World Orphan Drug Congress USA 2017

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[survey results] Patients Support Partnerships between Advocacy Groups and Pharmaceutical Companies

  A survey conducted by Rare Patient Voice of over 3,000 US patients and caregivers across several hundred diseases and conditions, found that  »

[[Whitepaper]] In the internet we trust? - Discerning quality rare disease information

Diagnosed with a rare disease?  Google it.  Receive a mess of returns, or perhaps nothing. Those living with a rare condition and their fa »

[Whitepaper] Challenging Conventional Patient Recruitment and Care Strategies for Rare Disease Studies

Medicines for rare diseases accounted for 41% of new FDA-approved drugs in 2016.   However, developing new treatments for orphan indications is  »

[[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches

The landscape is changing. Clinical development plans no longer depend solely on the outcomes of a phase 3 trial, as companies are adapting their app »

[[Whitepaper]] Predictive Analytics: A Case Study in Machine-Learning and Claims Databases

  Authors: Kvancz DA, Sredzinski MN, Tadlock CG. Publication: American Journal of Pharmacy Benefits   The study demonstrat »

Whitepaper: Orphan Diseases, Bringing the Trial to the Patient- Illingworth Research Group

An orphan disease may be a rare disease presenting within a small population or can also include conditions that are not receiving adequate attention »

[[whitepaper]] Life Changing Launch- AmerisourceBergen

Manufacturer perspectives on maximizing success for orphan drug commercialization   You’re a manufacturer of orphan drugs, which means t »

[[WHITEPAPER]] Global Rare Disease Drug Development: Understanding and mitigating its complexities

This insight brief examines the varied definitions and regulatory pathways for rare disease therapies. The authors describe QuintilesIMS’s pro »

[[Whitepaper]] Orphan Drugs in Germany

The AMNOG, the legislative framework governing the market access of innovative prescription drugs in Germany, has implicated a paradigm shift in the  »

[[Whitepaper]] Critical Considerations for Rare & Orphan Disease Trial Planning

A question- first approach to Rare Disease Studies  Overcoming the unique challenges of rare disease drug development starts with asking the r »

Towards a New Way of Evaluating Orphan Drugs at CADTH

  McKesson Canada is the leading provider of market access services connecting patients to orphan drugs and other drug therapies within the Ca »

Delivering Hope - A First-in-Class Orphan Drug Comes to Market

Hope is perhaps the one thing that 30 million Americans living with a rare disease have to hold on to. On average, it takes more than 7 years and sev »

Understanding the Unique Challenges and Opportunities in Rare Disease Drug Development

Nicole Sweeny, Shire joined us at the World Orphan Drug Congress USA 2016 to deliver her presentation on  "Understanding the Unique Challe »

Tackling the Rare Diseases therapy deficit –the nonsense mutation read-through agent

Mark Rothera, PTC Therapeutics, long time friend of the World Orphan Drug Congress USA, joined us once again in 2016. His presentation  "T »

Adaptive Biomedical Innovation: Innovating How We Innovate

Rare Disease research is innovative. The Orphan Drug industry has always embraced the NEW. This is why at the  World Orphan Drug Congress USA ,  »

Maintaining a relationship based on trust: finding common goals with a KOL and its institution

In a multi-stakeholder industry such as that of Orphan Drugs, communication is vital to ensuring strong, effective working relationships. Charles Sta »

‘Significant Benefit’ in the European Regulatory Framework for Orphan Medicinal Products

Orphan Drug regulation has changed over the past 30 years. New legislation like Prescription Drug User Fee Act (PDUFA VI) and 21ST Century Cures »

What is the Future Vision for Patients?

What is the Future Vision for Patients? A question posed by many an Orphan Drug stakeholder, often at the  World Orphan Drug Congress USA  »

Creating Medicines with Real Value for Orphan Diseases

Emil D. Kakkis, M.D., Ph.D., President and Founder joined us at the World Orphan Drug Congress USA 2016, to give a detailed keynote presentat »

Re-engineering the process rather than new legislations: Europe’s approach to get more, better, faster, cheaper orphan drugs

Yann Le Cam, Chief Executive Officer, EURORDIS joined us at the World Orphan Drug Congress USA 2016 to detail EURORDIS' view and plan for t »

A systematic approach to orphan drug discovery: starting with the unmet needs of payers and patients

Download Ed's presentation to discover more about: Identifying unmet needs Drug development Orphan drugs* approved in 2014 by nati »

How to capture the patients and caregivers perspectives in rare diseases?

Benoit  Arnould ,  Senior Director, Patient-Centered Outcomes,  MAPI  Group  joined us at the  World O »

Download Pursuing Accelerated Approval for Rare Disease Therapeutics

Download Alvin's presentation here for more information on: The Sparsentan Case Study Sparsentan for the Treatment of Focal Segmental »

How Are Patient Advocates Changing Clinical Research? Download here >>

Patient advocacy groups are just starting to realize their enormous potential to help advance treatment of cancer and rare diseases by supporting  »

Download the Rare Oncology white paper now

Download the whitepaper prepared by Premier Research here >> In this white paper, we provide insight on the major issues being raised in th »