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Edition of 13 November 2017

International News

NIH partners with 11 leading biopharmaceutical companies to accelerate the development of new cancer immunotherapy strategies

A report published by the NIH refers to the Partnership for Accelerating Cancer Therapies (PACT) a five-year plan. A fund of $53.6 million was award to support four cancer immune monitoring and analysis centres and one cancer immunologic data commons (CIDC) for adult and paediatric immunotherapy trials. The plan’s goals over the five years are:

To facilitate systematic and uniform clinical testing to provide a better understanding of the mechanisms of response and resistance to cancer therapy;
To coordinate clinical efforts by sharing information between stakeholders.

It is hoped that this partnership will help deliver innovative immunotherapy strategies to more patients.

Biopontis Alliance for Rare Diseases recognized by Ministry of Health of Belgium

Biopontis Alliance for Rare Diseases has recently been recognized by the Ministry of Health of Belgium. The aims of Biopontis are to give a worldwide access to therapies to patients living with a rare disease and, to develop more therapies by working on future projects by collaborating with experts, academic institutes and International institutions altogether.

Public Attitudes towards Gene Therapy in China

A study published in Molecular Therapy identifies public attitudes and perceptions towards gene therapy in China. The study shows that these attitudes and perceptions are mainly influenced by demographic factors.

The results show that women are more likely to accept gene therapy for children with an inherited disease and in germline cells, and on the other hand, men are more accepting of the application of gene editing compared to women. Overall, the study shows that respondents with personal knowledge of a fatal, debilitating, or inherited disease were more accepting gene therapy.

Participation in interdisciplinary meetings on genetic diagnostics (NGS)

An article published in the Journal of Human Genetics describes how interdisciplinary collaboration between various services can contribute to the success of next-generation sequencing (NGS) diagnostics. It highlights factors that could contribute to successful interdisciplinary activities, namely coordination, pooling of resources, individual learning and role blurring.

The article concludes, following linguistic analysis of transcripts of interdisciplinary meetings, that improved communication is necessary to improve the efficacy of these meetings.

The United Kingdom Sets Limits on Experimental Treatments

Robert D.Truog, MD from Harvard Medical School has published in the Journal of the American Medical Association a viewpoint that identifies the limits of experimental treatments after the Charlie Gard case in the United Kingdom that generated a media frenzy and great public and political debate across the globe. The author highlights the fundamental debates at play in this case. The article shows that societies and their governments have the obligation to regulate health care systems in ways that can ensure the best interest of patients and fairness. The article underlines the importance of scientific values, which must encourage the integrity of medical practice when a treatment fails to meet a reasonable threshold of scientific justification.

Third party interpretation of raw genetic data: an ethical exploration

A study published in the European Journal of Human Genetics refers to recent regulations targeting direct-to-consumer genetic testing (DTC-GT) and describes DTC-GT activities and their ethical implications. The study highlights the existence of third-party interpretation (TPI) services and the ethical challenges surrounding them.

Recontacting in clinical practice: the views and expectations of patients in the United Kingdom

A study published in the European Journal of Human Genetics reports patient expectations concerning the possibility of being re-contacted in clinical practice. The study aimed to identify the viewpoint of patients with a rare disease concerning the possibility of being re-contacted in light of new genetic findings regarding their disease. Some of the respondents think that it a professional’s responsibility to re-contact patients when new information is available following an improvement in the knowledge of a disease which could benefit both patients and their families. For others, it can be seen as desirable but unnecessary and should be managed with care so as not to trigger potentially negative psychological effects as a result of receiving new information.

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: 2009-13 cohort

A study published in the British Medical Journal identifies drugs approved by the European Medicines Agency (EMA) during 2009-13 that entered the market without demonstrating evidence of benefit as regards survival or quality of life and that after 3 years following approval, they can not yet demonstrate that they have improved survival or quality of life for most cancer indications. Among the oncology drugs cited in the study, there are seven orphan drugs. The authors suggest that the clinical meaningfulness of the benefits in terms of survival or quality of life offered by these drugs has been overestimated. This overestimation can have a negative economic impact on patients and their families.

Importance of Drug Development at the Portfolio Level

An article published in the Journal of American Medical Association describes the importance of clinical decision making, human protection, and policy in the development of new treatments.

Drug development trials in portfolios test hypotheses under different conditions, and authors suggest that decision makers should leverage evidence within portfolios to avoid potential exaggerated estimates arising from trials. The aim is to protect research study volunteers, patients, and payers due to large observed treatment effect, which is likely to represent an overestimate of the real benefit.

The promises and challenges of using gene mutations for patient stratification in follicular lymphoma

An article published in the Blood journal refers to the use of gene mutations for patient stratification in follicular lymphoma. The aim of the article is to highlight the promises and challenges of integrating gene mutation into clinically applicable stratification for patients. The authors emphasise that gene mutations can also serve as prognostic and/or predictive biomarkers for follicular lymphoma. The main challenge is to make both the analysis of the data and the analysis of the analysis available to all interested persons.

ERN-LUNG

ERN-LUNG just won the competition for a grant from the European Union (EU) and is willing to build an infrastructure that enables patient data management and to establish existing interoperable registries. ERN-LUNG has been raising money to support new projects and aims to keep it this way to bring more resources to lead to successful goals. ERN-LUNG is looking forward to addressing its main objective to serve patients and colleagues in a near future and is spreading the word to the medical community.

ERN-EYE Ontology Workshop

The first ERN-EYE workshop was held at Mont Ste-Odile in Alsace from 9 to 11 October 2017. The workshop was successful with up to 60 ERN-EYE members participants. It was opened by the coordinator Hélène Dollfus and was dedicated to the revision of the Orphanet classification revision and HPO terms revision.

Four major clinical themes were presented during this workshop: retina paediatrics, neuro-ophthalmology or anterior segment giving the opportunity to experts to work together according to their speciality. The workshop was very successful and allowed major advances in the network's activities.

The “Odile” workshop was ERN-EYE first achievement. The classification and well-designed ontology helped build an efficient IT platform and an EU-wide registry.

Editorial
Political news
Scientific news
And also...
- Courses & Educational Initiatives and Events
- Grants

International News

European News

Orphan Drugs

Study Design

European Reference Networks

Summary of the edition of 13 November 2017

Editorial

Political news

Scientific news

And also...

Edition of 13 November 2017

International News

NIH partners with 11 leading biopharmaceutical companies to accelerate the development of new cancer immunotherapy strategies

European News

Biopontis Alliance for Rare Diseases recognized by Ministry of Health of Belgium

Ethical Legal Social

Public Attitudes towards Gene Therapy in China

Participation in interdisciplinary meetings on genetic diagnostics (NGS)

The United Kingdom Sets Limits on Experimental Treatments

Third party interpretation of raw genetic data: an ethical exploration

Recontacting in clinical practice: the views and expectations of patients in the United Kingdom

Orphan Drugs

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: 2009-13 cohort

Study Design

Importance of Drug Development at the Portfolio Level

The promises and challenges of using gene mutations for patient stratification in follicular lymphoma

European Reference Networks

ERN-LUNG

ERN-EYE Ontology Workshop

Summary of the edition of 13 November 2017

Editorial

Political news

Scientific news

And also...