From the EveryLife staff, posted Feb. 1, 2012 Please call Representative Waxman’s Office at 202-225-3976 and tell his office that, as a parent with a child with a rare disease, you support HR 3737, the ULTRA Act. This morning, Representative Henry Waxman (D-CA), Ranking Member of the Energy and Commerce Committee voiced concerns about efforts … Continue reading
If you or a family member suffer and live with a rare disease, then drug development and regulatory approval processes aren’t just an abstract concept to you: such issues may literally mean life and death for you or a loved one. TAKE ACTION to Support ULTRA: “Click Here to ask your Representatives to co-sponsor H.R. … Continue reading
Initiatives to De-Risk Drug Development Could Include Redesigning Clinical Trial Requirement FDA officials have pointed out that orphan drugs must prove probable benefit and safety to the same degree as all other drugs. The complexity of these diseases added to FDA’s stance on approval translates into the fact that drug development for orphan diseases can … Continue reading
In November 2010, the American Society of Clinical Oncology convened a summit on drug shortages with the American Society for Health-System Pharmacists, the American Society of Anesthesiologists, FDA and the Institute for Safe Medication Practices. Despite all this discussion, no simple answer is in sight, and the number of drugs in short supply grows daily Continue reading
WASHINGTON, D.C. – The statistics on drug development are no secret: for every 10,000 compounds discovered, only one gets approved, and it usually takes 14 years and $1.2 billion to reach the market. “Surely there must be bottlenecks that can be tackled,” National Institutes of Health Director Francis Collins said in a speech Monday at … Continue reading
Business Week article on EU legal ruling (Updates with lawyer comment starting in 10th paragraph, researchers reactions starting in 13th paragraph.) Oct. 18 (Bloomberg) — The European Union’s highest court said that stem-cell research involving human embryos can’t be patented, in a ruling that scientists called “devastating” for medical research. Inventions based on the use … Continue reading
October 7, 2011 The National Institutes of Health has earmarked $50 million for the research and development of new treatments for rare and neglected diseases. The funds will go toward collaborating with researchers through a drug discovery and development program called Therapeutics for Rare and Neglected Disease (TRND). Recently accepted into TRND is Afraxis, a … Continue reading
WASHINGTON, Oct. 5, 2011 — The National Organization for Rare Disorders (NORD) and about 30 of its advocacy partners have sent letters to key officials at the Food and Drug Administration (FDA) seeking more frequent and regular opportunities to provide input on decisions related to relative risks and potential benefit for new drugs and medical … Continue reading
Developing a coordinated approach to access to orphan drugs and care for rare disease sufferers in the EU. This includes consideration of “compassionate use” programs for nonapproved drugs. Continue reading
The administration has become so concerned about the slowing pace of new drugs coming out of the pharmaceutical industry that officials have decided to start a billion-dollar government drug development center to help create medicines. Continue reading
Press release from Shire 22 Sep 2011 PHILADELPHIA, PA – September 22, 2011 – Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces an update on the approval status and availability of ProAmatine® (midodrine HCl), a medicine approved for the treatment of symptomatic orthostatic hypotension (SOH). Shire has engaged in consistent dialogue … Continue reading
A severe shortage of drugs for chemotherapy, infections and other serious ailments is endangering patients and forcing hospitals to buy life-saving medications from secondary suppliers at huge markups because they can’t get them any other way. Continue reading