There was quite a ruckus in the cancer drug development community when Memorial Sloan Kettering Cancer Center (MSKCC) suspended enrollment in three trials of therapies based on chimeric antigen receptor (CAR) technology. The news came from Twitter, where Jonah Lomu shared the following:
Aside from the science/clinical aspect, I thought the “news” and subsequent unfolding of the story were an interesting in relationship to regulations and implied data privacy.
FDA: CFR Title 21, Part 312
These trials are being run at MSKCC but in this case MSKCC is also the Sponsor of these trials, meaning they hold the Investigational New Drug (IND) application and have formal responsibilities that include “ensuring that FDA and all participating investigators are promptly informed of significant new adverse effects or risks with respect to the drug.” The requirements for safety reporting are spelled out according to seriousness and/or expectedness and again refer to FDA and other investigators.
At our company, we have sponsored a first in human clinical trial for a cancer drug that works by a novel mechanism. The people who enroll have advanced stage cancer and generally are experiencing various other health issues in addition. When adverse events happen, their cause is not always clear and at times a great deal of investigation and planning are needed to move forward. Our objective (and that of the regulations) is to ensure that the healthcare professionals working with patients have any and all available data to protect the safety of current and future patients.
ClinicalTrials.gov: Section 801 of FDAAA
The “news” came from a change made on clincialtrials.gov. The requirements to register a clinical trial on clinicaltrials.gov are spelled out in Section 801 of the Food and Drug Administration Amendments Act. Based on the registration instructions, records on clinicaltrials.gov are to be updated as needed or at least every six months. For the suspended CAR trials, MSKCC is both the Sponsor for FDA as well as the Responsible Party for clinicaltrials.gov. Their internal policies/procedures (based on the regulations) would determine how and when they update the clinicaltrials.gov registration.
Although MSKCC is the trial sponsor, the CAR technology in these trials is associated with Juno Therapeutics, a private company formed in late 2013 to commercialize immunotherapies from technologies out of Fred Hutchinson Cancer Research Center, Seattle Children’s Research Institute, and MSKCC.
As a small company, many people have suggested to us that we collaborate with an academic institution/investigator as Sponsor of our drug. In due diligence on these opportunities, I have heard from small companies that these relationships can be challenging as the goals of the institution/investigator do not necessarily overlap with company goals for the drug. Some common challenges I’ve heard include difference senses of urgency, sharing of data and ability to contribute to design of the trial. This MSKCC situation suggests another factor to explore in these relationships is the procedures for public disclosure, beyond the typical publication of study results.
SEC: Public vs. Private Company
Public companies are subject to specific SEC disclosure rules and biotech investors mine those disclosures carefully for information about development programs. One of the benefits of being a private in the early stages of clinical development of a new cancer therapy is the privacy. When I said that to a colleague who invests in public biotech, his response was clear and direct: that’s how companies hide data. As I mentioned above, adverse events in early stage oncology clinical trials can require significant effort to understand. Reflecting on my experience, learning about the data as the trial progresses (remember: no blinding) has been a series of incredible highs and very dark lows. Fortunately, none of our lows have ended up as deep as they appeared at first glance. I personally would not relish having to provide public guidance on events I don’t yet have data for, much less understand.
So if Juno is private, why all the excitement from the biotech investment community that generally focuses on the public market? The company made quite a splash by raising $120M in VC and going up against Novartis, which is working with CAR technology from University of Pennsylvania. Being in a hot area of development can lead to significant support as well as increased scrutiny at bumps along the road.
While there aren’t specific requirements for private companies to disclose data from ongoing clinical trials, there are risks related to collaborators and heightened scrutiny that may place a company in the spotlight unexpectedly.