast cancers, the disease is more aggressive and has a worse prognosis. The development of HER2-targeted drugs has forever changed the treatment approach and patient outcomes. In fact, some treatment combinations for this cancer extend survival to more than 4.5 years, compared to a life expectancy of 1.5 years achieved 14 years ago.¹

A similar transformation has occurred in the treatment of non-small cell lung cancer (NSCLC), another widespread cancer that has been historically difficult to treat and was previously treated with chemotherapy, radiotherapy or a combination of both. Over time, we’ve learned that about 70% of patients with NSCLC have a targetable mutation, such as EGFR, BRAF, ALK or KRAS. With improved diagnostic testing, these patients are being matched with treatments that target their cancer at its source. As a result, in recent years, population-level mortality from NSCLC has fallen sharply.²

In addition to longer survival durations, some targeted therapies have also improved upon the toxicity profiles and administration of standard chemotherapies. For example, in a study comparing neoadjuvant targeted therapy versus neoadjuvant chemotherapy in advanced-stage EGFR-mutant NSCLC, the incidence of grade 3/4 adverse events was found to be significantly less in the targeted therapy group compared to the chemotherapy group.³ Many of these targeted therapies are administered orally, providing a more convenient dosing regimen for patients.^4-10

In the 20+ years since the first targeted therapies for cancer were introduced, they have revolutionized the diagnosis and treatment of patients with breast cancer, lung cancer and many other types of cancer, and made the possibility of extending patients’ lives while improving convenience, adherence and safety, a reality.¹¹

While the rise in survival rates is an incredible accomplishment that will continue to be built upon as science advances, it has also brought about a paradigm shift in cancer care. With more cancer survivors living longer and more therapeutic options than ever before, there is greater impetus to evolve how we evaluate factors to identify the best treatment for each patient.

For example, quality of life (QoL) and method of treatment administration are important real-world factors that should be considered in addition to clinical outcomes, as they play a crucial role in the everyday lives and experiences of patients.

That said, how they are viewed and prioritized can differ by patient. For example, some patients may prioritize convenience and flexibility in treatment administration, while others may be more concerned about potential side effects or the impact of treatment on their ability to work or socialize. Some patients may prefer oral therapies that can be taken at home, while others may be comfortable with intravenous therapies administered in a clinical setting.

Evaluating these factors alongside traditional clinical standards when determining the best possible treatment ensures we are taking not only a patient’s survival into account – but also their quality of survivorship. Survivorship, in the context of cancer, refers to the journey and life experiences patients must navigate following their cancer diagnosis. Despite advancements in care over the past decade, there are still substantial gaps and challenges that need to be addressed to alleviate the short- and long-term effects of treatments – such as fatigue, cognitive problems and pain – and their impact on an individual’s survivorship and long-term disease management.

Because real-world factors and their role in survivorship are often not measured in randomized clinical trials, it’s crucial we invest in real-world evidence research and partner with patients and advocacy groups to understand how patients experience and prioritize factors such as location, family type, socioeconomic status, mental health, support systems and cultural background and how these considerations influence their decisions.

Ultimately, the goal is to provide patients with the best possible opportunity of achieving long-term survival while also maintaining a good quality of life. This requires a collaborative effort between patients, their healthcare providers, and the healthcare industry to ensure that the unique needs and preferences of each patient are considered to improve care.

While clinical outcomes will continue to be paramount, as treatment options improve, it is imperative that additional factors are considered, and patients are actively involved in the decision-making process. Considering patient preferences on factors like quality of life, treatment feasibility and emotional well-being alongside considerations on clinical efficacy can help optimize outcomes and patient experience.

By prioritizing an environment of trust, shared decision-making, and open communication, we can empower individuals to actively participate in their treatment journeys and become informed partners in their own care, which has been shown to improve their perception of quality of care and conversations with physicians.¹²

The future of cancer treatment lies in embracing an approach that considers a complete picture of patient health, recognizing not only survival as a key outcome but also incorporating the patient's overall well-being, or survivorship. By integrating real-world factors into treatment decision-making, prioritizing patient-centered care and fostering ongoing research, we can collectively transform the cancer survivorship landscape and, hopefully, empower individuals worldwide to lead fulfilling lives beyond their diagnosis.

References

1. Mendes D, Alves C, Afonso N, et al. The benefit of HER2-targeted therapies on overall survival of patients with metastatic HER2-positive breast cancer – a systematic review. Breast Cancer Res 17, 140 (2015). https://doi.org/10.1186/s13058-015-0648-2

2. Howlader H, Forjaz G, Mooradian M, et al. The Effect of Advances in Lung-Cancer Treatment on Population Mortality. N Engl J Med 2020;383:640-649. Published 2020 Aug 12. DOI: 10.1056/NEJMoa1916623

3. Chen D, Jin Z, Zhang J, et al. Efficacy and Safety of Neoadjuvant Targeted Therapy vs. Neoadjuvant Chemotherapy for Stage IIIA EGFR-Mutant Non-small Cell Lung Cancer: A Systematic Review and Meta-Analysis. Front Surg. 2021;8:715318. Published 2021 Aug 19. doi:10.3389/fsurg.2021.715318

4. Assoun S, Lemiale V, Azoulay E. Molecular targeted therapy-related life-threatening toxicity in patients with malignancies. A systematic review of published cases. Intensive Care Med. 2019;45(7):988-997. doi:10.1007/s00134-019-05650-w

5. Atrium Health. Treating Stage IV Lung Cancer With a Pill. Available at: https://atriumhealth.org/dailydose/2018/01/10/treating-stage-iv-lung-cancer-with-a-pill

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10. ALUNBRIG Prescribinig Information. Takeda. Available at: https://www.alunbrig.com/sites/default/files/2022-10/prescribing-information.pdf

11. American Cancer Society. History of Cancer Treatments: Targeted Therapy. Available at: https://www.cancer.org/cancer/understanding-cancer/history-of-cancer/cancer-treatment-targeted-therapy.html

12. Kehl KL, Landrum MB, Arora NK, et al. Association of Actual and Preferred Decision Roles With Patient-Reported Quality of Care: Shared Decision Making in Cancer Care. JAMA Oncol. 2015;1(1):50–58. doi:10.1001/jamaoncol.2014.112