A New Paradigm for Accelerating the Search for New Cures

The American Journal of Pharmacy Benefits, July/August 2011, Volume 3, Issue 4

A bill introduced in the US House of Representatives, the MODDERN Cures Solution, would ensure that science, not patent law, drives development of new therapies.

In the discovery phase for new drugs, researchers create thousands of potential compounds that might provide hope for millions of Americans with chronic diseases. Before these compounds evolve into commercial products, there is a necessary rigorous development and review process.

Let’s take a group of new compounds, say 10,000 of them. On average, only 5 compounds out of the group progress to the clinical trial phase, and only 1 out of those 5 compounds is eventually submitted to the Food and Drug Administration (FDA) for review.1

The process of turning 10,000 new compounds into 1 approved drug typically takes 15 years and is understandably expensive. One study estimated that the average total research and development cost for a new compound in the year 2005 was $1.3 billion. The average preclinical costs alone were more than $600 million per drug.2

Imagine the impact on patients if we could turn more of those 10,000 new compounds into breakthrough treatments for people with chronic conditions.

Major Toni Grimes enlisted in the US Army in 1991. Her military career was cut short not by any combat wounds but by lupus, a chronic disease that attacks the immune system. With no FDA-approved treatments to halt the progression of lupus, Toni was compelled to retire from military service after 19 years. She can’t help wondering what a different course her career would have taken if there were treatments that could have stopped lupus from nearly overcoming her body.

One pharmaceutical company told the National Health Council that 80% to 90% of new compounds not brought to market were abandoned because of a weak patent, not because of the science.

There are many reasons why compounds are not patentable. For example, if the discovery was publicly discussed at a medical conference, the company would not be able to file for a patent to protect its investment. Sometimes the patent application isdenied because the compound or how it treats a condition is not considered unique.

If a company does receive a patent on a discovery, it still may not advance the compound to the clinical setting. This could be because the drug does not address the original targeted condition or the patent clock will run out of time before the research phase is completed. This situation is common with drugs that address longterm degenerative conditions such as Alzheimer’s and amyotrophic lateral sclerosis (Lou Gehrig’s disease). In these circumstances, the compound is simply shelved and forgotten.

A bill being introduced in the US House of Representatives could dramatically change the development process for new treatments. Known as legislation to modernize our drug and diagnostics evaluation and regulatory network—or MODDERN Cures Solution for short—the bill would ensure that science, and not patent law, drives the development of new therapies.

MODDERN would establish a new category of drugs called “dormant therapies.” These are compounds that offer promise to treat conditions with unmet medical needs, as defined by the FDA, yet have insufficient patent protection.3 The inventor company would receive a period of data exclusivity upon approval in exchange for researching a dormant therapy. The bill also ensures a definitive timeline for generic access to the market.4

In addition, a pharmaceutical company that works with a diagnostic maker to develop tests to help determine which patients are most likely to benefit from the new compound would receive a brief extension on the data exclusivity period. This would help to advance personalized medicine by ensuring that the right treatments get to the right people, which is good for the individual patient as well as the overall healthcare system.

Jessica Hembrook of Delavan, Wisconsin, takes it in stride as she names the various medications her 2 sons have taken in an attempt to control their epileptic seizures. She wishes that there had been a better way to determine what therapy would have been most effective to treat her sons’ epilepsy at the time of their initial diagnoses, rather than having to go through a trial and-error process to determine the right treatment plan.

Imagine the impact on patients if we could better identify who would benefit from new discoveries.

The MODDERN Cures Solution is more detailed than what I have presented in this article.5 As with any bill in Congress, it is a proposal in motion and will change as the bill progresses through the House and Senate. The legislation is not designed to fix all of the problems in the development pipeline for new treatments. However, it will ensure that research dollars are spent in a more effective manner, that potential opportunities for new cures are not abandoned, and that new diagnostics bring targeted treatments to the right people living with a wide range of medical conditions.