FDA: Enrichment Strategies to Improve Efficiency of Drug Development
Conclusion
Temple noted that there are some issues with predictive enrichment. One is that you always believe the characteristic you use to enrich predicts the good responders; it may not do this as well as you hope. So it’s very important to characterize the test that leads you to select those patients; then see whether it’s true that patients with the characteristic always (or most of the time) respond, and that patients without the characteristic don’t respond very much.
An issue to consider in any enrichment design is how much you need to study the people who don’t have the enrichment characteristic...
Source: Policy and Medicine - February 12, 2013 Category: Health Medicine and Bioethics Commentators Authors: Thomas Sullivan Source Type: blogs
USMLE Questions – Characteristic Disease Findings
The United States Medical Licensing Examination (USMLE) is designed to emphasize knowledge of clinical scenarios and clinical pearls, even on Step I. Listed below are some commonly encountered disease findings and characteristics.
Feature
Disease
45, X chromosome
Turner’s syndrome
5-HIAA increased in urine
Carcinoid syndrome
Aganglionic rectum
Hirschsrpung’s disease
Apple-core sign on barium enema
Colon cancer
Arched back (opisthotonos)
Tetanus
Argyll-Robertson pupil
Syphilis
Ash leaf on forehead
Tuberous sclerosis
Auer rods
Acute myelogenous leukemia
Austin Flint murmur
Aortic regurgitation...
Source: Inside Surgery - January 18, 2013 Category: Surgeons Authors: Editor Tags: Surgpedia USMLE diseases findings VMA water hammer pulse Source Type: blogs
Pipeline 2012
39 drugs approved in the USA in 2012
US Drug approvals in 2012 have reached a 15-year high with regulators giving the thumbs up to 39 new drugs.
The figures from the US Food and Drug Administration show approved drugs were up on 2011, when 30 new medicines were given marketing authorisations. Of the 39 approved in 2012, 11 were for cancer treatments and almost 20 were designated orphan drug status.
Some of the drugs allowed on the US market in 2012 included: Pfizer’s leukaemia drug Bosulif (bosutinib); Ariad Pharmaceutical’s leukaemia drug Iclusig (ponatinib); Johnson & Johnson’s multi-drug resistant TB drug Si...
Source: PharmaGossip - January 6, 2013 Category: Pharma Commentators Authors: insider Source Type: blogs
Cystic Fibrosis and the Cost of Medicine
It’s difficult not to get excited about the prospect of genetic medicine, but as the results of research into genetics and personalized medicine continue to soar, so do the prices of disease-treating drugs. A recent story on NPR detailed the history behind...(read more) (Source: ADVANCE Discourse: Lab)
Source: ADVANCE Discourse: Lab - January 4, 2013 Category: Pathologists Authors: Michael Jones Tags: Current Events Molecular Diagnostics Pathology Research Source Type: blogs
Hello My Virtual Friend
Today's guest post comes from Emily Schaller, Founder/CEO of Rock CF Foundation. Follow her on twitter @RockCFem
Technology is both a scary and amazing thing. Humans around the globe are plugged in and online all day and all night waiting for work emails, social media updates, breaking world news Tweets, and so much more. I must admit that I am guilty of all of these things. Every day you can turn on the news and see something wonderful or something tragic that has come from a social media.
Digital communication has been a huge benefit for myself and patients with cystic fibrosis and other illnesses around the world. Bec...
Source: ePharma Summit - January 4, 2013 Category: Medical Marketing and PR Tags: social media and healthcare ePharma Source Type: blogs
Express Scripts CEO To Kids With Cystic Fibrosis: Find Another Way To Stay Alive
(Source: drugwonks.com Blog)
Source: drugwonks.com Blog - September 6, 2007 Category: Pharmaceuticals Source Type: blogs
