Trade Group Study: Hundreds of Rare Disease Drugs in Development

According to a new report released last month by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical pipeline is innovative and robust, with a high percentage of potential first-in-class medicines (meaning a new treatment where nothing currently exists) targeting diseases with limited treatment options. In addition to identifying medicines in development for conditions and diseases such as septic shock, ovarian cancer, sickle cell disease, and Lou Gehrig’s disease (amyotrophic lateral sclerosis), which haven’t had any new product approvals in the last ten years, the report offers positive news for the rare disease community: one third of the products currently in clinical development have a rare disease designation by the U.S. Food and Drug Administration (FDA).

PhRMA

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Easter Seals Living With Disabilities Study

Most of us take everyday adult life for granted; we have a place to live, access to transportation and the opportunity to live independently. The same can most likely be said for those of us with adult children. The basics are covered. But what happens if you’re one of the millions of adults living with a developmental disability in this country? What if you’re the parent and caregiver to an adult child with a disability? How is your life different? Are the basics covered?

When people with disabilities turn 21, they and their families are no longer eligible for the services and supports provided by law through the school system. To determine how this affects them, Easter Seals — the nonprofit, community-based health agency dedicated to helping children and adults with disabilities attain greater independence — commissioned Harris Interactive to perform an online poll of adults with disabilities and their parents [1]. The primary goals of the study were to call attention to the challenges these people face, help service providers better respond to their needs, and heighten awareness of the needs of adults living with disabilities and their families.