The World Orphan Drug Congress USA is where the rare disease industry comes together to network, learn from each other, and discuss the latest orphan drug developments.

Pat Furlong of Parents Project Muscular Dystrophy on the World Orphan Drug Congress USA

One of the advocacy leaders in the rare disease community is Pat Furlong, President of Parents Project Muscular Dystrophy (PPMD). This April, Ms Furlong will be speaking at the upcoming World Orphan Drug Congress USA in Washington D.C. In this exclusive interview with Rare Disease Report, Ms Furlong explains why she thinks this is one of the most important rare …

Study concludes visits conducted at home improves recruitment/retention and speeds trials

GlobalCare Clinical Trials, Ltd continues expansion of patient-centric services to over 25 countries across 5 continents. GlobalCare provides selected clinical study visits at patients' homes, workplace or alternate settings to support phase I-IV trials across a variety of therapeutic areas and genomics research. CEO, Gail Adinamis, states that "our service model provides faster patient recruitment, better protocol compliance and higher …

The Advantages of NIPT (Non-Invasive Prenatal Testing) provided by Genpharm

As a follow-up to yesterday's post introducing NIPT on Total Orphan Drugs (our sister blog), below is a summary of the advantages of this method of testing. Download yesterday's and today's post as a Whitepaper here > The Advantages of NIPT (Non-Invasive Prenatal Testing) Non-Invasive Prenatal Testing, otherwise known as NIPT, is a new method of prenatal testing without harming …

World Orphan Drug Congress agenda

Here it is! The brochure for the World Orphan Drug Congress is ready and we are releasing it to Total BioPharma first.  Highlights from the agenda include: Ultra-orphan therapies – broad skills for small populationsMartin Mackay, EVP & Global Head of R&D, Alexion Pharmaceuticals How to optimise clinical development and demonstrate value for an orphan drugRogerio Vivaldi, SVP and Head …

Reimbursement: economics of orphan drugs and the impact of rarity on the estimation of cost effectiveness ration

Increasing pressures on health care budgets have led to a growing interest in the use of economic evaluation in reimbursement decisions for drugs and other health technologies. This has a great impact in the access of orphan drugs by rare disease patients. In this scenario, insurance companies are starting to assess the value for money by comparing the incremental costs …

Download presentation- Why transparency between stakeholders is essential when discussing pricing and reimbursement

In November 2012 over 250 delegates participated in the 3rd Annual World Orphan Drug Congress Europe. This event covers strategies and opportunities from Clinical Development and R&D, Market Access, Pricing & Reimbursement , Corporate Development and Drug Repositioning & Indication Expansion. Attendees at this highly successful event were fortunate enough to hear this exciting presentation delivered by Ri De Ridder, …

Download presentation- How are larger organizations viewing small biotech as innovative developers?

In November 2012 over 250 delegates participated in the 3rd Annual World Orphan Drug Congress Europe. This event covers strategies and opportunities from Clinical Development and R&D, Market Access, Pricing & Reimbursement , Corporate Development and Drug Repositioning & Indication Expansion. Attendees at this highly successful event were fortunate enough to hear this exciting presentation delivered by Jeannie Joughin, VP …

Download presentation- How are National Rare Disease Plans seeking to solve Market Access Challenges?

In November 2012 over 250 delegates participated in the 3rd Annual World Orphan Drug Congress Europe. This event covers strategies and opportunities from Clinical Development and R&D, Market Access, Pricing & Reimbursement , Corporate Development and Drug Repositioning & Indication Expansion. Attendees at this highly successful event were fortunate enough to hear this exciting presentation delivered by Ségolène Ayme, Director …