What some stories missed about the muscular dystrophy drug approval: Pharma’s close ties to vocal patient advocacy groups

child receiving IV infusionMany news stories about the Food and Drug Administration’s (FDA) Sept. 19 approval of the first drug to treat Duchenne’s muscular dystrophy rightly noted that the controversial decision signals a dangerous erosion of FDA standards. I wrote about the issue back in June in a post describing concerns with the data Sarepta Therapeutics submitted to support approval of their drug, eteplirsen.

Last week’s stories also did a nice job of describing the emotionally charged circumstances of the approval: FDA meetings attended by the boys in wheelchairs asking for hope against a fatal disease, and the political lobbying efforts of parents and advocacy groups demanding access to the drug.

Here’s a sampling of the coverage:

But only one article that I could find alluded to the close ties between advocacy organizations such as CureDuchenne and drug companies that are looking to gain marketing approval for new medicines. Ed Silverman of STAT noted that CureDuchenne raises money to invest in drug companies and has donated money to Sarepta.

[Editor’s note: This article originally stated that CureDuchenne invested money in Sarepta — a statement which we’ve clarified. The money was provided to the company as a grant, not an investment. CureDuchenne does invest in some small companies.]

Blurring the line between patient advocacy and drug marketing

This is a strategy used by organizations supporting work for diseases that affect relatively few patients, and it’s not a bad thing. In the past, companies were reluctant to invest in research for rare diseases given the limited number of patients and correspondingly low revenue potential. Now there are many companies pursuing research for these conditions.


FDA approval of eteplirsen has been good for Sarepta’s stock price. Will it prove as beneficial to patients? Source: Marketwatch.com.

It should be noted that the funding stream flows both ways, with many drug companies providing behind-the-scenes financial support for advocacy groups’ lobbying efforts.

The development of these close ties raises a thorny question: Whose interests are being served when an advocacy organization lobbies vigorously on behalf of a drug that could earn millions in profits for a drug company? Tellingly, news of eteplirsen’s approval was often reported in the Business section of newspapers, rather than the Health section — and appropriately so, as the drug clearly is helping stock prices, with no evidence it helps or will help anyone’s health.

If a consumer organization were to lobby to have an expensive new safety device installed into cars, wouldn’t we want to know if the manufacturer of the device (or the maker of the cars it was being installed in) was giving money to the consumer organization or its leaders?  Especially if there was no evidence that the device really contributed to safety and was going to raise the price of cars?

Relationships that are shrouded in secrecy

Following the money in any news story is important, but the relationship between advocacy organizations and their pharmaceutical backers often receives less attention than it should.

In a recent blog post from the Hastings Center, Drs. Sharon Batt and Adriane Fugh-Berman note that very few disease advocacy organizations refuse to accept industry funding. It is difficult if not impossible to find out if and how much these groups are taking, as they aren’t required to disclose this.

Others, such as Dr. Barbara Mintzes, who also has studied these relationships extensively, notes that the groups can be “disguised promotion channeled through a seemingly neutral third party.” She adds that these groups are described in the pharmaceutical marketing literature as “allies to help advance brand objectives.”

A victory that may be short-lived

Sadly, the so-called “victory” that these advocacy groups are touting for Duchenne’s–based on an outcome that doesn’t prove the drug works–may well turn out to be a defeat, as researchers in other fields have found through hard experience.

Cancer researcher Dr. Tito Fojo and colleagues detailed how the approval of drugs that do little or nothing–approvals based on surrogate endpoints like those used to justify the eteplirsen approval — can actually discourage innovation. Once such a drug is approved, other companies jump on the band-wagon with “me-too drugs” that are similarly ineffective. These drugs that do little or nothing still cost upwards of $150,000 year for treatment and siphon resources from other research areas that might yield more effective treatments.

Experience with Alzheimer’s disease also gives us reason for pause. Over 30 years ago, researchers found that people with Alzheimer’s have low levels of the chemical acetylcholine in their brains. They developed drugs to prevent the breakdown of acetylcholine so it could stay around longer. Companies promoted these drugs with misleading ads about their benefits that helped generate billions in revenue.  Then, a couple of years ago, the American Geriatrics Society put the drugs on their “Choosing Wisely” list, stating that they shouldn’t be prescribed without more assessment of whether they are doing any good. The organization said that after all this time, it remains unclear whether the drugs produce clinically meaningful effects.

Similarly, billions have been poured into the development of drugs for Alzheimer’s that focus on reducing levels of the protein amyloid. Although these drugs do a good job of getting rid of the protein, they do not make patients better, and sometimes make them worse.

Will the same thing happen with eteplirsen? For the patients’ sakes, I’m hoping absolutely not. But the signs are strong that we may see a similar process unfold, and I hope journalists stay on the story to find out.

You might also like

Comments (3)

Please note, comments are no longer published through this website. All previously made comments are still archived and available for viewing through select posts.

Debra Miller

September 28, 2016 at 12:23 pm

I would like to make a correction to your article. CureDuchenne provided a grant to Sarepta in 2010 which was used to conduct toxicology studies that allowed the company to get off FDA clinical hold and proceed with human clinical trials. This was strictly a donation, CureDuchenne did not make an investment in the company at any time. And, CureDuchenne has never paid lobbyists or taken any funding from any company to provide lobbying services. CureDuchenne was not part of the group that provided free travel, accommodations and lost wages to the families that attended the Advisory Committee meetings. Neither CureDuchenne nor it’s employees received any compensation from Sarepta investors, or investors in any biotech company in the Duchenne space. I would appreciate your correction on these facts, and I would be happy to inform you of our business model at any time.

    Susan Molchan

    September 29, 2016 at 11:47 am

    Thanks Ms. Miller for your comments and I didn’t mean to imply that CureDuchenne specifically was involved in lobbying for the Sarepta approval. Thank you for clarifying that your group does not hire or pay lobbyists. Also Mr. Silverman made a clarification to his article to note that CureDuchenne had “donated” money to Sarepta, rather than invested in the company as he’d originally stated. As noted, some advocacy organizations invest in small companies to good effect, to allow them to complete the trials needed (the Cystic Fibrosis Foundation is a good example), and CureDuchenne has invested in small companies at times.

Thomas Rogowsky

September 29, 2016 at 6:01 am

This article is also incorrect in its assertion that no benefits have been experienced by youngsters suffering from DMD and their families through the administration of this medicine.