It has been acknowledged over recent years that, while the EU Orphan Drugs Regulation has stimulated research and development of orphan medicinal products in the EU, equitable and timely access to approved Orphan Drugs for rare diseases patients remains an issue. As underlined by the final conclusions and recommendations on Pricing & Reimbursement of the EU High Level Pharmaceutical Forum, “effective market access and utilisation vary strongly between and within Member States”.
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 [...]
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, [...]
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 [...]
Being diagnosed with cancer, or experiencing a recurrence, often puts individuals and their families in an emotional roller coaster ride. Moreover, treatment decisions often need to be taken quickly. To make an informed choice between the bad and the ugly, patients desperately seek for quality information. Whilst the political debate around “information to patients” and “advertising” has been going on for years as if a walled garden would still exist, or could still be built around Internet websites, European patients continue to suffer from the lack of access to quality information while the alluring bait of miracle healers, counterfeit online pharmacies and door-to-door advertisers continues to be omnipresent. “Information to Patients” now requires new courage, commitment and real action now – the current EU political momentum on “Information to Patients” is timely and welcome.
