Severe Asthma, Smarter Decisions: Aligning Objective Assessment with Patient Voices

As biologic therapies for severe asthma, monoclonal antibodies targeting key type‑2 inflammatory pathways, have transformed treatment, they remain costly and not without potential adverse effects; therefore, their optimal use requires careful patient selection based on objective criteria (e.g., adherence assessment, biomarker profile, corticosteroid responsiveness) rather than symptom reporting alone [1][2]. In clinical practice, poor adherence to inhaled corticosteroids (ICS) is well documented among patients with difficult‑to‑control asthma and contributes to persistent symptoms and exacerbations, complicating the identification of patients with true corticosteroid‑resistant disease who might benefit most from biologic therapy [3][4][5][6].

For this reason, stratified management protocols that systematically assess treatment adherence and corticosteroid responsiveness are recommended in international and national asthma guidelines before considering initiation of biologics, as they help improve targeting of advanced therapies and avoid unnecessary escalation [5][6][7][8].

One of the major goals of the UK Refractory Asthma Stratification Programme (RASP‑UK) over the past 3 years has been to develop and validate such protocols, focusing on methods that distinguish non‑adherence from true corticosteroid resistance using objective tests such as remote monitoring of ICS use and exhaled nitric oxide suppression testing (rasp.org.uk).

At the recent RASP‑UK Annual Consortium Meeting, researchers reported published findings from one strand of the programme that evaluated outcomes when people with severe asthma do not adhere to their prescribed treatment plans. These results underscore that non‑adherence is both common and clinically important; it can be mistaken for refractory disease, leading to unnecessary treatment escalation including inappropriate biologic prescribing. Objective adherence assessment, such as medication possession ratios and biomarker‑guided adherence tests, helps refine patient characterization, ensuring that biologic therapies are reserved for those who are adherent yet remain uncontrolled despite optimal standard care (rasp.org.uk).

Importantly, actively involving patients in research enhances the relevance, feasibility, and impact of clinical studies. Drawing on evidence from systematic reviews, when patients are engaged early and meaningfully in research design and execution, studies are more likely to address questions that matter to patients, improve enrolment and retention, and enhance translation of findings into practice [9][10][11]. In RASP‑UK, the Patient Input Platform comprising people living with severe asthma has informed study design, recruitment strategies, and dissemination planning; this has helped ensure that research outcomes align with patient needs, increased engagement, and improved uptake of findings in clinical practice.

So, what are the five key principles for successful and meaningful patient involvement identified in the source material from RASP‑UK?

• Involve Early: Patient input is most impactful during the project formation phase. Researchers often focus on scientific questions, but patients provide the context of living with a disease. Early input can range from making scientific documents understandable to people without a science background to strategic advice on recruiting a diverse breadth of participants.
• Involve Deeply: Meaningful involvement requires more than just meetings at the beginning and end of a project. Our experience demonstrated that the more patients are involved in day-to-day activities and informed of progress, the more they can understand and contribute. In practice, this meant having patient representatives participate in research working groups and operational conference calls where they can influence decision-making. They were also lively and informative contributors to our annual consortium conference.
• Have Patients Feedback on Project Progress: Patients have the biggest stake in a project's success. Their feedback can be highly motivating, their enthusiasm pushed our clinical partners to move forward more quickly, forcing consortium partners to reflect on the ultimate goal of the study: improving patient health.
• Include Patients in Dissemination: Patient advocates were often more active and better at spreading the word about our activities and goals through social media and online communities than researchers. They act as reviewers of dissemination resources and help ensure that important outcomes are shared with the general public, who are the ultimate source of support for research. This certainly seemed too have helped with recruitment to our various trials.
• Help Patients Convey Their Own Story: This principle focuses on enabling patients to share their unique perspectives and experiences. An example was again our annual conference where participants actively engaged with discussions defining their disease; this helped convey their personal reality to researchers and increased the passion and motivation of the project partners.

In essence, integrating objective pre‑biologic assessments with authentic patient partnership ensures that advanced therapies are used where they will truly make a difference, making precision asthma care both effective and patient‑centred.

References:

1. National Institute for Health and Care Excellence (NICE). Asthma: diagnosis, monitoring and chronic asthma management. NICE guideline [NG80]; 2017.
2. Global Initiative for Asthma (GINA). Global Strategy for Asthma Management and Prevention; 2018.
3. Normansell R, Kew KM, Stovold E. Interventions to improve adherence to inhaled steroids for asthma. Cochrane Database Syst Rev. 2017;CD012226.
4. Barnes PJ. Inhaled corticosteroids. Pharmaceuticals (Basel). 2010;3(3):514–540.
5. Reddel HK, Bateman ED, Becker A, et al. A summary of the new GINA strategy: a roadmap to asthma control. Eur Respir J. 2015;46(3):622–639.
6. Gamble J, Stevenson M, McClean E, Heaney LG. The prevalence of nonadherence in difficult asthma. Am J Respir Crit Care Med. 2009;180(9):817–822.
7. O’Byrne PM, et al. Severe asthma: future treatments and unmet needs. J Allergy Clin Immunol Pract. 2017;5(4):1165–1174.
8. Chung KF, Wenzel SE, Brozek JL, et al. International ERS/ATS guidelines on definition, evaluation and treatment of severe asthma. Eur Respir J. 2014;43(2):343–373.
9. Domecq JP, Prutsky G, Elraiyah T, et al. Patient engagement in research: a systematic review. BMC Health Serv Res. 2014;14:89.
10. Sacristán JA, Aguarón A, Avendaño‑Solá C, et al. Patient involvement in clinical research: why, when, and how. Patient Prefer Adherence. 2016;10:631–640.
11. Tapp H, Derkowski D, Calvert M, et al. Patient perspectives on engagement in shared decision‑making for asthma care. Fam Pract. 2016;34(3):353–357.