Cognition and communication in patients with spinal muscular atrophy: A systematic review.

•Synthesizes evidence from 12 studies on cognitive and communicative impacts in SMA, focusing on nuanced functional outcomes.•Highlights cognitive variability in SMA1, revealing subtle challenges in SMA2 and 3, and stresses tailored assessment methodologies.•Identifies communication barriers in SMA, emphasizing the urgency of investigating their potential interplay with cognitive functions.

What matters most to patients with multiple myeloma? A Pan-European patient preference study.

Introduction: Given the rapid increase in novel treatments for patients with multiple myeloma (MM), this patient preference study aimed to establish which treatment attributes matter most to MM patients and evaluate discrete choice experiment (DCE) and swing weighting (SW) as two elicitation methods for quantifying patients' preferences.

Methods: A survey incorporating DCE and SW was disseminated among European MM patients. The survey included attributes and levels informed by a previous qualitative study with 24 MM patients.

TRIPS to Where? A Narrative Review of the Empirical Literature on Intellectual Property Licensing Models to Promote Global Diffusion of Essential Medicines.

Governed through the World Trade Organization Agreement on Trade-Related Aspects of Intellectual Property Rights (TRIPS) since 1995, the current medical R&D system requires significant trade-offs between innovation and high monopoly prices for patented drugs that restrict patient access to medicines. Since its implementation, few amendments have been made to the original TRIPS agreement to allow low- and middle-income countries (LMICs) to facilitate access by generic manufacturers through flexible provisions, such as compulsory licensing and parallel import. Although a useful policy tool in theory, the routine use of TRIPS flexibilities in LMICs in the procurement of new essential medicines (EMs) is regarded as a 'last resort' due to strong political response in high-income countries (HICs) and new trade agreements' restrictions.

Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study.

Investigational and marketed drugs for the treatment of multiple myeloma (MM) are associated with a range of characteristics and uncertainties regarding long term side-effects and efficacy. This raises questions about what matters most to patients living with this disease. This study aimed to understand which characteristics MM patients find most important, and hence should be included as attributes and levels in a subsequent quantitative preference survey among MM patients.

Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions.

This review can inform gene therapy developers on challenges that can be encountered when seeking market access. Moreover, it provides an overview of trends among challenges and potential solutions.

Improving Patient Involvement in the Lifecycle of Medicines: Insights From the EUPATI BE Survey.

EUPATI Belgium (EUPATI.be) is an informal gathering of local partners who are interested in improving patient involvement in healthcare innovation and medicines research and development. EUPATI.

Opportunities and challenges for the inclusion of patient preferences in the medical product life cycle: a systematic review.

Background: The inclusion of patient preferences (PP) in the medical product life cycle is a topic of growing interest to stakeholders such as academics, Health Technology Assessment (HTA) bodies, reimbursement agencies, industry, patients, physicians and regulators. This review aimed to understand the potential roles, reasons for using PP and the expectations, concerns and requirements associated with PP in industry processes, regulatory benefit-risk assessment (BRA) and marketing authorization (MA), and HTA and reimbursement decision-making.

Methods: A systematic review of peer-reviewed and grey literature published between January 2011 and March 2018 was performed.

Patient Preferences in the Medical Product Life Cycle: What do Stakeholders Think? Semi-Structured Qualitative Interviews in Europe and the USA.

Background: Patient preferences (PP), which are investigated in PP studies using qualitative or quantitative methods, are a growing area of interest to the following stakeholders involved in the medical product lifecycle: academics, health technology assessment bodies, payers, industry, patients, physicians, and regulators. However, the use of PP in decisions along the medical product lifecycle remains limited. As the adoption of PP heavily relies on these stakeholders, knowledge of their perceptions of PP is critical.

From Opioid Pain Management to Opioid Crisis in the USA: How Can Public-Private Partnerships Help?

The current opioid crisis in the USA arose from (at first) successful opioid pain management in three waves, starting in the'90s. Today, USA patients consume opioid drugs on a massive scale. Considering their potential for tolerance, as well as their potential for lethality in relatively small overdose, the overuse of opioids form an urgent threat to public health in the USA.

Patient Involvement in the Lifecycle of Medicines According to Belgian Stakeholders: The Gap Between Theory and Practice.

Patient involvement is often acknowledged as an important aspect of the lifecycle of medicines. Although different typologies exist, patient involvement has been described as the involvement of patients in decision-making regarding medicines. In view of the diversity of stakeholders and types of decisions in which patients might be involved, an in-depth understanding of these stakeholders' views toward involving patients in the lifecycle of medicines is essential.

Innovative Approaches to Increase Access to Medicines in Developing Countries.

Access to essential medicines is problematic for one third of all persons worldwide. The price of many medicines (i.e.