A recent article in the New York Times once again missed the mark when it comes to reporting on the great promise in the biopharmaceutical pipeline. Just last year, PhRMA released a report analyzing the pipeline and found that more than 5,000 medicines are in development worldwide, 70 percent of which are potential first-in-class treatments and providing new ways to target or treat disease for patients with serious unmet medical needs. While this important point was overlooked in the article, we shouldn’t ignore the fact that hope has never been greater for patients battling disease.
Here are some other highlights in the pipeline report:
- There are 610 medicines in development for neurological diseases, including Alzheimer’s
- A large percentage of medicines in the pipeline focused on psychiatry are also potential first-in-class treatments, including potential medicines for depression
- 185 medicines are in the pipeline for HIV/AIDS
- Personalized medicines for rare diseases are a rapidly growing part of the pipeline
A separate study found that there are 45 medicines in development for antibiotics in the U.S. While it has been particularly challenging to develop new antibiotic treatments, for a variety of reasons, the Generating Antibiotic Incentives Now (GAIN) Act was signed into law in 2012 to help address this growing public health problem and encourage the development of medicines in this area. As Edward Cox, M.D., M.P.H., director of the Office of Antimicrobial Products in FDA's Center for Drug Evaluation and Research, pointed out, “Establishing new ways of developing safe and effective new antibacterial drugs is an enormous challenge and not an effort that can be accomplished alone.”
Already, a new antibacterial medication aimed at stopping the rapid spread of antibiotic-resistant “superbugs”, otherwise known as “MRSA”, has gained approval under GAIN. For more on medicines in development targeting infectious disease, particularly MRSA, click here.
The collaborative medical innovation ecosystem is critically important to help overcome some of the scientific hurdles associated with the research and development of complex diseases. An example of how collaboration is helping to address some of the greatest health care threats facing patients is the Accelerating Medicines Partnership (AMP), which is a public-private partnership between biopharmaceutical companies, the National Institutes of Health and non-profit and patient advocacygroups. This partnership represents an integrated approach to treatment discovery and seeks to increase the number of new diagnostics and therapies for patients while reducing the time and cost associated with their development. AMP is currently focusing its efforts on three to five-year pilot projects focused on three disease areas: Alzheimer’s, type 2 diabetes and autoimmune disorders, including rheumatoid arthritis and lupus.
As we have said repeatedly on the Catalyst, biopharmaceutical scientists are trying to break the code to Alzheimer’s – one of the most complex diseases with a particularly high drug development failure rate. A PhRMA report titled, “Alzheimer’s Research: Setbacks and Stepping Stones,” detailed the failure rate and found that between 1998 and 2011, 101 treatments have failed to reach patients. In the same time period, three medicines have been approved to treat symptoms of the disease. This 34 to one ratio of setbacks to successes underlines the difficulty of developing new medicines for Alzheimer’s. While this might be the case, the reality is that so-called failures in the development of new drugs for Alzheimer’s are helping redirect research – providing new information that allows science to move forward.
In our view, it is easy to talk about cost but what isn’t easy – in fact, it is pretty darn hard – is the lengthy and risky research and development process for medicines. It takes 10 to 15 years and more than $2.6 billion, on average, to develop just one medicine. And as mentioned previously, the failure rate is very high in the biopharmaceutical field. For all the thousands of compounds entering the pipeline, only one will ultimately receive approval from the Food and Drug Administration.
But perhaps most importantly, we must have a public dialogue about the value that innovative medicines provide to patients and their families and whether or not we as a society believe that investing in new treatments in cures is worth saving a child or parent’s life. The current debate not only overlooks this important point; it ignores the long-term value of these medicines to the U.S. health care system.