“Orphan drugs” are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare.
These drugs are called “orphan” because the pharmaceutical industry has little interest under normal market conditions in developing and marketing products intended for only a small number of patients suffering from very rare conditions.
The 1st US Conference on Rare Diseases and Orphan Products, co-organised by NORD (National Organization for Rare Disorders, USA) and the DIA (Drug Information Association), took place in Washington DC from 11 – 13 October 2011. Organised in partnership with the FDA, NIH and EURORDIS, this first conference was a huge success, assembling over 250 [...]
I came home late from London last night. We’d just had a brainstorm with a group of friends to think about how to build a strong rare disease movement in the UK. As you’ll see from my previous posts, I’m convinced that more can be done to create a mass social movement for rare diseases [...]
Yesterday, I was not happy. I’d been talking to a major funder, trying to find out more about whether we could apply for funding for our fast moving scientific research and upcoming clinical trials for Alkaptonuria (AKU), the rare genetic disease affecting my two sons. The funder was very helpful in explaining the different funding [...]
A recent article published in the Journal of the American Medical Association (JAMA) discussed a comparison of clinical trials for orphan vs. non-orphan oncology drugs. The article questioned whether safety and efficacy standards were as stringent in the orphan trials as in the non-orphan trials. A news story circulated after the article was published went [...]
Date: 11–14 May 2011
Venue: Antalya, Turkey
TIF’s largest and longest-established international event for medical professionals, scientists, patients and parents is the biennial International Conferences on Thalassaemia & Haemoglobinopathies, organised jointly with the TIF International Thalassaemia Patients/Parents’ Conference, which attracts hundreds of delegates from all corners of the world. TIF has had impressive results over the [...]
What are the key factors that could explain the rise of the rare disease movement and help us understand how to make it grow faster and more effectively?
A few years ago I finished my PhD in social movement studies, a sub-group of sociology that studies why social movements rise and fall. My PhD was on [...]
Haemoglobin (Hb) disorders, including thalassaemia and sickle cell anaemia, are a group of genetic blood disorders classified as rare diseases as they affect less than 1 in 2000 inhabitants of the population. Approximately 7% of the global population carry an abnormal haemoglobin gene, and more than half a million affected children are born each year. [...]
We’ve just finished our fourth international conference on AKU (Alkaptonuria), here in Cambridge. It was a huge success, although it raised, once again, the questions we face as we work to find treatments for rare diseases.
Sixty scientists, industry representatives, patients and relatives attended, including from the US, Slovakia, Jordan, France, Italy, the UK, the Netherlands, [...]
I’m just back from the Partnering for Cures conference in New York (www.partneringforcures.org), where we were presenting our international findAKUre project on Alkaptonuria and trying to raise funds for our plans for new clinical trials for treatments. (You can download our presentation here: http://www.partneringforcures.org/2010_innovators/18_findAKUre.html).
The conference, organised by the Faster Cures action tank, was impressive. It [...]
Last week was the first World Orphan Drug Congress, held in Geneva. It brought together hundreds of participants from across the industry. I attended as one of the few representatives of a patient group. I was hugely impressed. It was inspiring to see how more and more companies – including big pharma – are entering [...]
