A Risk Worth Taking

As the chief government affairs officer of a national financial services trade association, I see Washington DC politics – warts and all – from the window of an office overlooking Pennsylvania Avenue, equidistant from the White House and the Capitol.

As the parent of a kid with a dread genetic disorder and leader of a non-profit foundation, I also have witnessed (and been engaged in) the politics of disease.  Alas, the DC “swamp,” too often, can look better in comparison.

May 2000:  My wife and I sit numbly in the room at Children’s National Medical Center, being told that our two-year-old son James has Duchenne Muscular Dystrophy – a uniformly fatal disease which throughout history has cruelly taken the lives of boys and young men.  Equally sobering, the investment of private biopharma companies in DMD at that time:   virtually zero.

Fast forward, 2018:  DMD is still a dread, awful disease, but billions in investments are in play.  Scores of drugs are in the clinical trial pipeline.  Life expectancy has expanded by a decade.   James is now a 21-year-old college kid and still having fun despite his disability.  In the last two years, the first two drugs specifically for this disease have been approved by the FDA.   One of them targets a universe of only 13 percent of DMD boys based on their genetic mutation (which does not include my son).   The other – deflazacort (marketed now as Emflaza) can help 100 percent of DMD patients.

We found a caring clinician who was willing to prescribe deflazacort, and we spent thousands of dollars (legally) securing it from a London provider.  My family is blessed to have had the means and an aggressive clinician.  Let’s face it, the overwhelming majority of DMD kids do not have that blessing.

So we rolled the dice, and our experience with deflazacort was excellent, however, it was anecdotal.  Along came Marathon Pharmaceuticals, a small biopharma firm that was likewise willing to roll the dice under the premise that deflazacort could be proven to exacting FDA standards.   Years of investing in gathering data and doing the necessary clinical trials paid off with the FDA approval in early 2017.

The result of that risky investment and that approval is that more than a thousand boys and young men are getting this drug who did not get it before.  Clinicians are now willing to write the prescription for the drug because it is proven and manufactured domestically under exacting standards.  It is not my opinion, but rather a fact, that thousands of boys and men are going to live longer, and with a better quality of life, as a result of that investment.

That really was the story, in my view. But for others, there was confusion and consternation about the announced list price, even though none of us parents would ever pay that. A few headlines took the story in a radically different direction.

Because a few people wanted to gin up a controversy and ignore the facts, what could have been celebrated was instead confused. I guess we shouldn’t care that thousands are now getting access to the drug or that Marathon (and now PTC Therapeutics, which bought the drug rights) established a generous patient assistance plan that ensures that no family endures hardship to buy the drug.  In fact, in our case, our insurer initially declined coverage, and we were provided the drug at zero cost.   The insurer eventually reversed itself, as virtually all insurers nationally have done.   And all of those insurers have negotiated their pricing arrangements to their satisfaction.

To be sure, the escalating price of specialty drugs is a significant national concern.   If Americans are ever to bend the health care cost curve, the cost-reward curve of drug development must be addressed.  (While we’re at it, how about we debate the costs of obesity, the costs of chronic illness, of end-of-life issues, of over-utilization of specialists, and self-referrals to specialists, and the costs of defensive medicine?  How did a company taking a risk to invest in a disease that afflicts only one in 4,000 boys get the bad rap when one considers the full landscape?).

The truth is – the headlines about price gouging were inaccurate.   So too were the comments and accusations of all too many in the DMD community.  In part, this was because families in the DMD community were not accustomed to having any drug covered by insurance, and there was an irrational fear factor about what insurers would do when deflazacort was approved.  Those fears were not realized.

Now that a couple of years have elapsed, we can see that one medicine went from being available only to a select few who could afford the complex process to get it from Europe, to now being indicated for all DMD patients and accessible by everyone who needs it.  It’s time for all of the stakeholders to thank Marathon for taking that risk for our sons.

As President of a non-profit organization (the Foundation to Eradicate Duchenne) that exists to encourage research and development for new DMD treatments, I have a disclosure to make: my foundation’s largest investment is in a drug (currently in Phase 2 clinical trials) that hopefully will put deflazacort out of business – a different and potentially better form of a corticosteroid. We fight for our kids. All of the DMD advocates and organizations should be similarly transparent.

Headlines are cheap.  Facts on the ground, just a couple years later, are another thing entirely.

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