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).


2012: Banner Year for New Drugs

Fueled by new cancer therapeutics, last year the annual new molecular and biological entity approval count from the U.S. Food and Drug Administration (FDA) saw its highest year since 1997. One-third of the novel products approved by the FDA’s Center for Drug Evaluation and Research (CDER) are used to treat cancers of the blood, breast, colon, prostate, skin and thyroid.

FDA approved

NIH Launches Collaborative Program, Matches Researchers with Compounds to Explore New Treatments

The National Institutes of Health recently unveiled a collaborative program that will match researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients. NIH’s new National Center for Advancing Translational Sciences (NCATS) has partnered initially with Pfizer, AstraZeneca, and Eli Lilly and Company which have agreed to make dozens of their compounds available for this initiative’s pilot phase.

NIH collaborative program

SODIS Method Makes Water Safe to Drink

Each year, nearly one billion people around the world lack access to safe, clean water [1]. Water is essential for life, yet less than 1% of water on the planet is safe to drink. This is especially a problem in developing countries or during natural disasters. Take Hurricane Katrina: back in 2005 when it hit the Gulf Coast, one of the biggest needs for storm victims was access to clean drinking water.

In the United States and Europe, people take it for granted that when they turn on the faucet, clean water will flow out. Indeed, a single flush of a toilet in the West uses more water than most Africans have to perform an entire day’s washing, cleaning, cooking and drinking [2].

Securing access to safe water worldwide is vitally important. Clean water is essential for agriculture, food and energy production, recreation and reduction of poverty. More than 2 million people, most of them children, die every year from water-borne diseases. And time is of the essence: by 2020, more people could die of water-related diseases than those that have died due to HIV/AIDS [2].

SODIS in Indonesia

Viral-based Human Disease and the Nobel Prize for Medicine

nobel medal in medicineThe 2008 Nobel Prize in Physiology or Medicine was announced earlier this week. The prize was awarded to three europeans for the discoveries of two viruses that cause severe human disease; the cancer-causing human papilloma virus (HPV) and the human immunodeficiency virus (HIV).

Two French scientists, Luc Montagnier, age 76, at the University of Paris in Paris, France and Francoise Barré-Sinoussi, age 61, at the Institut Pasteur in Garches, France will split half the prize for their discovery of the HIV virus. Barré-Sinoussi is the 8th woman to receive the Nobel award for Physiology or Medicine.

A German researcher, Harald zur Hausen, age 72, at the University of Dusseldorf, Germany, will receive the other half of the $1.3 million prize for establishing that most cervical cancers are caused by two types of human papilloma virus.