Experts to talk rare and orphan diseases, patient experience at webinar

By Jenni Spinner

- Last updated on GMT

(AlenaPaulus/iStock via Getty Images Plus)
(AlenaPaulus/iStock via Getty Images Plus)

Related tags Rare disease orphan disease Webinar Fda Patient centricity

On July 7, a group of seasoned industry leaders will offer insights at Rare/orphan diseases, special patient population, a focused, free online event.

While one rare disease might impact a relatively small segment of the global population, added up the patients living with these conditions are a big deal. According to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases affecting between 2m and 30m people in the US alone—about one in every 10 people in the country.

As a result, rare and orphan diseases are a big deal to the pharma industry. There exists a wealth of opportunities for drug companies and their research partners if they understand the particular needs of such patients and the conditions they face.

For Rare/orphan diseases, special patient population​, ​the Outsourcing-Pharma team has put together an impressive group of experienced experts who have graciously agreed to take time out of their busy schedules to talk to our audience about key issues that impact rare/orphan disease research and drug development. Topics they plan to tackle include addressing recruitment and enrollment, ins and outs of the patient experience, and how sites and sponsors can best address their needs.

Dominique Pichard​ is the chief science officer of the International Rett Syndrome Foundation and the mother of a child living with the disease. She helps drive the organization in its mission to supporting families dealing with Rett syndrome, building awareness, and pursuing potential treatments.

Sanjeev Luther​ serves as president and CEO of Rafael Pharmaceuticals, a drug firm focused on developing treatments for rare cancers. Bringing more than 30 years of experience to the firm, he also has held positions with Bristol-Myers Squibb, Novartis, Bausch and Lomb, GE Healthcare, and Mallinckrodt.

Teresa Rubio​ is a health scientist administrator with the US Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD), which provides a range of resources for professionals in the field. Her past positions include senior safety evaluator at the CDER Office of Surveillance and Epidemiology’s Division of Pharmacovigilance.

Contributing to the question and answer session after the trio of presentations is Lewis Fermaglich​, acting senior clinical advisor at the OOPD. He brings to the table a range of experience with orphan drugs and rare pediatric disease designations.

Rare/orphan diseases, special patient population goes live on July 7. Register now​ and catch invaluable information from leading experts during this exclusive, free online event.

Related news

Show more

Related products

show more

Saama accelerates data review processes

Saama accelerates data review processes

Content provided by Saama | 25-Mar-2024 | Infographic

In this new infographic, learn how Saama accelerates data review processes. Only Saama has AI/ML models trained for life sciences on over 300 million data...

More Data, More Insights, More Progress

More Data, More Insights, More Progress

Content provided by Saama | 04-Mar-2024 | Case Study

The sponsor’s clinical development team needed a flexible solution to quickly visualize patient and site data in a single location

Using Define-XML to build more efficient studies

Using Define-XML to build more efficient studies

Content provided by Formedix | 14-Nov-2023 | White Paper

It is commonly thought that Define-XML is simply a dataset descriptor: a way to document what datasets look like, including the names and labels of datasets...

Related suppliers

Follow us


View more