Part II: A step-by-step guide to conducting an informal clinical study
As previously discussed (see: Part I: The case for informal clinical studies, September
2012), an informal clinical study can be a great way to develop the relationships and
credibility needed to de-risk a product and accelerate time to market. The process for
approving a study is fairly straightforward and can be divided into four key steps:
The first step is physician recruitment. To obtain maximum benefit, a company should
select a physician with a strong reputation. A quick way to develop a short-list of
physicians is to research the names of academic society heads and published authors.
Once this list has been prepared, it is helpful to speak to people in the industry to obtain
recommendations. Not all famous physicians are well liked. Ideally, the physician
selected for the study will be 1) well-liked with many followers in the physician
community and 2) have international experience and a mind-set that supports open
collaboration with a foreign partner.
Once a physician has been selected, the physician and manufacturer will need to obtain
a hospital director approval. To do this, the manufacturer must submit a formal
request to conduct the research. At this stage, the request does not need to contain a
great deal of detail. Typically, all that is needed is: an overview of the product, the
purpose of the research, the name of the sponsoring physician, information validating
the safety of the product, and a description of the product's track record overseas. At this
stage, the manufacturer will also need to describe the economics of the arrangement,
including any research stipends or costs that will be covered. The need for stipends will
depend upon the financial condition of the hospital and the importance of the research.
For a financially stable hospital, being the first to use a product can be enough of an
incentive to support the research.
Once an approval has been received from the hospital director, the doctor and
manufacturer must collaborate on protocol development. In many cases, the
manufacturer will simply want to duplicate a study that has already been completed
overseas. Although this may be reasonable in some cases, it may be necessary to make
adjustments to the protocol to meet local needs and ensure the feasibility of patient
recruitment.
Japanese institutions tend to have relatively low patient volumes vs. their foreign
counterparts. As a result, it may be necessary to broaden inclusion criteria or reduce
exclusion criteria to make a study feasible in Japan. It is not uncommon to lower BMI
requirements and / or increase the targeted age range. Japanese patients tend to be older
and have lower BMI's than their counterparts in the United States and Europe.
After the protocol has been developed, the manufacturer and sponsoring physician must
submit the research for an ethics committee review. As part of this review, the
manufacturer and physician must demonstrate that the study is safe and ethical. A
typical outline is:
a.
b.
c.
d.
Overview of treatment safety
Substantiation that the benefits of participation outweigh the potential risks
Description of the research methodology
Outline of the ethical guidelines for the study, including:
i.
appropriate patient selection criteria
ii.
adherence to informed consent
iii.
a draft patient application
e. Manufacturer's approval of ethical procedures
f. Manufacturer's approval of research methodology
After the above steps have been completed, the manufacturer and physician should
receive a final approval and the research can begin.
In most cases, a well-seasoned physician sponsor will be adept at navigating the
approval process. Assuming the physician is supportive and the study does not result in
financial hardship for the institution or create risk for the patient, the study should be
approved.
To maximize the chance of success, the manufacturer should consider offering support
where appropriate. In an informal study, it is not uncommon for the manufacturer to
support data collection, analysis, publication and dissemination. Foreign manufacturers
can be particularly helpful in creating English language versions of the research and
supporting publication and / or creating opportunities for the researcher to present the
results of the study at overseas conferences.
For most companies, it will take 3-4 months to obtain an approval for the research. The
amount of time required for the study will then vary depending on the ease of patient
recruitment, length of treatment, and the number of patients required. Although 3-4
months may seem lengthy, this is often shorter than the amount of time needed to
negotiate a partnership agreement or lay the groundwork for entering Japan. If
conducted in parallel, an informal study does not increase time to market and will
actually increase both the likelihood and magnitude of success.