Trinity Partners, a leading global life sciences consulting firm, announced that their client, Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc., has published results from one of the first-ever research studies measuring the considerable burden of patients with familial chylomicronemia syndrome (FCS) and the impact the disease has on their activities of daily living and employment. The results, from an interim analysis of the largest survey in patients with FCS, were published in Expert Review of Cardiovascular Therapy.
FCS, also known as lipoprotein lipase deficiency (LPLD) or type I hyperlipoproteinemia, is a rare genetic disease characterized by extremely high levels of triglycerides and increased risk of other health complications, including pancreatitis. IN-FOCUS, a comprehensive web-based research survey of patients with FCS, was designed to capture the associated burden of illness and impact on quality of life. Sixty patients from the United States diagnosed with FCS participated; patients described multiple symptoms spanning across physical, emotional and cognitive domains.
An analysis of the survey data found that patients with FCS saw five physicians on average with FCS-related symptoms before receiving a proper diagnosis. Nearly all respondents reported frequent, debilitating symptoms leading to reduced quality of life. Respondents also indicated that FCS significantly influenced their career choice and employment status, and resulted in substantial work loss.
Learn more about the study results: http://bit.ly/2oNlzbE
Read Endocrine Today’s article on the findings: http://bit.ly/2oaDxDE
He asserts that the first product to offer patients the ability to self-administer at home could drive separation from the pack. Read more: http://bit.ly/2nBn2Bm
Principal Jillian Godfrey Scaife recently spoke with Specialty Pharmacy Times about the impact that the FDA’s recent biosimilar interchangeability draft guidance will have on specialty pharmacy. Read the Q&A here: http://bit.ly/2jZdudr
Members of the Trinity team recently researched adherence to clinic recommendations among patients with phenylketonuria. Review their findings on Molecular Genetics and Metabolism: http://bit.ly/2kAM9lE
See her article “New Rules of Engagement: The Challenges Associated with Commercializing Biosimilars” here: http://bit.ly/2h3APKA
Trinity’s Ryan Million and Jillian Godfrey Scaife discuss the future of precision medicine and gene editing, respectively, in the November/December PharmaVOICE Year in Preview issue. View Ryan’s insight on precision medicine: http://bit.ly/2fZOamA. Read Jillian’s comments on gene therapy: http://bit.ly/2gaV0ZA.
Trinity Co-Founder and President John Corcoran discussed Trinity’s 2016 Drug Index and 20th anniversary on the Bloomberg Baystate Business Hour with Anne Mostue and Tom Moroney. Listen to the full program here: http://bloom.bg/2gzr59P. John’s segment begins at 7 minutes, 20 seconds.
The Trinity 2016 Drug Index argues that Biogen’s blockbuster multiple sclerosis treatment Tecfidera was the biggest commercial success of roughly two dozen novel drugs approved in 2013, but other biotech firms in the Bay State didn’t fare quite as well. In the study released on Wednesday, Trinity examined 22 novel drugs — those that represented a significant step forward in innovation — approved by the FDA in 2013. Trinity then ranked them based on commercial success, therapeutic success, and the cost and duration of research and development efforts. Not enough data was available to assess five other novel drugs approved in 2013. See the full article here: http://bit.ly/2fAhSBa
Trinity Partners announced today the findings of its first annual ‘Trinity Drug Index,’ a comprehensive research-based report which assessed 22 novel drugs approved by the FDA in 2013. The Index rates drug performance as measured by a retrospective look at commercial success relative to therapeutic value and R&D complexity, assigning each drug a score in those three areas as well as an overall composite score.
Top findings of this year’s Trinity Drug Index include:
- Sovaldi, which treats Hepatitis C, received the highest ranking with an overall score of 4.4 out of 5
- Most of the top performers were specialty drugs
- Drugs for primary care markets such as COPD and diabetes tend to show weaker differentiation and limited commercial performance, exacerbated by significant R&D expense
- Commercial underperformance given significant therapeutic value is rare, and could be due to a rapidly changing competitive environment
- Well-executed business strategy may boost commercial performance of drugs that are not vastly superior therapeutically
- Moving forward, it will be more critical than ever to demonstrate a product’s value vis-à-vis existing competition
If you would like to read the full whitepaper, it is available for free on the Trinity Partners website HERE.
New science drives biopharma interest in neurological conditions.
Read the article HERE