Except that they have released preliminary data in the past. Dr. Buzdar, specifically, has done so. Therefore, they are either hypocritical, or reacting without thinking in defense of one of their staff (which, admittedly is more attractive from an organization than throwing him to the wolves, but I prefer my reactions with thought).
Companies routinely release such information. It may not be accepted practice, but it is common practice.
We just got new (and extremely promising) data 5 days before. What else were you looking for?
The news was shared in the week leading up to the conference. Noticeable tumor regression early in treatment. What else are you looking for?
And yes, if you're going to keep ignoring our response, then we are going to suggest you e-mail the company. In the specific post you're referring to (since when is one answer a battle cry?), I'd answered most or all of his questions a month ago, and there's not much else I can do if he's going to ignore that fact.
P-value goes the other way. P-value is the chance that the result would occur as a matter of random chance. A P-value of 5%(0.05) is a chance of 1 time in 20, and a P-Value of 2% (0.02) is one chance in 50. You decrease that chance by enrolling more patients. Given that the study is already powered to P=0.02, and 0.05 is the industry standard, they are not enrolling to improve the P-value.
They might (emphasis on might) be considering increasing enrollment to decrease the required PF S improvement. This would be because the probability of a patient randomly not progressing for five months is higher than a patient randomly not progressing for six months. Therefore, to reach the same value, more patients would be required. However, they cannot know if this is needed (blinded trial, remember), so it would be, at most, a precaution.
An alternate possibility is that there is some sub-group that is of interest (pseudo-progressers), and they want to increase enrollment in that subgroup in order to improve their chances of getting adequate certainty on that group out of this study. That need could come from analysis of data outside the study (direct, or the Ovarian Cancer study), so it's a real possibility that they would have it. This would generally be a positive reason to add, since it would offer more bang for the study buck.
Another possibility is that there is some uncertainty over whether the original (phase 2) enrollment of 33 individuals is going to be admissible to the FDA for some reason, in which case they would want to add another 33 patients, again, most likely as a precaution.
One final possibility is that they are referring to the patients who will be treated under the expanded access protocol, and just not being real clear about it.
Regardless, at the same time Linda mentioned the possibility of this expansion, she still mentioned completing enrollment this year, and primary results in mid-2015. I don't think we'll see major slippage.
"In that case, I am forced to reiterate my statement that I believe you have committed a breach of trust by disclosing this to the media without disclosing it to the sponsor in question first. I especially feel this to be the case since the sponsor has made no secret of their intent to disclose data as soon as it became available, and had done so in one case already prior to the specific disclosure that triggered this news article.
In addition, while I understand the academic preference is for the data to be disclosed in a controlled manner, it is not atypical for corporations to release data as it becomes available. In fact Dr. Buzdar, himself, has apparently released data prior to the endpoint (or interim point) of an open label trial for paclitaxel (Clinicaltrails.gov identifier NTC00050167, Paclitaxel Seems Equivalent to FAC as Neoadjuvant Chemo). Many other examples of such releases can also be found. To say that it is not accepted practice in your field seems to be contrary to the facts.
Dear Sir or Madam:
As I am sure you are aware by this time, a blogger by the name of Adam Feurstein is reporting that a Dr. Aman Buzdar, one of your staff, made statements critical of one of your clinical trial sponsors, Northwest Biotherapeutics. More to the point, Mr. Feurstein is doing it in a way that makes it appear that these statements are the formal position of MD Anderson as a whole. If this is true, I would appreciate you releasing a statement to that effect, in which case you will have lost much of my respect as an impartial arbiter of such trials. Especially since apparently no attempt was made to express this position to the sponsor before going public with it. On the other hand, if this was a single doctor stating his own opinion, a statement to that effect would also be much appreciated. Upon releasing that statement, if I were in your position, I would start looking into a defamation action against Mr. Feurstein for what appears to be a deliberate misrepresentation, as his statement is rather definitely causing harm to your reputation, and thus your ability to continue practicing. Obviously, that is entirely at your discretion. Your prompt response is appreciated.
(real name withheld)
Let's keep it classy folks.
I think it's mostly a non-event. People had been assuming that the sites were open for some time. Today's release answers an open question (what's going on in Germany), but it doesn't really provide any new catalyst.
You did catch the bit where German Insurance is authorized to pay for the treatment, right? The German Hospital exemption is not the same as FDA Compassionate use.
What they are saying is that it's a royal pain to open a trial site in Germany and it pretty much has to be finished one at a time, which is why it has taken so long, but that the first one opened in late May, and they are planning to open more in the next couple of months.
As a rule, that sort of thing gets published before a random blogger can write about it, not after.
There is no formal rebuke, so Adam is writing headlines that do not match reality. As usual.
There is the issue that their own personnel have released exactly such information in the past, indicating that they are, at best, inconsistent on this subject.
All developmental biotech companies have tons of red flags, that's the nature of small start-up companies in general, and medical/biotech in particular. The management almost always lacks specific experience, since starting a company is the sort of thing you typically only do once or twice in a lifetime. There are always major regulatory headaches.
It's always a gamble. But in this case, every bit of actual evidence (as opposed to speculation) is positive, and it's probably a risk worth taking.
Except that Phase 1 trials are not typically blinded, nor do they need to be, and results like this are typical from them. If the company were cherry picking data to release, you might have a point, but they are not. They have given us complete data on the state of the trial with every announcement, and have never claimed the data is anything but preliminary information. There is no wrong-doing on the part of the company, and the simple fact that AF found one doctor who disagrees with NWBO's approach does not mean that they have done anything wrong or even inappropriate.
It does, however, say that they did something wrong or unusual, which they didn't. They did exactly what every other company in their situation has done.
Could you please comment on the fact that the sources of all of this fuss have done (or praised companies for doing) exactly what they have attacked NWBO for doing?
Considering he is simply regurgitating AFs arguments, and considering that there are at least two false statements of fact in his article (that NWBO is hiding data when the DMC has confirmed they aren't, and the Buzdar is receiving money from NWBO), I would say no.
Low white cell count and the 55 rapid progressers are two completely different issues. The low white cell count group is now a sub-group within the trial, and separating them out for analysis greatly increases the chance of the remainder of the trial succeeding.
The rapid progressors are individuals who were never included in the trial based on the criteria "Patients must not have progressive disease at completion of radiation therapy. Patients with suspected pseudoprogression will be enrolled and analyzed separately". These patients were treated under the expanded access program under clinical trial listing NCT02146066.