Introduction
Drug development is an increasingly challenging process due to rising costs, strict regulatory requirements, a demanding market access environment, and high research and development attrition rates.1,2 New treatments should simultaneously improve patients’ quality of life while reducing overall healthcare costs.3 To facilitate the development of novel therapeutics that significantly improve quality of life over existing available treatments, the involvement of patient stakeholders (patients, families, caregivers, and advocacy organizations) as team members in the drug development process is becoming increasingly common in the pharmaceutical industry. Many regulatory agencies and associated committees and initiatives, like the European Medicines Agency (EMA), Scientific Advice Working Party (SAWP), Patient Engagement Advisory Committee (PEAC), Patient-Focused Drug Development (PFDD), and the United States Food and Drug Administration (FDA) have acknowledged the importance of patient-centric drug development.4–7 Furthermore, funding agencies and journal editors have recently begun to endorse patient engagement in patient-oriented research prior to funding drug development and publications.
Engagement of patients across all phases of the drug development life cycle and systematic inclusion enables patients to be deeply involved in the interactions between advocacy organizations and bio-pharmaceutical companies to better understand and capture patient experience, burden of illness, and unmet clinical needs. Additionally, studies that incorporate accountability into the research process and empower patients to take on team-oriented roles re-define patient and stakeholder engagement by ensuring patients’ needs are understood early in drug development and incorporated into research priorities. Patient-focused medicines development includes consistently informing drug research, expanding access and awareness of clinical trial opportunities, improving clinical study design and outcomes, supporting a shared decision-making model, understanding tradeoffs of benefit/risk scenarios, and co-creation of meaningful and culturally appropriate study materials.8 Patient-focused clinical study design can also expand trial access to a pool of diverse patients who may consider clinical trials as a potential care option.9 Sub-optimal engagement can result in patients perceiving that clinical trial design is driven by “checking off boxes” rather than having a real mutual partnership.10–12
In asthma, drug development and treatment are particularly challenging because of the complex and heterogeneous nature of the disease, which presents with varying clinical symptoms and treatment responses. Asthma triggers, response to medications, effectiveness of treatments, and asthma severity and exacerbations may vary from patient to patient and over the course of their disease.13 Hence, mutual collaboration between researchers and patients is instrumental to facilitate drug discovery and development. Moreover, collaborating with patients early in drug discovery process is crucial to design drugs that meet their unmet needs. Sanofi, a global biopharmaceutical company, and Allergy and Asthma Network (AAN), a US-based patient advocacy group, collaborated with patients with asthma from AAN’s patient community to better understand the challenges they face in the participation of clinical trials, medication adherence, their unmet medical needs, and the burdens impacting their quality of life.14 Engendering respect, trust, honesty, integrity, and accountability are tied to the mutual mission of putting patients first as key participants in the research process. When researchers understand patient needs and perspectives, they are better equipped to align their research efforts and decision-making structures to an integrated, patient-centric approach that is relevant to basic drug discovery.15 Pharmaceutical companies collaborating with patients living with asthma understand patient and caregiver lived experiences and can advance meaningful patient-centric research and development. The insights from these collaborations allowed Sanofi to better align research objectives with patients’ unmet medical needs, design better clinical studies to be patient-friendly and accessible, and ensured that the data generated in these studies reflect patients’ true health priorities.
To advance patient-centric drug discovery and development in asthma, Sanofi, AAN, and members of AAN’s asthma patient community worked together through multiple structured interviews to better understand their perspectives and gaps in current asthma treatments, with an aim of accelerating the development of future therapies for asthma.
Materials and Methods
Literature Review
A literature search was conducted using Medline® for English language articles published between July 2013 and July 2023. Published full-text articles regarding patient needs and expectations related to asthma treatment and patient-centric drug development in asthma were hand searched and included.
Stakeholder Input
To explore the unmet needs of patients living with asthma and understand their perspectives to guide future treatments, several virtual patient panels between patient advocates (n=5) and researchers (n=5) from Sanofi were conducted. All patients were contacted through the patient advocacy group AAN. They were selected based on their symptoms, disease severity, and treatment history. The selection was in alignment with the “Global Initiative for Asthma (GINA) clinical criteria for diagnosing asthma” to give a diverse perspective based on varying severity, and to reflect the diversity of lived experiences across geographies.16 A total of four virtual interviews were conducted with the patients, each of approximately 60 to 90 minutes in duration. The initial interviews included structured questions (Table 1), followed by open dialogues/transparent communication with patients about their experiences with asthma, treatment options, gaps, needs, and priorities.
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Table 1 Questions Used in the Patient Interview
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Results
Results from the Virtual Interview Panels
The interview panel included five patients of whom four had moderate-severe asthma as per the GINA clinical criteria and predominantly consisted of female patients (n=4/5) (Table 2).16
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Table 2 Patient Characteristics
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Unmet Needs of Patients with Asthma
Based on patient panels led by advocates from the AAN, insights into patient experiences with asthma, unmet needs, priorities, adherence factors, treatment options, and treatment detractors were better understood. Key factors affecting asthma treatment adherence, gathered from patient input were side effects, lack of information about correct inhaler techniques, medication costs, misunderstanding medication dosing schedules, lack of communication between researchers and patients, lack of awareness about patient advocacy organizations and research led programs, and fear of discrimination or having concerns disregarded by healthcare providers (Table 3).
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Table 3 Key Factors Affecting Asthma Treatment Adherence
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Takeaways from Patient Interviews and Adopting a Patient-Centric Approach as a Way Forward
The discussion with patient advocates emphasized that researchers must consider patients as partners to guide the decision-making processes and enable patient-centric principles across asthma research and treatment (Figures 1 and 2). Patient advocates have indicated that currently marketed medications have limitations, which may impede treatment adherence, and still do not achieve adequate asthma control. Of note, patients expect that new treatments should ease patient burden and treat the multiplicity of asthma symptoms, including the reduction of inflammation, relaxation of airways, and a reduction in response to asthma triggers with one treatment. For patients living with asthma, an ideal treatment would not require daily administration, must effectively control asthma symptoms within the least amount of elapsed time, and feature monthly dosing to eliminate the need for reliever inhalers or nebulizers in public. Furthermore, asthma medications must be cost-effective for all patients, regardless of their insurance status or coverage.
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Figure 1 Patient feedbacks.
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Figure 2 Takeaways from patient interviews.
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Discussion
In this study, Sanofi collaborated with AAN, a patient advocacy group, to conduct interviews among patient stakeholders who have asthma to understand their perspectives about the disease and gaps in current asthma treatments, with an aim of developing novel and efficacious therapies. The patient stakeholders gave their inputs on factors affecting treatment adherence, unmet needs, and expectations for future treatments.
Understanding patients’ unmet needs, their perspectives about current treatments, expectations from future treatments and challenges about participation in clinical trials is essential for the development of novel and efficacious drugs.1,2 The importance of involving patients early in drug development is emphasized by many regulatory agencies, funding agencies, and journal editors.4–7 Patient-focused drug development is particularly important in asthma because of the complex and heterogeneous nature of the disease.13
In addition to patient interviews, evidence from published literature informed researchers at Sanofi about objective measures to link to prioritization decisions and the selection of novel drug targets and new research programs in asthma. It also helped researchers understand the unmet burden of disease, preferred formulations and modes of administration, biomarker and patient readiness considerations, standards of competing care (patient preference for differentiation), preferred therapies in the market (and gaps), adherence influencers, and patient burden from participation in clinical trials, among other factors.
Expanding patient access to asthma clinical trial opportunities may also serve as a gateway to novel therapies for patients who otherwise may not have access to effectively manage their asthma and achieve a better health-related quality of life.17 Such opportunities can be difficult for patients who may not be aware of the availability of medical information, the rise of patient advocacy organizations, or the paradigm shift towards value-based healthcare.18 Enabling an ecosystem that integrates patients at each layer may help to decrease barriers, broaden access to clinical trials, and garner visibility on trial data and results in a patient-friendly manner.19
The COVID-19 pandemic has also accelerated the pathway to new care settings, including digital technologies, which have the potential to help patients manage their asthma across different points in the care journey.20 These include digital applications and tools for maintaining a healthy lifestyle and monitoring tools with reminders to help patients take their medications on schedule while incorporating applications to track triggers (e.g., weather, air quality index, pollen, etc) moods, sleep, peak flow recordings, and nutrition; all of which have the potential to improve patients’ treatment adherence, quality of life, and health outcomes.21,22 Additionally, it would also be helpful to design broader symptom scales that collect data over a more extended period (e.g., weekly, monthly) as opposed to daily reporting linked to a numeric rating scale that evaluates asthma symptoms or trigger severity.23
Developing opportunities for greater patient engagement, such as patient panels and advisory boards with patient advocacy groups can re-define patient and stakeholder engagement while providing patient-focused intelligence to inform, support, and facilitate product development.24 The cohesiveness of such relationships will allow more patients the opportunity to share their experiences and journeys and unlock greater health equity through meaningful interactions.25 Educating patients on treatment costs, including medication support programs, companion diagnostics, and routine examinations when at their healthcare provider’s office is advisable.26,27 Providing medication samples or valved-holding chambers/spacers for inhalers to people who can use them may also help to defray out-of-pocket costs. Increasing health literacy among patients with asthma also remains a crucial part of asthma management including achieving optimal symptom control.28 Other opportunities may include the development of a patient research partner course where the patient would learn about the expectations of participating in research with a research peer, creating a network of educated patients, and identifying other opportunities for researchers to engage patients. This will allow researchers and patients to partner to develop different types of studies.29
Conclusion
Asthma is a heterogeneous disease with each patient having their own unique treatment needs. As researchers may not often interact with patients living with asthma, a greater awareness and understanding of the needs and preferences of patients while focused on delivering personalized care, tailored to the needs of each patient, is required. Patient advocacy groups can bridge the conceptual gap between biopharmaceutical companies and patients to gather insights that emphasize patient experiences. Based on the input received from patient panels, the key factors affecting asthma treatment adherence are: medication side effects and cost, lack of knowledge among patients about correct inhaler techniques, dosing, dosing schedules, and lack of communication with advocacy groups and researchers. To overcome these limitations, patients suggested that new treatments could involve a single drug which reduces inflammation, relaxes airways and reduces response to triggers. Patients also suggested the drug be quick-acting and need not be taken daily to avoid the use of inhalers in public. The patient perspectives gleaned from this pilot study and previous research done on the topic would be valuable for researchers to design a novel drug to treat asthma and address patient unmet needs; however, further research involving more patients across different countries was deemed warranted. Sanofi has continued similar research across all disease areas.
Abbreviations
AAN, Allergy & Asthma Network; EMA, European Medicines Agency; FDA, Food and Drug Administration; PEAC, Patient Engagement Advisory Committee; PFDD, Patient-Focused Drug Development; SAWP, Scientific Advice Working Party.
Ethics Approval and Informed Consent
The study included a series of interviews and a workshop, hence there was no approval required from ethics committee. Informed consent and permission to publish results were obtained from all individual participants included in the study. The participants also provided consent to publish anonymized responses/direct quotes. The study was conducted in accordance with European Pharmaceutical Market Research Association (EPHMRA) guidelines.
Consent for Publication
All authors consented to publish the manuscript.
Acknowledgments
The medical writing and editorial assistance were provided by Rahul Nikam and Deepti Sharda from Sanofi. The authors would like to thank the patient advocates and advocacy organization (Allergy & Asthma Network) who participated in the interviews.
Author Contributions
AH, MJG, and DDG conceived, designed, and executed the study and acquired, analyzed, and interpreted the data. All authors contributed to data analysis, drafting or revising the article, have agreed on the journal to which the article will be submitted, gave final approval of the version to be published, and agree to be accountable for all aspects of the work.
Funding
The study was funded by Sanofi.
Disclosure
AH, MJG, MB, and EL are employees of Sanofi. DDG is an employee of Allergy & Asthma Network and reports grants from Sanofi. The authors report no other conflicts of interest in this work.
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