Regulation

Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positive

Key Points: 


Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positive

Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Key Points: 


Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive

Key Points: 


Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive

Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Key Points: 


Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positive

Key Points: 


Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positive

Orphan designation: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positive

Key Points: 


Orphan designation: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positive

Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positive

Key Points: 


Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positive

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Retrieved on: 
Thursday, April 18, 2024

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Key Points: 


Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

EMA/FVE info session on restrictions for the use of certain antimicrobials in animals, Online, European Medicines Agency, Amsterdam, the Netherlands, from 23 May 2024, 15:00 (CEST) to 23 May 2024, 16:00 (CEST)

Retrieved on: 
Thursday, April 18, 2024

During this session, participants will have the opportunity to engage with experts who contributed to the development of the available guidance.

Key Points: 
  • During this session, participants will have the opportunity to engage with experts who contributed to the development of the available guidance.
  • Registration
    Please register for the webinar using the below online form:
    EMA/FVE info session on restrictions for the use of certain antimicrobials in animals
    Please note this event will be recorded and published on the EMA website.
  • By registering for this event, you are consenting to EMA to process your personal data in accordance with Regulation (EU) 2018/1725.
  • EMA's data protection notice below explains how personal data is processed by the Agency and via the tool Webex:

NZ’s mental healthcare is in crisis – but research shows us how to shorten wait times and keep staff

Retrieved on: 
Wednesday, April 10, 2024

New Zealand’s youth mental healthcare continues to experience a rolling crisis with long waiting times for services.

Key Points: 
  • New Zealand’s youth mental healthcare continues to experience a rolling crisis with long waiting times for services.
  • There have been calls to expand the mental health workforce and to diversify the range of available services.
  • As our research shows, it can help to shorten bulging waiting lists and retain staff in the workforce.

Training is necessary, but not sufficient

  • These are often talking therapies that have been proven effective for most people through research studies such as clinical trials.
  • Many mental health services deliver therapies based on dialectical behaviour therapy to help young people and adults improve their emotional regulation.
  • There will always be a need to develop and adapt therapies, but a central challenge is better implementation of those we already have.

Better implementation of proven therapies

  • It studies methods to enhance the adoption, implementation and sustained delivery of evidence-based practices to improve the quality of routine care.
  • Rather than asking tired clinicians to do more, implementation science can identify the influences on clinician behaviour and target supports accordingly.
  • Implementation science teaches us that providing guidelines or training in evidence-based therapy is necessary, but often not enough to achieve quality care.

Quality in action

  • Their aim is to promote national standards for care quality in partnership with whānau, consumers and local communities.
  • Beyond mental health, apparently simple solutions such as surgical checklists have been shown to substantially improve quality, even in resource-limited settings.
  • Service leaders, funders and policymakers must urgently consider how we can best equip existing and new staff to deliver quality care, based on insights from implementation science.


Melanie Woodfield works as a clinical psychologist for Te Whatu Ora (Health New Zealand). She receives funding from the Health Research Council of New Zealand. Hiran Thabrew does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.