Blog | April 27, 2015

Clinical Trial Data Transparency: The Dangers Of "Putting It In Layman's Terms"

By Anna Rose Welch, Editor, Biosimilar Development
Follow Me On Twitter @AnnaRoseWelch

data transparency

It’s been an exciting and tumultuous few months for ALS patients and the small biotech Genervon. The lengthening saga among the company, the FDA, and the army of eager, treatment-seeking ALS patients culminated recently in an order from the FDA that Genervon publicly release all data from the Phase 2 trial of its ALS candidate GM604. In a statement released early this week, Genervon said it had submitted all required data to the FDA for review. 

This request speaks to the continued challenges presented by a lack of data transparency in the industry. In fact, the timing of the latest move in the FDA-Genervon saga seems almost too-perfectly aligned with a statement from the World Health Organization last week which urged the industry to boost transparency by releasing all results — including the negative ones — of any vaccine, drug, or medical device clinical trials.

But behind the back-and-forth among the drug company, the industry, the FDA, and the patients, it seems to me there’s also another issue at hand — and that’s how exactly a company should present itself and its data as bolstering transparency becomes a key pharma initiative.

Some background first: The FDA is currently seeking more information from Genervon’s 12-week, Phase 2 trial of GM604, which enrolled 12 patients. Over the past few months, the company has released numerous positive statements lauding the drug for providing “very robust and promising” Phase 2A trial data — including data showing a patient who received the treatment in a compassionate use trial saw improved swallowing capabilities. According to Genervon, this data suggests GM604 is a prime candidate for accelerated approval from the FDA.

According to The Wall Street Journal blog, however, many experts are calling the company’s data into question because of the short duration and small size of the trial. One of these experts is Steve Perrin, CEO of the ALS Therapy Development Institute, who recently wrote a blog challenging the company’s request for approval. In the blog, following analysis of the GM604 data in comparison to data compiled from a sample study population of ALS patients, Perrin concluded (in brief) that GM604 “has not been adequately tested in patients as of today.” I urge you to read the full blog post for specific details on Perrin’s method of comparison and his findings. 

Genervon was far from pleased with his blog, publishing its own response Monday critiquing his “deliberate effort to use wrong assumptions to perform his confusing analysis.” (This is actually a politely edited version from the release originally posted, which used the phrase “evil deceptions” to describe Perrin’s analysis. This original post has since been removed.)

However, taking a step back from the nitty-gritty technical data sprinkled throughout the company’s releases, I’ve found myself caught more on the company’s abilities to use Pathos to appeal to its audience’s emotions. In a statement posted on March 21, the company claimed that a New Drug Application (NDA) was still at least 3 years away, meaning “the majority of this generation of ALS patients would not survive to try GM604.” In the April 17 release announcing the FDA’s data request, the company supported its desire to bypass the “secure and conservative” Phase 3 trial approach in favor of the accelerated approval plus Phase 4 surveillance requirements — a move which takes “a lot more courage.” According to Genervon, “We do not think that people really understand the sacrifice Genervon is making to try and save the lives of 30,000 U.S. ALS patients alone.”

Statements like these (and the martyr -type tone that is present in certain phrases) no doubt served as a call to action for many of the 30,000 ALS patients currently faced with only one approved treatment option that extends life by just a few months. According to The WSJ blog, there was a huge push from patients urging the FDA to accelerate approval and access to GM604, including an online petition with 535,000 signatures and a peaceful rally on Capitol Hill pushing Congress to influence the FDA to act.

A recent article speculated about the driving force behind such a huge turnout in support of the drug’s accelerated approval. I’d argue, in agreement with FierceBiotech’s John Carroll, that an enthusiastic presentation had a lot to do with it. Carroll argues that enthusiasm and optimism about a new drug is only natural during preclinical and clinical development, but that Genervon has “pushed far past routine optimism into the realm of using questionable statements to promote a social media campaign.”  

A large portion of the struggle with data transparency in this age of patient centricity centers around the way a company presents its data. What good is transparency if those who want to know how a drug candidate in development could help them are unable to grasp its benefits from a release jam-packed with highly technical, scientific data?

This is certainly a concern it seems that Genervon followers and the company hoped to address. At the beginning of its April 13 press release, the company states, “While many ALS patients wrote to Genervon apologizing for not having the energy to read or the ability to understand the science of GM604, Genervon should be the one who apologizes for not expressing the innovative science in layman [sic] terms.”

If this was the company’s goal — to present its progress in layman’s terms — perhaps it has succeeded, considering the tone present in some of its releases. We are, I’d argue, a nation that thrives on enthusiasm, hope, and sensationalism. As social media becomes a central form of interaction among patient groups and companies and their customers, we’ve become obsessed with engaging others, whether it be by garnering more likes, more comments, or more praise for the accomplishments and insights we choose to announce.

This latest incident with Genervon serves as a good example of what the industry needs to keep in mind as it strives to keep its technical data and its optimism about candidates in layman’s terms: there is a thin line between a healthy sense of optimism and sensationalism. While this incident is surely causing frustration amongst all involved parties, I think it highlights a hurdle this industry needs to face and address in the push to become more transparent.