Representation of Patients With Rare Diseases in Spanish Media

There are more than 360 associations of patients with rare diseases in Spain that strive for visibility to obtain funding and encourage clinical pathologies. The Spanish Year of Rare Diseases has been a considerable effort to be part of media agenda since 2013 and a “collective voice” throughout the media has been encouraged with the international initiatives devoted to the cause. Over the past years, representation of patients with rare diseases in Spanish media has been very superficial, despite the renewed interest during the Spanish Year of Rare Diseases. Certain cases as “Paco Sanz” or “Los Padres de la Pequeña Nadia” have negatively affected this representation by using the disease to pursue economic benefit. This chapter reports on the representation of rare diseases through Spanish media and the way it evolved in the last 6 years. The findings highlight the effort that has been made by patient advocacy groups with rare diseases and their relatives and caregivers, who have been recognized and proactive to get the treatment and medication needed.

Patient Advocacy and Health Literacy

This chapter presents a reflective analysis based on the literature and on the author's experience regarding the challenges for health professionals and for patients within the scope of the patient advocacy activity, especially in Portugal, where the formal figure of the patient advocate is almost non-existent. Patient Associations, professionals, patients and their families, and citizens in general are unaware of this concept, making it a priority to invest in health literacy and to structure training programs in patient advocacy. The authors believe that there is still a long way to go in Portugal in this matter and that the model, Assertiveness, Clarity of Language, and Positivity (ACP), is an essential model for the entire patient advocacy development process and implementation of the patient advocate figure in the area healthcare. In this chapter, some experiences, projects and the first results of an exploratory study will be presented in order to help in improving the understanding and knowledge of these concepts in Portugal.

Physician-Assisted Suicide in Dementia: Paradoxes, Pitfalls and the Need for Prudence

There has been an increasing drive towards the legalization of physician-assisted suicide (PAS) in patients with dementia, particularly in patients with advanced disease and severe cognitive impairment. Advocacy for this position is often based on utilitarian philosophical principles, on appeals to the quality of life of the patient and their caregiver(s), or on economic constraints faced by caregivers as well as healthcare systems. In this paper, two lines of evidence against this position are presented. First, data on attitudes towards euthanasia for twenty-eight countries, obtained from the World Values Survey, is analyzed. An examination of this data shows that, paradoxically, positive attitudes towards this procedure are found in more economically advanced countries, and are strongly associated with specific cultural factors. Second, the literature on existing attitudes towards PAS in cases of dementia, along with ethical arguments for and against the practice, is reviewed and specific hazards for patients, caregivers and healthcare professionals are identified. On the basis of these findings, the author suggests that the practice of PAS in dementia is not one that can be widely or safely endorsed, on both cultural and ethical grounds. Instead, the medical field should work in collaboration with governmental, social welfare and patient advocacy services to ensure optimal physical, emotional and financial support to this group of patients and their caregivers.

NCCN Virtual Patient Advocacy Summit: Cancer Across the Lifespan

Patients with cancer have widely divergent experiences throughout their care from screening through survivorship. Differences in care delivery and outcomes may be due to varying patient preferences, patient needs according to stage of life, access to care, and implicit or explicit bias in care according to patient age. NCCN convened a series of stakeholder meetings with patients, caregivers, and patient advocacy groups to discuss the complex challenges and robust opportunities in this space. These meetings informed the NCCN Virtual Patient Advocacy Summit: Cancer Across the Lifespan held on December 10, 2020, which featured a keynote presentation, multidisciplinary panels, and presentations from patient advocacy organizations. This article encapsulates and expounds upon the findings from the stakeholder meetings and discussions during the summit.

Patient advocacy group involvement in health technology assessments: an observational study

Background In some jurisdictions, patients and patient groups may be invited to provide input when Health Technology Assessment (HTA) is used to inform decisions about which medicines should be subsidised or funded. This input can help frame the evidence from a patient perspective, address uncertainties in the evidence and interpret it for the local setting. However, there is currently no evidence linking patient involvement with positive reimbursement decisions. Aim We aimed to understand the expectations of patient involvement in the reimbursement process, especially among cancer patient advocacy groups (PAGs) in New Zealand (Aotearoa), South Korea and Taiwan. Methods We developed an online survey to help understand the role that cancer PAGs play in reimbursement processes and identify knowledge gaps about the processes that might impact the efforts of PAGs. The survey elicited the views of staff and patients affiliated with PAGs (n = 43) on current practices and how the assessment and reimbursement of new cancer drugs might be improved. Results There was variability in knowledge of the HTA assessment processes and in experience of being involved in them. Those with HTA experience were more likely to have confidence in the process. Those who had not been involved tended to have little awareness of, or frustration with, decision-making processes. Most identified cost, finances and economic assessments as key considerations in current processes. Some respondents had clear ideas about how their knowledge and involvement could improve processes to determine the value of new medicines. However, for many, a lack of information about the basis for decision making and opportunities to be involved was a barrier to identifying process improvement. Conclusions HTA is implemented primarily in countries seeking to have fair and equitable processes for funding medicines. PAGs often recognise the financial challenges of funding new medicines and share the desire for procedural fairness. The connection PAGs make between patient involvement and improved access to new medicines may be based on the belief they can add information to the evidence base, help solve problems, ensure fairness through transparency and/or influence the culture towards increased access to medicines they value.

Introduction

In addition to setting out the themes of the book, the Introduction tells the story of cancer patient Abigail Burroughs and the case of Abigail Alliance v. von Eschenbach (2008), a suit against the FDA brought by a patient advocacy group founded by Abigail’s father after her death. In this case, the US Court of Appeals for the DC Circuit denied terminally ill patients the right to obtain investigational drugs prior to FDA approval. Using a test established by the Supreme Court in its physician-assisted suicide decision, Washington v. Glucksberg (1997), the DC Circuit held that a right to access unapproved drugs is not “deeply rooted in this Nation’s history and tradition.” The Introduction asserts that the Abigail Alliance court’s version of history was incomplete and one-dimensional, as the rest of the book will demonstrate.