Patient-focused drug development and “beyond the pill” strategies could improve the safety of cancer drugs

Industry Insights


Increasingly, pharma is striving towards true patient centricity and here we explore how patient-focused drug development (PFDD) could have a direct impact on drug safety and adverse event management. The FDA is developing guidance for PFDD with the goal to better incorporate the patient’s voice throughout the process.1 In addition to collecting outcomes that are meaningful to patients, this PFDD approach asks that sponsors “identify the information that is most important to patients related to treatment benefits, risks, and burden, and how to best communicate the information to support their decision making”.1 Using behavior science to ensure the individuals receive the most appropriate information and support at the right time and in the right way can have a significant influence on patient-centered outcomes.

Addressing expectations and concerns about side effects

Take for example, the potential value of addressing individuals’ concerns about and expectations of side effects before they start cancer treatment. There is strong evidence from studies in cancer2 and other long-term conditions,3 that patients’ prior expectations and concerns about adverse events impact their subsequent experience and reporting of side effects including hair loss, pain and fatigue.2 The relationship between expectations and experience of side effects occurs in both women and men,4 and remains significant when controlling for other factors that could explain the association, including patients’ knowledge of their susceptibility to side effects and increased monitoring of their bodies for side effects.5 It has therefore been suggested that patients’ expectations may directly influence the emergence of side effects.2

The need to provide patient-centric support at the right time becomes even more pressing when we consider that the most common reasons for stopping cancer treatments, or taking less than prescribed, are concerns about side effects, inadequate information and a lack of support.6,7 Given that nonadherence and nonpersistence with treatment have been linked to poorer outcomes and increased risk of death,8,9 providing this support could not only improve quality of life, but also be literally life-saving.

How can we best support patients?

Behavioral scientists select intervention components based on analysis of the factors influencing behavior in the specific patient population, including those affecting capability, opportunity and motivation.10 Much of the support available to patients is educational in nature and does not address individual barriers or motivators. There are also programs facilitating psychological and practical support: an excellent example is CaringBridge,11 which provides a free platform for people going through a health crisis to share health updates so that loved ones can provide vital emotional and practical support.  However, individualized support directly focused on addressing misconceptions and concerns about illness and treatment, preventing and alleviating side effects and enhancing communication with health professionals, is often lacking.

‘Beyond the pill’ solutions

Pharma is revolutionizing the way that clinical trials are conducted, by ensuring outcomes that are meaningful to patients are captured, digitalizing trial procedures and making trial participation more convenient for patients. We recommend that pharma further enhance the patient centricity of their drug development by incorporating ‘beyond the pill’ solutions to maximize the benefit patients get from their treatments. Since we know that the safety profile can be negatively affected once the drug comes to market,12 it is important to consider how this kind of support can also be continued in post-marketing settings.


1.  U.S. Food and Drug Administration. CDER Patient-Focused Drug Development
2.  Devlin EJ, Denson LA, Whitford HS. Cancer Treatment Side Effects: A Meta-analysis of the Relationship Between Response Expectancies and Experience. J Pain Symptom Manage. 2017;54(2):245-258 e242.
3.  Horne R, Chapman S, Glendinning E, Date HL, Guitart J, Cooper V. Mind Matters: Treatment Concerns Predict the Emergence of Antiretroviral Therapy Side Effects in People with HIV. AIDS Behav. 2019;23(2):489-498
4.  Devlin EJ, Whitford HS, Denson LA, Potter AE. “Just As I Expected”: A Longitudinal Cohort Study of the Impact of Response Expectancies on Side Effect Experiences During Radiotherapy for Prostate Cancer. J Pain Symptom Manage. 2019;57(2):273-281 e274.
5.  Colagiuri B. Participant expectancies in double-blind randomized placebo-controlled trials: potential limitations to trial validity. Clin Trials. 2010;7(3):246-255.
6.  Clancy C, Lynch J, P OC, Dowling M. Breast cancer patients’ experiences of adherence and persistence to oral endocrine therapy: A qualitative evidence synthesis. Eur J Oncol Nurs. 2020;44:101706.
7.  Lin C, Clark R, Tu P, Bosworth HB, Zullig LL. Breast cancer oral anti-cancer medication adherence: a systematic review of psychosocial motivators and barriers. Breast Cancer Res Treat. 2017;165(2):247-260.
8.  Marin D, Bazeos A, Mahon F.-X, Eliasson L. et al. (2010). Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib. Journal of Clinical Oncology, 28(14), 2381–2388
9.  Greer JA, Amoyal N, Nisotel L, et al. A Systematic Review of Adherence to Oral Antineoplastic Therapies. Oncologist. 2016;21(3):354-376
10.  Jackson C, Eliasson, L., Barber, N., Weinman, J. Applying COM-B to medication adherence: A suggested framework for research and interventions. The European Health Psychologist. 2014;Vol 16 (1)
11.  Caring Bridge. Accessed 3. February, 2021.
12.  Cross, J., Lee, H., Westelinck, A., Nelson, J., Grudzinskas, C., & Peck, C. (2002). Postmarketing drug dosage changes of 499 FDA-approved new molecular entities, 1980-1999. Pharmacoepidemiology and Drug Safety, 11(6), 439–446.