April 1, 2019What's New
There is a new world in drug development to treat diseases that affect small numbers of patients. The key now is how to make this process both humane and cost-effective. Perhaps, expanding the patent life of drugs is one solution, by taking into consideration how long it takes for a product to reach the market in the medical space compared with other industries.
The generic drug business is able to provide relatively low cost solutions to treat certain common infectious diseases, as well as to treat mild to moderate hypertension. However, there is a major unmet medical need to cost-effectively, and humanely, treat patients with those rare diseases that affect a small percentage of the population.
What we can now say is that the FDA has become an active partner to accelerate medicinal products to the market, where there is an unmet medical need for a serious and potential life threatening condition, and/or where there is no adequate or limited treatment options for a rare/orphan disease.
Target Health is expanding even more into the orphan/rare disease space following the approval of a drug in 2012 which treats a disease which occurs in approximately 1/20,000 live births. Current programs include a disease that affects 1/50,000 live births and another program in a rapidly progressing neurodegenerative disorder with a prevalence of about 5/100,000 Americans. In this latter disease, symptoms generally appear later in life, but can affect people as young as 40. An FDA meeting will occur in May for another disease that affects 5/100,000.
Target Health is very pleased that not only has FDA provided financial incentives to sponsors to develop drugs to treat rare pediatric diseases, but FDA has also issued clear Guidances which address novel approaches to develop treatments for all rare diseases. Equally important, we have observed at our Type B and Type C meetings with FDA, a proactive approach to smooth the path to NDA/BLA applications. In one case, FDA agreed to assess an approach to accelerated approval, and in another case, FDA, in writing, provided suggestions as to which measures would be best to assess endpoints that were not just meaningful to the treating clinician, but more importantly, meaningful to patients and caregivers. Target Health Inc. also just discovered data from a study performed around 15 years ago that could support, at a minimum, Breakthrough Designation in a rare pediatric disease population.
For more information about Target Health, contact Warren Pearlson (212-681-2100 ext. 165). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel. The Target Health software tools are designed to partner with both CROs and Sponsors. Also visit the Target Health Eating Website to see all of the fantastic recipes since 2012.
Joyce Hays, Founder and Editor in Chief of On Target
Jules Mitchel, Editor