The importance of patient perspectives in pulmonary hypertension

Narrative medicine

A key feature of patient-centred medicine in chronic conditions such as PH is that the patient presents with a spectrum of symptoms and associated psychological responses which occur within a sociological and cognitive framework unique to that patient, and which contribute to the patient's fears, coping mechanisms and goals. Treatment focused exclusively on the underlying disease often fails to address the ripples of impact provoked by PH which may become the main source of concern to the patient. The ability of HCPs to “acknowledge, absorb, interpret, and act on the stories and plights of others” has been referred to as narrative competence, the foundation of narrative medicine [56]. The intention of narrative medicine is to adopt a broad perspective in terms of appreciating the entire spectrum of the patient's presentation and also by enlisting elements of other disciplines (such as the humanities and literature) to best understand that spectrum [57, 58]. The contribution of narrative knowledge is to provide a balance or context for the scientific dimension of medicine [59, 60]. Narrative medicine addresses the issue of how a unique individual under unique circumstances can be uniquely helped, whereas scientific medicine asks how the features of patients' presentations make them statistically similar or dissimilar to a demographic which responds in a given way to a particular management strategy.

In a practical sense, patients should be able to discuss with HCPs not only symptoms, but their experience of their individual illness in terms of functional burden and psychosocial impact, as well as their objectives of treatment. HCPs should encourage patients to express their perspective, be receptive to and attempt to comprehend and incorporate their views into management. An interaction of this type is crucial since the HCPs' goals for PH therapy tend towards achieving symptomatic and functional improvement, delaying worsening of disease, and extending life expectancy. Patients may emphasise quality of life, capability for coping with daily demands and minimising the burdens of complex therapies [61]. This mutually respectful bidirectional interaction would establish common ground for the process of shared decision making.

Shared decision making

The definition of shared decision making is succinctly stated in commonsense terminology: “a conversation between the clinician [and others, such as pastoral counsellor and social worker] and the patient in which they figure out together what to do to address the patient's situation” [62]. This concept incorporates components that are “iterative and interactive steps in a conversational dance”, which include: clarifying the patient's situation; identifying the aspect of the situation that requires action; recognising that more than one way of addressing the situation exists; discussing the pros and cons of the available approaches with the patient and caregivers; and gaining understanding of what the patient values about these options, and why [62].

For the patient perspective to be valid and relevant, it must be informed by an accurate comprehension by the patient of the facts of the situation. In some medical disciplines, decision aid tools have been developed to facilitate the patient's understanding of the clinical context and potential outcomes of alternative treatment strategies, including those which are probabilistic in nature. Decision aids promote the patient's knowledge, estimation of treatment risk and engagement in the discussion. However, there has not yet been a demonstrable benefit in terms of outcome [62]. Standardised decision aids have not been developed for PH management.

Palliative care

Palliative care was traditionally a change in the focus of care at the end of life away from therapies intended to cure or slow the progression of disease, to management of physical symptoms (e.g. pain) and emotional distress. Palliative care is currently better understood as an interdisciplinary approach to promoting quality of life and reducing suffering at any stage of chronic disease [63]. Palliative care has been defined as “patient and family-centered care that optimizes quality of life by anticipating, preventing, and treating suffering. Palliative care throughout the continuum of illness involves addressing physical, intellectual, emotional, social, and spiritual needs and to facilitate patient autonomy, access to information, and choice” [64].

In a survey of 774 PAH patients, 276 analysable responses revealed that awareness of and access to palliative care resources were low despite marked impairment of HRQoL, physical and emotional wellbeing, social activity, and pain control [49]. This degree of diminished HRQoL is consistent with that found in other studies [20, 49, 65–69]. Since PH-targeted therapies do not usually alleviate all physical symptoms or necessarily improve patients' psychosocial function, a role for integrating palliative care techniques and expertise early to optimise HRQoL has been advocated [70].

Obstacles to expanding the use of palliative care in PH patients are lack of awareness of its purpose (e.g. symptom management and psychological wellbeing), inadequate local medical payer coverage, misunderstanding palliative care as synonymous with hospice care and implies “giving up” or being resigned to an imminent death, and not understanding that PH-targeted medications can continue within a palliative care strategy [63]. Therapies for PH have side-effects which may negatively impact HRQoL despite achieving benefits in terms of haemodynamics and some symptoms. Thus, strategies to focus on HRQoL during otherwise beneficial treatment arguably become even more imperative.

Patient associations

Organisations have been created to facilitate the expression of the PH population perspective and to aid in dealing with the needs of PH patient communities worldwide (table 3).

Support groups

Support groups are smaller local patient networks with less infrastructure and expense which provide informal interactions to offer empathetic sharing of experiences and education. Support groups may be independent or promoted by medical institutions or pharmaceutical companies, and may receive logistical or financial assistance from national patient associations. Such local/regional support groups exist in many countries especially in North America (e.g. more than 240 identified by the Pulmonary Hypertension Association (PHA) in the USA), but also in a number of European, Latin American, Asian and African nations.

Education/awareness

Acquiring information about PH is an essential coping mechanism for many patients. In addition, the perception that others lack awareness and knowledge about their disease is distressing. Insights provided to patients in subspecialty clinics by dedicated staff helps address these needs [50, 71], but public dissemination of information also is central to patient organisations' mission. A pivotal component of this effort has been to increase awareness of PH both in HCPs and the general population in order to improve the timeliness and accuracy of PH diagnosis in patients presenting with suggestive symptoms (e.g. the “Early Diagnosis” campaign of the US PHA), catalysed by observations that there is a significant interval between symptom onset and definitive diagnosis [4, 5, 72].

The annual World Pulmonary Hypertension Day was first held on May 5, 2012 in Madrid, Spain to raise global awareness and this was adopted by PHA Europe (http://worldphday.org). The World Pulmonary Hypertension Day has gained increasing momentum, with 86 global partner organisations involved in an international awareness campaign in 2018. Through its involvement of national political and health authorities, academia, HCPs, and celebrities, World Pulmonary Hypertension Day has generated media interest and awareness across the world.

Evaluation of patients at referral centres frequently identifies aspects of pre-referral management that do not align with best practices and guidelines [73]. Concerned physicians and patients in the PH community re-emphasised the need for professional education, including periodic international symposia (e.g. the World Symposia on Pulmonary Hypertension (WSPH) [74] and the European Society of Cardiology (ESC)/European Respiratory Society (ERS) guidelines for PH [45], culminating in publications of state-of-the-art knowledge summaries, management guidelines and algorithms), web-based educational programmes, on-site preceptorships and PH-focused publications.

Access to healthcare/coverage

Disparity in healthcare access is a worldwide issue which patient organisations address on behalf of their constituencies. A major concern of patients, their families and primary HCPs is where the patient should be most effectively managed. This concern embodies many elements: how can qualified experts and well-resourced medical facilities be identified; how can geographical and financial constraints be navigated; by whom are complex treatment strategies best prescribed, managed and followed; and what is the best approach to medical emergencies? The answers are largely dependent on the geographical location and nationality of the patient.

In the USA, under the aegis of the PHA, the PH Care Centers (PHCC; https://phassociation.org/phcarecenters) programme of site accreditation was developed to identify facilities which are staffed and equipped to deliver patient management that meets expert consensus for best practices.

The European Reference Networks (ERNs; https://ec.europa.eu/health/ern_en) for rare diseases, launched in 2017, introduced a European Union (EU) accreditation system for centres of expertise for rare diseases to whom referrals can be made for “virtual” consults. The EU initiative is intended to facilitate cross-border sharing of knowledge, experience, medical research, teaching, training and resources. Patients are closely involved and each ERN has a Patient Advisory Group (ePAG) which advises on strategy, policy and organisational processes. PHA Europe has three representatives on ERN Lung.

Latin American and Asian countries also confront difficulties with early diagnosis and access to treatment for PH patients due to lack of knowledge and insufficient legal frameworks to protect the right to good healthcare by World Health Organization criteria. Scarcity of referral centres, inconsistency among health systems, wide distribution of patients, geographical barriers, economic disparity and limited accessibility to medical records have made it difficult to generate awareness of PH.

Advocacy and empowerment

Patient-centred organisations acknowledge patients' needs within a supportive environment and provide opportunities for patients and caregivers to advocate for themselves by being proactive in defining their situations, proposing solutions, participating in political activity to promote those solutions, being engaged in their own self-care and counselling each other [55]. Patients' understanding of their illness and engagement in the care process appears to contribute to improved coping ability [71] and outcomes [75, 76] in a variety of chronic diseases. Patient associations and individual patients in many parts of the world are active advocates for better access to treatment, organ transplant and greater attention to quality of life.

PHA Europe has been successful in involving members of the European Parliament, top EU officials, members of the ESC and ERS, and representatives of influential public health European non-governmental organisation in PH-related issues, including for two European Parliament meetings (2012 and 2016). PHA Europe is also active in the context of larger European organisations, and gives support and strategic advice to national advocacy activities.

Lacking a national single-payer system, the US PHA undertakes simultaneous efforts to educate policy makers at both the federal and state levels, and inform and educate federal and state regulatory agencies (e.g. Centers for Medicare & Medicaid Services and the US Food and Drug Administration (FDA), and state health and insurance agencies) and commercial payers. The US PHA uses a combination of advocacy staff, volunteer PH patients and caregivers, and coalitions of patient organisations with similar advocacy goals to influence policy makers.

The Latin Society of Pulmonary Hypertension (Sociedad Latina de Hipertensión Pulmonar (SLHP)) has supported improved living conditions of patients. One result of this is the Framework Law approved by the Health Commission of the Latin Parliament in 2015 and by other legislative forums to promote national laws that protect the Human Right to Health. This law regulates the healthcare for people with rare diseases in Latin America. However, the diversity, complexity, fracture and corruption of the health systems make it difficult to achieve a direct impact.

Role in research/registries

Patients have had a vital role in PAH-related clinical trials and registries, almost exclusively as study subjects. An early manifestation of the relevance of the patient perspective in research is the concept of patient-reported outcome (PRO) as a meaningful measurement of treatment effect of investigational drugs. In 2009, the US FDA stated that “A PRO is any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else” [77]. The 4th and 5th WSPH, in 2008 and 2013, respectively, advocated that PROs be included as a secondary end-point in clinical drug studies [78, 79]. Patient input is needed to understand their perspective and experience so that PROs can be developed as relevant end-points in clinical trials [80]. The US FDA launched the Patient-Focused Drug Development initiative to assess and integrate patient perspectives into the early phases of drug development in a range of diseases [81]. A meeting involving PAH patients and representatives was held in 2014, and emphasised the impact of PAH on the individual patient's life beyond the physical burden of symptoms, including loss of independence, inability to perform routine daily activities and fully participate in relationships with significant others and children, embarrassment, fear of being alone, and impact of treatment side-effects on HRQoL [82]. This initiative represents an important step in incorporating patient perspective and engagement at an early stage of clinical trial design.

Promote access to optimal management

Optimal care must be multidimensional to address the physical, psychological, social and informational needs of patients and caregivers, alongside their clinical needs. This approach is suggested by the latest ESC/ERS clinical guidelines: “[expert] Referral centres are recommended to provide care by an interprofessional team …” [45]. Nevertheless, guidelines are largely silent about the desirability of patient/HCP bidirectional interactions that address patients' need for emotional support conducted by a collaborative team at a “tempo” which permits the patients to understand the HCP's information and to convey their perspectives, concerns and goals. Future guidelines should point out that HRQoL objectives require a culture of shared decision making and narrative competence to support patient and caregiver emotional resilience. This should include access to focused psychological management, social work, palliative care, spiritual counselling and physical therapy (figure 2).

• Download figure
• Open in new tab
• Download powerpoint

FIGURE 2

Representation of components of a multidimensional approach to care of the pulmonary hypertension patient.

Optimal care must be accessible to the patient. Mechanisms, such as ERNs and the PHCC, to evaluate and identify sites able to provide clinical management consistent with consensus guidelines are important steps. The next challenge is to promote restriction of care to such centres once sufficient numbers have been identified to serve the PH population. This is most likely to be achieved in systems with a single payer/commissioner, usually state provided.

In Latin America and in developing countries, future advances are particularly challenging because of the barriers noted earlier. A primary objective of patient organisations both in Latin America and worldwide will be to overcome these barriers and advocate for social, political and legal measures to procure accessible healthcare for PH patients. “Twinning relationships” between individual centres of expertise and centres seeking consultative input could be a means of promoting optimal care in needy regions. The regional disparity of care available to PH patients mandates that governments, insurers, the pharmaceutical industry, and patient and professional organisations promote global accessibility and affordable treatment for all PH patients. For a compelling narrative regarding the need for globalisation of PH care, please watch the Jenna Lowe Trust video at: https://phassociation.us13.list-manage.com/track/click?u=acd152c7c169b59aaec993284&id=4874e4af3a&e=99080b0e01.

Empower patients’ participation in their management

PH patients and HCPs collaboratively make decisions which have a major impact and require an understanding by all parties of the components and priorities involved in achieving an appropriate course of action. To achieve the necessary mutual understanding requires a complexity, duration and frequency of interaction that poses a challenge within the time constraints of medical practices. The process may be facilitated by incorporating formal principles of shared decision making, including the use of decision aid tools. A synthesis of the qualitative precepts of narrative medicine and the implementation of more quantitative HRQoL instruments in clinical practice might also enhance the physician's awareness of the patient's needs and systematically reflect aspects of illness that are not otherwise measured.

Patient associations frequently offer educational materials which complement the role of the HCP by providing information about the disease, treatment options and day-to-day issues in living with PH. Since 2015, over 200 resources in 24 languages have been collected in the “PH Library” (www.ourphlibrary.com), an online joint project of PHA in the USA and PHA Europe, which should be continued and expanded. An additional role that patient organisations and support groups might play is to help patients develop narrative medicine knowledge and skills so they can be more effective during their limited time with HCPs.

Role and timing of palliative care

The assimilation of palliative care principles into the PH management strategy at an earlier phase of disease should be encouraged to promote all means of improving HRQoL by pain management, symptomatic improvement of dyspnoea, addressing psychological issues and sleep disturbances, and assisting in end-of-life concerns. Equating the term “palliative care”, which is currently burdened with the notion of “end-of-life” care, with “holistic” or “quality-of-life” care will be an important strategy for reorienting perceptions about this discipline.

Integrate concepts of patients’ perspectives into medical education

Medical education has recognised the importance of the patient perspective to a degree, incorporating concepts of shared decision making, narrative-based medicine and HRQoL into educational curricula. Narrative medicine can potentially increase efficiency and bring economic benefits to healthcare systems. A closer and more collaborative patient–doctor relationship can improve treatment compliance and satisfaction, and reduce unnecessary tests and medicolegal disputes.

It is important to further integrate these concepts into all clinical disciplines, including PH management, at all levels of medical education from medical school to continuing medical education of all HCPs.

Support, expand and harmonise HRQoL databases

Surveys of patient perspectives are an important source of information by which HCPs can orient themselves to their patients' multiple and complex needs. Since perspectives may vary as the treatment milieu changes over time, it will be important to periodically reassess using updated survey instruments. Information derived from patient surveys can differ by geographical location. This suggests that the use of surveys in other regions of the world would be informative in terms of comparisons and would also provide new insights into the attitudes of previously unexamined populations, including those in Latin America, the Middle East, Africa and Asia.

Surveys of patient perceptions should be integrated with traditional clinical database registries Incorporation of these variables into a single or cross-linked case report form would potentially simplify (unify) data collection. Ultimately data analyses might better determine whether treatment strategies and traditional outcome measures (e.g. hospitalisation and survival) correlate with HRQoL. It is likely that these analyses would be hypothesis generating and lead to further formal study.

It would be useful if registries and/or patient surveys were designed to prospectively harmonise data variables in such a way that global analyses could be performed without resorting to post hoc statistical adjustment.

Incorporate patients' perspectives early into clinical study design and outcome measurements

Based on information gleaned from surveys, including patients in the clinical trial design process may increase the importance of “overall wellbeing” as a central objective of medical care and potentially as a clinical trial outcome on an equal footing with more traditional end-points. HRQoL could be included as an end-point by itself or as part of a multiparameter score, rather than simply a surrogate for other end-points such as survival or clinical deterioration.

The PAH patient and caregiver community needs to be cognisant of the developing opportunity to participate in relevant advisory and steering committees in order to advocate for the patients' perspective in clinical trial design. This might be best pursued by patient organisations by advocating for and facilitating patients to serve as steering committee members, patient liaisons or focus group members for study designs, as an outgrowth of US FDA and European Medicines Agency initiatives to promote “patient-focused drug development”.

Recognise and enhance the role of PAH patient organisations/professional organisations

As noted, existing patient organisations (e.g. in the USA, Europe and elsewhere) work as a loose confederation with a similarity of general objectives adapted to their constituencies' specific realities and needs (table 3). Such patient associations have a track record of providing a voice and support for the PH patient community. Thus, continued development of these organisations should be supported and similar associations should be established in underserved areas. Obstacles to globalisation of PH patient organisations are significant and include: funding, geographic dispersion of rare disease patients, communication barriers, questions of organisational governance and substantial differences in delivery of medical care. The commonality of objectives and needs of PH patients suggest that stronger networking for the purposes of educational efforts and sharing of productive strategies should be promoted. An important step forward in this direction was taken in 2017 when the US PHA, SLHP and PHA Europe decide to join forces for the first time in organising the World Pulmonary Hypertension Day, which, in turn rallied many other associations from other parts of the world, including from Canada, Africa, Asia and Australia. Social media can play an increasing role in the future in breaking down geographical barriers and having a unifying function, and other avenues for collaboration should be explored.