Title Page / Table of Contents
Public Health Genomics 2016;19:125-128 (Source: Public Health Genomics)
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Early Patient Access to Medicines: Health Technology Assessment Bodies Need to Catch Up with New Marketing Authorization Methods
National and international medicines agencies have developed innovative methods to expedite promising new medicines to the market and facilitate early patient access. Some of these approval pathways are the conditional approval and the adaptive pathways by the European Medicines Agency (EMA); the Promising Innovative Medicine (PIM) designation and the Early Access to Medicines Scheme (EAMS) by the Medicines and Healthcare Products Regulatory Agency (MHRA), as well as the Fast Track, Breakthrough or Accelerated Approval methods by the Food and Drug Administration (FDA). However, at least in Europe, these methods cannot achi...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Personalised Medicine and Scarce Resources: A Discussion of Ethical Chances and Challenges from the Perspective of the Capability Approach
In the aftermath of the economic crisis that started in 2008, resources have become scarcer than ever in some countries, also in health care. Priority setting and rationalisation of existing resources also affect pharmaceutical innovations, including those that would contribute to what is called personalised medicine. In this paper, we will highlight the ethical issues surrounding rationalisation and its impact on personalised medicine through the lens of the capability approach. Thereby, challenges to and opportunities for personalised medicine will be examined, assessing how they affect patients' ‘real options' to acce...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Gene-Tailored Treatments for Brain Disorders: Challenges and Opportunities
Brain disorders pose major challenges to medicine and treatment innovation. This is because their spectrum spans inflammatory, degenerative, traumatic/ischaemic, and neoplastic disease processes with a complex and often ill- understood aetiology. An improved genetic and genomic understanding of specific disease pathways offers new approaches to these challenges, but at present it is in its infancy. Here, we review different aspects of the challenges facing neuromedicine, give examples of where there are advances, and highlight challenges to be overcome. We see that some disorders such as Huntington's disease are the produc...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Accelerating the Development and Validation of New Value-Based Diagnostics by Leveraging Biobanks
The challenges faced in developing value-based diagnostics has resulted in few of these tests reaching the clinic, leaving many treatment modalities without matching diagnostics to select patients for particular therapies. Many patients receive therapies from which they are unlikely to benefit, resulting in worse outcomes and wasted health care resources. The paucity of value-based diagnostics is a result of the scientific challenges in developing predictive markers, specifically: (1) complex biology, (2) a limited research infrastructure supporting diagnostic development, and (3) the lack of incentives for diagnostic deve...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
A Conceptual Framework of Mapping Access to Health Care across EU Countries: The Patient Access Initiative
Research evidence suggests that access to health care is the key influential factor for improved population health outcomes and health care system sustainability. Although the importance of addressing barriers in access to health care across European countries is well documented, little has been done to improve the situation. This is due to different definitions, approaches and policies, and partly due to persisting disparities in access within and between European countries. To bridge this gap, the Patient Access Partnership (PACT) developed (a) the ‘5As' definition of access, which details the five critical elements (a...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Enabling Equal Access to Molecular Diagnostics: What Are the Implications for Policy and Health Technology Assessment
This article will consider the perspectives of different stakeholders (patients, health care payers, health care professionals, and manufacturers) on the provision of a research-enabled, patient-focused molecular diagnostics platform that supports optimal patient care. Through the discussion of specific case studies, and building on the experience from countries that have successfully integrated molecular diagnostics into clinical practice, this article will discuss the necessary evolutions in policy and health technology assessment to ensure that patients can have equal access to appropriate molecular diagnostics.Public H...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
The Value of Companion Diagnostics: Overcoming Access Barriers to Transform Personalised Health Care into an Affordable Reality in Europe
This article outlines some of these market access barriers for companion diagnostics in the EU, including reimbursement challenges specific to some member states (Germany, the UK, and France). Furthermore, proposals addressing barriers and increasing timely patient access to companion diagnostics in the EU are presented.Public Health Genomics 2016;19:137-143 (Source: Public Health Genomics)
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Shooting for the Moon or Flying Too Near the Sun Crossing the Value Rubicon in Precision Cancer Care
In his last two State of the Union addresses, President Barack Obama has focused on the need to deliver innovative solutions to improve human health, through the Precision Medicine Initiative in 2015 and the recently announced Cancer Moonshot in 2016. Precision cancer care has delivered clear patient benefit, but even for high-impact medicines such as imatinib mesylate (Glivec) in chronic myeloid leukaemia, the excitement at the success of this practice-changing clinical intervention has been somewhat tempered by the escalating price of this ‘poster child' for precision cancer medicine (PCM). Recent studies on the costs ...
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Access for All: A Personalised Approach
Public Health Genomics 2016;19:129-131 (Source: Public Health Genomics)
Source: Public Health Genomics - May 30, 2016 Category: Genetics & Stem Cells Source Type: research
Do Public Involvement Activities in Biomedical Research and Innovation Recruit Representatively A Systematic Qualitative Review
Conclusion: Representation of study samples in PIAs in biomedical research and innovation is currently not reported systematically. Improved reporting on representation would not only improve the validity and value of PIAs, but could also contribute to PIA results being used more often in relevant policy and decision-making processes.Public Health Genomics (Source: Public Health Genomics)
Source: Public Health Genomics - March 31, 2016 Category: Genetics & Stem Cells Source Type: research
Do Public Involvement Activities in Biomedical Research and Innovation Recruit Representatively? A Systematic Qualitative Review
Conclusion: Representation of study samples in PIAs in biomedical research and innovation is currently not reported systematically. Improved reporting on representation would not only improve the validity and value of PIAs, but could also contribute to PIA results being used more often in relevant policy and decision-making processes.Public Health Genomics (Source: Public Health Genomics)
Source: Public Health Genomics - March 30, 2016 Category: Genetics & Stem Cells Source Type: research
Status of Emergency Contraceptives in Europe One Year after the European Medicines Agency's Recommendation to Switch Ulipristal Acetate to Non-Prescription Status
Conclusions: An EMA recommendation can strongly contribute to the harmonization of a drug's legal status in the EU. In most European countries, ulipristal acetate and/or levonorgestrel are now freely available.Public Health Genomics (Source: Public Health Genomics)
Source: Public Health Genomics - March 29, 2016 Category: Genetics & Stem Cells Source Type: research
The Quality of Rare Disease Registries: Evaluation and Characterization
Conclusions: The quality of RDRs is usually associated with a good oversight and governance mechanism and with durable funding. The results suggest that RDRs would benefit from support in management, information technology, epidemiology, and statistics.Public Health Genomics (Source: Public Health Genomics)
Source: Public Health Genomics - March 21, 2016 Category: Genetics & Stem Cells Source Type: research
Sickle-Cell Disease in Nigerian Children: Parental Knowledge and Laboratory Results
Conclusion: There is the need to promote public awareness about SCD and the benefit of early diagnosis, quality assurance in laboratory diagnosis and institution of sustainable patient care pathways.Public Health Genomics (Source: Public Health Genomics)
Source: Public Health Genomics - March 19, 2016 Category: Genetics & Stem Cells Source Type: research
