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Nov. 16, 2012

TOPIC: Advocacy, Industry, Medical, Patients & Members, Research

Risk and Benefit, Effectiveness and Safety, Drug Approval, Drug Use, and Rare Disorders

Posted at November 16, 2012 5:44 pm by E. Michael D. (Mike) Scott

The terms risk, benefit, effectiveness, and safety are key to the approval of all new medical treatments (and of previously approved treatments for new uses). However, there is a vast divide between the concepts of relative risk and relative benefit (for a well defined group of patients) and the concepts of effectiveness and safety. The distinctions between these concepts are particularly important in the world of rare diseases. read more >

TAGS: biomarkers, FDA, FDASIA, Huntington's disease, Mike Scott, risk benefit

Nov. 07, 2012

TOPIC: Advocacy, Industry, Medical, Patients & Members, Research

Could You Be a Reviewer for PCORI?

Posted at November 7, 2012 3:39 pm by E. Michael D. (Mike) Scott

I got a message just this morning telling me that the evolving Patient Centered Outcomes Research Institute (PCORI) is still looking for a wide variety of “non-scientist stakeholders,” including patients, caregivers, and advocacy group representatives, to act as reviewers of research grant applications submitted to PCORI in response to their recent funding announcements. read more >

TAGS: Mike Scott, patient centered outcomes, PCORI

Nov. 02, 2012

TOPIC: Featured News, Industry, Medical, Research

Glybera Becomes First-ever Gene Therapy Approved in Europe

Posted at November 2, 2012 7:37 pm by E. Michael D. (Mike) Scott

According to a report on the BBC News web site today, the European Commission has — for the very first time anywhere in the Western world — given final approval to market a gene therapy that corrects an inborn error of metabolism for the treatment of any type of human genetic disorder. read more >

TAGS: European Commission, Gene therapy, Glybera, Lipoprotein Lipase Deficiency, LPLD, Marketing Approval, Michael Scott

Nov. 01, 2012

TOPIC: Advocacy, Industry, Medical, Patients & Members, Research

Making the Case for Rare Disease Research

Posted at November 1, 2012 1:45 pm by Jason Barron

There are many reasons why one might support research into a specific rare disease. Perhaps a family member or other loved one has fallen ill to the disease. read more >

TAGS: Biomedical research, Jason Barron, NIH, R & D investment, Rare disease research, Scientific Discovery

Oct. 31, 2012

TOPIC: Medical, Patients & Members, Research

Creative Collaborations May Be Key to Reducing Chemotherapy Risks

Posted at October 31, 2012 1:33 pm by Marsha Lanes

My friend’s 18-year-old son died recently following a 6-month battle with acute myeloid leukemia. There are many different stories I could tell about Tucker’s experience that are all too familiar to those in the rare disease community: read more >