Patient Engagement and Patient Experience Data in Regulatory Review and Health Technology Assessment: A Global Landscape Review.

Background: Working with patients through meaningful patient engagement (PE) and incorporating patient experience data (PXD) is increasingly important in medicines and medical device development. However, PE in the planning, organization, generation, and interpretation of PXD within regulatory and health technology assessment (HTA) decision-making processes remains challenging. We conducted a global review of the PE and PXD landscape to identify evolving resources by geography to support and highlight the potential of integration of PE and PXD in regulatory assessment and HTA.

Culture and Process Change as a Priority for Patient Engagement in Medicines Development.

Patient Focused Medicines Development (PFMD) is a not-for-profit independent multinational coalition of patients, patient stakeholders, and the pharmaceutical industry with interests across diverse disease areas and conditions. PFMD aims to facilitate an integrated approach to medicines development with all stakeholders involved early in the development process. A key strength of the coalition that differentiates it from other groups that involve patients or patient groups is that PFMD has patient organizations as founding members, ensuring that the patient perspective is the starting point when identifying priorities and developing solutions to meet patients' needs.

Position statement from the European Board and College of Obstetrics & Gynaecology (EBCOG): The use of medicines during pregnancy - call for action.

Less than 10% of medicines approved by the FDA since 1980 have provided enough information as regards risks for birth defects associated with their use (Adam et al. (2011) [1]). Nevertheless, it is estimated that over 90% of pregnant women take over-the-counter (OTC) or prescription medication (Ke et al.

Position Statement from the European Board and College of Obstetrics & Gynaecology (EBCOG) : The use of medicines during pregnancy: call for action.

Less than 10% of medicines approved by the FDA since 1980 have provided enough information as regard risks for birth defects associated with their use [1]. Nevertheless, it is estimated that over 90% of pregnant women take over-the-counter (OTC) or prescription medication [2]. Considering the fact that the use of medication in the period before conception and during lactation can also influence the development of the child, information on the impact of their usage during reproductive life is important for everyone.

Partnering With Patients in the Development and Lifecycle of Medicines: A Call for Action.

The purpose of medicines is to improve patients' lives. Stakeholders involved in the development and lifecycle management of medicines agree that more effective patient involvement is needed to ensure that patient needs and priorities are identified and met. Despite the increasing number and scope of patient involvement initiatives, there is no accepted master framework for systematic patient involvement in industry-led medicines research and development, regulatory review, or market access decisions.

Medicines in Pregnancy Forum: Proceedings on Ethical and Legal Considerations.

To raise awareness and promote dialogue leading to action, this article provides proceedings on ethical and legal considerations associated with medicine use during pregnancy discussed during the 2014 DIA Medicines and Pregnancy Forum. A key focus of discussion at the forum was "When is it ethically appropriate to include or unethical not to include pregnant patients in clinical studies, and how can ethical barriers be addressed?" Also debated was the question "What are the most appropriate methods to collect and share data on medication use in pregnancy, and what is the best process for sharing such information?" Goals of the forum were to gain participant alignment on answers to these ethical questions, offer rationale for the answers, and provide insight into which stakeholders might be needed to facilitate discussion and action. Participants felt that under the right circumstances, drug research in pregnant women is justified and necessary.

Patient Engagement by Pharma-Why and How? A Framework for Compliant Patient Engagement.

Engagement is increasingly recognized as a decisive factor for health-related outcomes in people living with a medical issue. It is their experience that drives this engagement. Therefore, providers who seek to develop better solutions, including medicines, must gain a deeper understanding of the patient experience.

Clinical Data for Informed Medication Use in Pregnancy: Strengths, Limitations, Gaps, and a Need to Continue Moving Forward.

The objective of this paper is to explore the strengths, weaknesses, gaps, and needs in research on medication use in pregnancy, where opportunities have been bypassed to develop standards and collaborations for collecting data to better understand how medications can impact clinical outcomes in pregnant women and developing fetuses. The availability of existing data and the methods of its capture are reviewed, including registries, claims and health record databases, and meta-analyses. The paper focuses on why these efforts have not fundamentally provided benefit-risk information and clinical treatment algorithms for medication use in pregnant women.

A Proposed Framework to Address Needs of Clinical Data for Informed Medication Use in Pregnancy.

The objective of this paper is to communicate a proposed framework for addressing research limitations and communication barriers that contribute to a lack of data for making clinical treatment decisions about medication use in pregnancy. To address this global public health concern, a cross-stakeholder coalition composed of several workstreams is proposed. The intent is to foster collaborative discussion regarding potential solutions to address gaps in communication, engagement, and data generation and collection.

Spinal radiographic changes in ankylosing spondylitis: association with clinical characteristics and functional outcome.

Objective: To determine which patients with ankylosing spondylitis (AS) have radiographic spinal damage and to investigate the relation between radiographic spinal changes and limitations in physical function.

Methods: A cross-sectional nationwide study in Belgium of patients with AS under the care of a rheumatologist. The treating physician completed a questionnaire including clinical disease manifestations and laboratory findings (HLA-B27 and C-reactive protein), and classified spinal radiographs into 3 categories: (1) no AS-related spinal abnormalities; (2) syndesmophytes; and (3) spinal ankylosis.

The epidemiology of ankylosing spondylitis and the commencement of anti-TNF therapy in daily rheumatology practice.

Objectives: This study aimed to describe the epidemiology of ankylosing spondylitis (AS) in rheumatology practice at the beginning of the anti-TNF (tumour necrosis factor) era, and to evaluate the initiation of anti-TNF therapy in a clinical setting where prescription is regulated by the authority's imposed reimbursement criteria.

Methods: Between February 2004 and February 2005, all Belgian rheumatologists in academic and non-academic outpatient settings were invited to register all AS patients who visited their practice. A random sample of these patients was further examined by an in-depth clinical profile.