Background The current standard of care for multiple myeloma requires several regimens of treatment, with patients experiencing high symptom burden and side effects, which negatively impact health-related quality of life (HRQoL). Thus, it is crucial to understand patient perceptions of multiple myeloma and how patients value different treatment options. Objective The purpose of this study was to conduct an exploratory investigation into concepts that could form attributes that influence treatment choices for patients with multiple myeloma and to identify trade-offs that patients are willing to make between treatment attributes. Methods In total, 30 patients with newly diagnosed or relapsed/refractory multiple myeloma from the UK, France, and Germany participated in semistructured interviews talking about their disease experience and symptoms, treatment benefits, treatment burden, perceived side effects, and benefit/risk trade-offs in treatment. The interview audio recordings were transcribed and analyzed using content analysis to identify treatment and disease aspects relevant to patients. Results Symptoms of fatigue and bone pain and treatment side effects of peripheral neuropathy, diarrhea, and constipation were cited by patients as the most disruptive to their HRQoL. Treatment duration was reported most frequently as a major treatment burden, and patients emphasized the importance of increased life expectancy as a treatment benefit. All patients showed good understanding of benefit/risk trade-offs in treatment, and some patients expressed a preference for more convenient modes of treatment administration. Conclusions Qualitative interviews identified key aspects of multiple myeloma treatment that are most important to patients. These findings will inform a wider patient-preferences study, which could improve treatment choice and HRQoL for patients with multiple myeloma.

Background Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder defined by left ventricular hypertrophy that cannot be explained by another cardiac or systemic disease. There is a general lack of knowledge about patients’ perspectives on the symptoms and day-to-day limitations they experience as a result of HCM. We therefore sought an in-depth understanding of patients’ experiences of obstructive (oHCM) and nonobstructive (nHCM) forms of the disease, including symptoms and their quality of life impacts, and to develop a conceptual model to capture them. Methods Development of the HCM conceptual model involved a web-based survey to capture patients’ insights, a targeted literature review (which included relevant guidelines and patient advocacy websites), one-to-one interviews with clinical experts, and one-to-one qualitative concept elicitation interviews with patients. Key symptoms and their impacts most important to patients’ experiences were identified and used to develop a conceptual model of the patient experience with HCM. Results The HCM symptoms reported by patient interviewees (n = 27) were largely consistent with findings from the patient web survey (n = 444), literature review, and interviews with three expert clinicians. The symptoms most commonly reported in patient interviews included tiredness (89%), shortness of breath (89%), shortness of breath with physical activity (89%), and dizziness/light-headedness (89%). Other symptoms commonly reported included chest pain (angina) (70%), chest pain (angina) with physical exertion (70%), and palpitations (fluttering or rapid heartbeat) (81%). The most commonly reported impacts of HCM symptoms on patients’ lives included limitations to physical activities (78%), emotional impacts, including feeling anxious or depressed (78%), and impacts on work (63%). Symptoms and impacts were similar for both oHCM and nHCM. Conclusions A conceptual model was developed, which identifies the core symptoms that patients with oHCM and nHCM reported as most frequent and most important: shortness of breath, palpitations, fatigue/tiredness, dizziness/light-headedness, and chest pain, as well as the impacts those symptoms have on patients’ lives. This HCM conceptual model reflecting patients’ experiences and perspectives was used in the development of a patient-reported outcomes instrument for use in clinical trials and it may also help inform the clinical management of HCM.

Health-related quality of life (HRQoL) outcome can be used to monitor health and well-being and is one of the important outcomes that may predict overall survival (OS) in cancer patients. This study examined the association of OS and HRQoL measured before (T0), after cancer diagnosis (T1), and HRQoL changes from T0 to T1 (T1-T0) in patients diagnosed with bladder cancer (BC). Methods: This longitudinal retrospective cohort study used the 1998-2013 Surveillance, Epidemiology and End Results (SEER) database linked with Medicare Health Outcomes Survey (MHOS). HRQoL was measured using the SF-36 (1998-2005)/ Veterans Rand-12 (2006 onwards) and reported as physical (PCS), mental (MCS) component scores, and 8 domain scores. Adjusted hazard ratios (aHRs [95%CI]) were estimated using multivariable logistic regression models, adjusting for demographics, comorbidities, and cancer specific characteristics. Results: 438 BC patients aged $65 years who had HRQoL data at both T0 and T1 were identified. Among those, 220 (50.2%) died and 218 (49.8%) remained alive (censored). Compared to deceased patients, the censored patients were younger (75.366.0 vs. 77.266.8 years; p,0.01), had longer OS (90.4639.9 vs. 67.9642.0 months; p,0.001), had a lower-stage tumor at diagnosis (p,0.001), and had non-invasive tumors (p,0.01). Risk of death was negatively associated with increased PCS at T0 and T1 (aHR T0 =0.

Background The effective impact of patient engagement (PE) across the medicines development continuum is widely acknowledged across diverse health stakeholder groups, including health authorities; however, the practical applications of how to implement meaningful and consistent PE are not always addressed. Guidance for the practical implementation of PE requires granularity, and the need for such guidance has been identified as a priority. We describe the co-production and summarize the content of how-to guides that focus on PE in the early stages of medicines development. Methods Multi-stakeholder working groups (WGs) were established by Patient Focused Medicines Development (PFMD) for how-to guide development. How-to guides were co-produced with patients for PE activities identified as priorities through public consultation and by WGs. Guides were developed by applying PE quality guidance and associated quality criteria in an iterative process. How-to guides underwent internal review and validation by experts (ie, those with relevant experience in the particular PE activity or focus area) in specific focus groups and external review and validation through appropriate events and public consultation. Results Overall, 103 individual contributors from 38 organizations (representing eight stakeholder groups, including patients/patient organizations) and from 14 countries were organized into WGs and workstreams. Each WG comprised 15–30 contributors with PE experience relevant to the specific how-to guide. How-to guides were developed for PE in the early discovery and preclinical phases; PE in the development of a clinical outcomes assessment strategy; and PE in clinical trial protocol design. The how-to guides have a standardized format and structure to promote user familiarity. They provide detailed guidance and examples that are relevant to the individual PE activity and aim to facilitate the practical implementation of PE. Conclusions The how-to guides form a comprehensive series of actionable and stepwise resources that build from and integrate the PE quality criteria across the medicines continuum. They will be made freely available through PFMD’s Patient Engagement Management Suite (pemsuite.org) and shared widely to a variety of audiences in different settings, ensuring access to diverse patient populations. Implementation of these guides should advance the field of PE in bringing new medicines to the market and ultimately will benefit patients. Plain English summary Medicines are developed to help patients improve their health and lives. Many organizations and individuals want to ensure that medicines are developed to meet real patient needs and to address what is most important to patients. Finding out what patients need and what patients want requires good patient engagement, but knowing how to do patient engagement is not always clear. This is because medicines development is complicated, and a lot of different steps, people, and organizations are involved. Patient Focused Medicines Development (PFMD) was established in 2015 to connect individuals and organizations that are committed to making medicines not just for patients but with patients. To do this, PFMD brought together patients and other groups of people with relevant experience and good ideas on how to achieve patient engagement in the real-world setting. Together, PFMD has developed “how-to guides” for patient engagement that cover the main activities along the medicines development process. The guides are free to use and provide practical advice and examples that anyone can use in their patient engagement activities. The how-to guides will also help patients to understand medicines development and how best they can participate in this process to address their needs.

Objectives Treatments for systemic sclerosis-associated interstitial lung disease (SSc-ILD) differ in attributes, i.e. mode of administration, adverse events (AEs) and efficacy. As physicians and patients may perceive treatments differently, shared decision-making can be essential for optimal treatment provision. We therefore aimed to quantify patient preferences for different treatment attributes. Methods Seven SSc-ILD attributes were identified from mixed-methods research and clinician input: mode of administration, shortness of breath, skin tightness, cough, tiredness, risk of gastrointestinal AEs (GI-AEs), and risk of serious and non-serious infections. Patients with SSc-ILD completed an online discrete choice experiment (DCE) in which they were asked to repeatedly choose between two alternatives characterized by varying severity levels of the included attributes. The data were analysed using a multinomial logit model; relative attribute importance and maximum acceptable risk measures were calculated. Results Overall, 231 patients with SSc-ILD completed the DCE. Patients preferred twice-daily oral treatments and 6–12-monthly infusions. Patients’ choices were mostly influenced by the risk of GI-AEs or infections. Improvement was more important in respiratory symptoms than in skin tightness. Concerning trade-offs, patients accepted different levels of increase in GI-AE risk: +21% if it reduced the infusions’ frequency; +15% if changing to an oral treatment; up to + 37% if it improved breathlessness; and up to + 36% if it reduced the risk of infections. Conclusions This is the first study to quantitatively elicit patients’ preferences for treatment attributes in SSc-ILD. Patients showed willingness to make trade-offs, providing a firm basis for shared decision-making in clinical practice.