21st Century Cures Act – Is It Truly Progress?

By Glenn Petrie, Ph.D.
Senior Scientific Advisor
ABC Laboratories
www.abclabs.com

Dr. Francis Collins, Director of NIH, has stated that it takes “around 14 years and $2 billion or more “to develop a new drug. Based on statements like these, Congress has come to the conclusion that the drug approval process is too onerous and lengthy and is the primary impediment to the discovery and approval of new drugs. In response they have passed the 21^st Century Cures Act with nearly unanimous, bipartisan approval. But is this a fallacious assumption? Some argue that the FDA review and approval process is the fastest and most streamlined in the world and that accelerating the approval process may impact patient safety.

In an editorial in the New England Journal of Medicine, Dr. Jerry Arvon states ”The bill would also encourage the FDA to rely more on biomarkers and other surrogate measures rather than actual clinical end points in assessing the efficacy of both drugs and devices.” While surrogates have previously been utilized as endpoints by the FDA to support accelerated approvals, this was only for drugs intended to treat life threatening illnesses.  Dr. Arvon goes on to state that biomarkers “may not always predict the drug’s capacity to improve patient outcomes “. The literature has many examples of cancer drugs approved utilizing these criteria that not only do not extend life, but have side effects.

While the argument regarding expedited drug approval continues, one aspect of the new law has unanimous support; an additional $8.75 billion in funding for NIH over the next 5 years.  According to a study published in Health Affairs, federal funding of basic research played a major role in the origin of transformative drugs approved between 1984 and 2009.