Regulatory Issues

Regulatory bodies such as the Federal Drug Administration (FDA) already have a full plate. In the US, FDA-regulated products account for 20 percent of each dollar spent by American consumers each year. More specifically, the work of the regulatory authorities include the following:

• Carefully considering benefits and risks when evaluating medical products
• Staying on top of rapidly advancing scientific innovations
• Providing industry guidance to encourage the development of new therapies in promising areas
• Ensuring the appropriate usage of the most recent science and technology in appropriate ways
• Advising in clinical trial design, drug and device development and clinical practice
• Coordinating the dialog between key constituents such as scientists, government agencies, standards organizations and clinicians to evaluate new diagnostics and therapeutics

We anticipate more targeted therapies in the area of Precision Medicine. This means more drugs that need to be approved and more therapies that need to be evaluated. Does this mean that this will very quickly overwhelm organizations such as the FDA that are already stretched thin?

Well, not so fast!

Firstly, the primary order of business for regulatory bodies such as the FDA is to ensure that a medical product is safe and effective. It also makes sure that its intended use and benefits outweigh any associated risks. The traditional approach leaves a lot of room for improvement. According to Spear, Heath-Chiozzi, Huff (2001), here are the response rates per drugs classes for a general cross section of the population:

• Pain Management: 80%
• Depression: 62%
• Asthma: 60%
• Cardiac Arrythmias: 60%
• Diabetes: 57%
• Migraine: 52%
• Arthritis: 50%
• Osteoporosis: 58%
• Alzheimer’s: 30%
• Cancer: 25%

By better understanding why some patients respond to new or already approved drugs we most certainly will be able to tailor drug indication and dosage to certain populations. This will essentially allow the FDA to approve drugs for more specific indications. It will increase the FDA’s ability to advise practitioners. It will reduce unnecessary exposure of patients to ineffective treatments.

Secondly, the FDA estimates that every year about 2.2 million adverse drug reactions occur in the US. Some are so severe, that they lead to death. This number is estimated to be more than 100,000 fatalities per year. The current state of regulatory affairs is forced to accept those incidents as the “necessary evil” based on current best practices. However, a better understanding of the underlying genetic causes can reduce those numbers substantially.

Figure 1: Probability of success from drug development stage – see Arrowsmith (2012)

Thirdly, precision medicine has the potential to fundamentally streamline the current approach to drug development. As it stands, many drugs never reach the final stage of submission to the FDA requesting the approval to go to market. Very often drugs wash out, because they are not able to show efficacy and sufficient safety in a test group that mimics the a general cross section of our population. Again, understanding the underlying variability among potential patients would allow us to use drugs more targeted and allow for a higher number of drugs to be used in safe manner.

Literature
Spear, Heath-Chiozzi, Huff (2001), Clinical application of pharmacogenetics, Trends in Molecular Medicine, 7(5), 201-204

Arrowsmith, J. (2012). A decade of change. Nature Reviews Drug Discovery, 11, 17-18