NIH Announces New Program to Develop Therapeutics for Rare and Neglected Diseases
The National Institutes of Health is launching its first drug development pipeline to produce new treatments for rare and neglected diseases. With a new program specifically intended to stimulate research collaborations, NIH is hoping to take the risk out of making drugs that are often less profitable.
Datta: More than 25 million Americans suffer from rare diseases.
Guttmacher: And millions of people who have diseases for which there's no effective therapy today.
Datta: Dr. Alan Guttmacher is the acting director of the National Human Genome Research Institute.
Guttmacher: What we're trying to do is develop new effective therapies for both rare diseases and so-called neglected diseases.
Datta: Neglected diseases are often common in some parts of the world where people cannot afford or otherwise access treatments, if they exist at all. One example of a neglected disease is malaria, a tropical disease carried by mosquitoes that affects over 300-million people worldwide. A rare disease is a disease that affects fewer than 200-thousand Americans. Cystic fibrosis is one example, affecting the respiratory and digestive systems; another example is Huntington's disease, which affects the brain and nervous system.
Guttmacher: We're not going to be able to develop therapies for all 7,000 rare diseases in one fell swoop. But we're going to both tackle a number of those diseases and come up with processes that we hope others can apply to finding new effective drugs for rare diseases.
Datta: Dr. Guttmacher explains that a 24-million dollar program, called Therapeutics for Rare and Neglected Diseases, plans to bridge the gap between initial drug discovery and patient use.
Guttmacher: There exists a gap today between the early part of the scientific research needed to develop drugs to the clinical trials that are eventually done.
Datta: Typically, drug development begins when academic researchers studying the cause of a disease discover a new molecule or chemical that may have a beneficial effect. Too often, the process gets stuck there because few academic researchers can conduct all the types of studies needed to develop a new drug. If a drug company with resources to advance the research does get involved, substantial preclinical work begins.
Guttmacher: The steps called medicinal chemistry, some toxicology testing, those kinds of things, move the potential drug candidates farther along the pipeline so that they can then be used in clinical trials.
Datta: Only if preclinical work is successful can a potential treatment move to clinical trials in patients. Unfortunately, preclinical success rates are low, with 80 to 90 percent of projects failing. And the costs are high: it takes two to four years of work and roughly 10 million dollars to move a potential medicine though this process.
Guttmacher: It's referred to in the pharmaceutical industry as the Valley of Death, because it's where many promising new drugs go to die.
Datta: The Therapeutics for Rare and Neglected Diseases program will work closely with disease-specific experts on selected projects. Dr. Guttmacher adds the entire process itself is going to be examined closely.
Guttmacher: So we're going to focus on rare and neglected diseases and try to cross this Valley of Death to do the kinds of steps that are necessary. And to improve the way those steps are done compared to how they've been done traditionally. To make them shorter steps, to make them less expensive steps so we can really develop new therapies for these rare and neglected diseases.
Datta: Dr. Guttmacher says it will be a few years before the first drugs emerge from this program.
Guttmacher: But we hope that once they do start emerging, it'll be a steady stream of drugs emerging from it.
Datta: Of the thousands of rare and neglected diseases, only a few will be studied each year. The Therapeutics for Rare and Neglected Diseases program aims to collaborate with academic researchers, patient advocacy organizations, disease-oriented foundations, and private pharmaceutical companies—whatever is needed to get new drugs through clinical trials and to patients. For more information on the program, visit rarediseases.info.nih.gov/TRND. This is Indrani Datta, National Institutes of Health, Bethesda, Maryland.