A recent report released by PhRMA states that more drugs are currently in development for rare diseases than have been released in over 20 years since the Orphan Drug Act!

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading pharmaceutical research and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier, and more productive lives.  PhRMA companies are leading the way in the search for new cures.  And it appears that research efforts have started to shift, focusing on rare, orphan and neglected diseases.    “Once you’ve talked to a deseperate parent whose child is suffering or dying, you wil be forever convincd of the need for medical innovation, including the terrible diseases that afflict even just a few”, said PhRMA President and CEO John J. Castellani.   “Researchers at biopharmaceutical companies are working every day to bring hope to patients with limited options.”

The 460 medicines for rare diseases in the report are all in laterstages of the pipeline, meaning in clinical trials or under reveiw by the FDA.  A major area of research involves rare cancers.  Solid tumors of the liver and thyroid, cancer of the blood and melanoma, or skin cancer, account for more than one-third of all rare disease drugs under development.

Other major areas of research include:  genetic disorders, neurologic disorders, and infectious diseases.  For more detailed information and to download a copy of this report please visit www.phrma.org.

1 thought on “More Than 400 Medicines in Development to Treat or Prevent Rare Diseases”

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