[Ip-health] Bayh Dole Rights, Size of Clinical Trials, 2004 Approvals

[Mike Palmedo]

Dear Dr. DiMasi.

I have looked at the number of patients in trials that the FDA cites
when approving New Molecular Entities. My earlier study look at FDA NMEs
approvals from 2000-2002. In the first study, I look at all FDA NME
approvals. In the second, I looked at all NME approvals, except for
antibiotics (because I was looking at orange book patents).

It is my recollection that your study excluded products developed by
smaller companies. Do you think your exclusions were important, when
estimating the average number of patients in clinical trials?

Mike

Joseph DiMasi wrote:
> Mike,
>
> I have been down this road on clinical trial sizes before with Jamie.
> First of all, the results from the study refer to a particular period
> in the past, not 2004. Second, and more important, the numbers that
> you pull out of labels or reviewer summary documents posted on the web
> need not come anywhere close to the number of subjects actually
> studied prior to a U.S. approval. The FDA labels and documents will
> often just note what the reviewers considered to be trials that were
> pivotal to the indication that was approved (i.e., often a few
> relevant phase III trials). You cannot assume that what you pick out
> of these sources represents the entirety of clinical testing. Third,
> we noted in our study data gathered independently by PAREXEL and
> reported for various years in their compendium of pharmaceutical
> industry R&D statistics that had a mean of 5,621 for a period relevant
> to our study.
>
> Joe DiMasi
>
>
>
> Mike Palmedo wrote:
>
>> http://www.cptech.org/blogs/drugdevelopment/2006/07/bayh-dole-rights-siz=
e-of-clinical.html
>>
>>
>> Bayh Dole Rights, Size of Clinical Trials, 2004 Approvals
>>
>> July 10, 2006
>> Mike Palmedo
>>
>> CPTech has looked at the patents for New Molecular Entities (NMEs) that
>> came to the market in 2004.
>>
>> Excluding antibiotics, which do not have Bayh-Dole listings in their
>> patents, the FDA approved 19 NMEs in 2004. Nine of these received
>> priority status for approval, meaning they were found to have
>> significant therapeutic gain over existing medicines. The remaining 10
>> NMEs were given standard approvals.
>>
>> For the products for which data was available, I looked up the number of
>> patients cited by the FDA in approving the medicines. (I had to exclude
>> three of the priority NMEs approved in 2004 for which the label did not
>> include the number of patients) The average (mean) number of patients in
>> the clinical trials on which the FDA approvals were based was 1073 for
>> priority drugs and 1840 for standard drugs. The median numbers of
>> patients in these clinical trials were 1290 for the priority drugs and
>> 2058 for the standard drugs. These figures are considerably lower than
>> the average size of clinical trials used by DiMasi in his often-cited
>> research on the cost of drug development =96 5,303 patients.
>>
>> The Orange Book lists 45 patents on the 19 NMEs. Three of these patents
>> include clauses citing government funding and subsequent Bayh-Dole
>> rights to use or license the patent. These three patents cover two of
>> the nine drugs which received priority approval =96 two patents for Clol=
ar
>> (a leukemia drug sold by Genzyme) and one for Lyrica (a diabetes drug
>> sold by Pfizer).
>>
>> A spreadsheet with the drugs, patents, and size of trials is online
>> here:
>> http://www.cptech.org/ip/health/rnd/2004nmes-07102006.xls
>>
>> [Posted by Mike Palmedo to Drug Development (with access) at 7/10/2006
>> 03:59:00 PM]
>>
>>
>> _______________________________________________
>> Ip-health mailing list
>> Ip-health@lists.essential.org
>> http://lists.essential.org/mailman/listinfo/ip-health
>>
>