– Dr. Harsha K Rajasimha
The scientific program can be found here.
It will be my pleasure to welcome you to San Antonio, TX on Oct 30th 2017, for the third annual international conference on rare diseases and orphan drugs. Rare disorders affect over 350 Million people globally – which means if all people affected by one of ~7000 rare disorders are put together in a country, that would be the third most populous country in the world.
In the United States, we have over 30 Million patients suffering from these debilitating rare diseases. About 80% of the rare diseases are estimated to be genetic in origin and a half of those are early onset before 5 years of age. In 1983, US became the first country to adopt an orphan drug act (ODA) that incentivized biopharmaceutical industry to invest in research and development of orphan drugs. Some key incentives include: tax breaks on R&D expenses incurred on orphan drugs/products, seven years of market exclusivity on orphan drugs in USA even without other intellectual property (IP) protection, among other allowances.
We are in an exciting era of Precision Medicine with a growing number of orphan drugs approved and in the pipeline. Of the ~3,800 orphan drug designations issued by FDA OOPD since 1983, about 600 have resulted in marketing approval, the vast majority with orphan exclusivity. EU, Japan and several countries have adopted some form of ODA over the decades yet many countries are lagging behind – including the two most populous countries in the world – China and India.
Adopting a standard and consistent definition of rare disease is a critical pre-requisite milestone for a nation so that everything else can follow – including a national health policy, database of rare diseases, orphan designation, budget decisions, etc. The Organization for Rare Diseases India (ORDIndia) has played a vital catalytic role in accelerating the pace of progress by unifying all disease specific patient advocacy groups together to represent the collective voice of all stakeholders to the Government policy makers. India now has a definition: A disease is rare if it affects less than 1 in 2500 people. With the number of patients with rare diseases living in India estimated at over 70 million, getting them proper diagnosis and connecting them with global clinical studies is a big goal for #ORDIndia.
A number of international consortia and organizations have sprung up recently to support the rare disease revolution and for sharing of best practices for national umbrella organizations. These include: the international rare disease research consortium (#IRDiRC), Rare Diseases International (RDI), International Conference on Rare Diseases and Orphan Drugs (#ICORD), the NGO committee for rare diseases by United Nations. Numerous regional alliances are also being formed particularly in Asia including the Asia Pacific Alliance for Rare Disease Organizations (#APARDO) and Rainbow Across Borders. Conferences such as this one and the world orphan drugs congress where global organizations converge enable exchange of ideas, innovations and best practices benefit the cause of patients with rare diseases globally.
Whether you are a patient advocate, scientist, technologist, student, entrepreneur, pharmaceutical executive, biotech investor, or philanthropist, I truly hope you find this conference very informative and help you gain global insights for local action to continue in your own journey in this exciting area of rare diseases and orphan drugs.
You will have the opportunity to connect with speakers from organizations such as #Apostle #Elsevier #JohnsHopkins #NTTDataServices #Ordindia #QuintilesIMS #Raregenomics #VibrentHealth