Innovative
Therapy and Diagnosis
Background/Rationale:
Delivering care in an environment
that challenges the limits of knowledge, technology and
skill means the need to innovate is ever present. Often
innovative thinking fosters the development of promising
new approaches to therapy and diagnosis. Innovation in
medicine is valued and should be encouraged within our
academic environment, understanding that innovation entails
risk. A clinical situation that requires an innovative
solution should be carefully evaluated to examine the
risks and benefits to the patient. Outside assistance
and evaluation from the IRB may be helpful (though not
required by regulation) since the IRB has expertise and
experience weighing the risks and benefits of novel treatment
protocols.
The
near and longer-term consequences of the innovative approach
should be considered, particularly when special follow-up
may be indicated. The patient should be fully aware that
an innovative approach is being proposed, so that consent
can be given. In some situations, it may not be possible
to obtain consent from either a patient or a responsible
family member, particularly in cases requiring emergency
intervention. Nevertheless, a reasonable effort must be
made. There may be situations where physicians elect not
to pursue approval of innovative approaches to the practice
of medicine through the PHRC. This mechanism is offered
as a service to the clinical and research community for
applications at the interface of medicine and research
and is not intended to review the practice of medicine
by a licensed physician.
Distinction
between clinical care and research: Many innovative
therapies and diagnostic techniques are developed at the
interface between well-established clinical practice and
research. These activities do not, however, become research
until they are carried out in a systematic fashion to
develop or contribute to generalizable knowledge. The
category of "innovative therapy" is proposed
as an intermediary step between clinical care and formal
research. This review mechanism is intended to cover a
very limited number of patients in whom unusual, innovative
approaches are used for the primary goal of clinical diagnosis
or therapy. The mechanism is intended to provide limited
peer review for innovative approaches to unusual clinical
situations, especially when the dissemination of knowledge
of the treatment through case reports, presentations or
other means may contribute significantly to medical knowledge.
This mechanism is not intended
to replace formal human studies research protocols involving
small numbers of patients. Also, these guidelines do not
apply to
- the
planned or emergency use of an investigational drug
or device;
- the
common, accepted "off label" uses of FDA-approved
medications; or
- "pilot"
studies of approaches where additional larger studies
are needed and/or planned.
Examples
of innovative therapy appropriate for this mechanism:
-
Cryotherapy probe (FDA-approved) for osseous tumor ablation
used elsewhere in the body in a patient unable to tolerate
any other surgical approaches or standard ablative therapies
like radiation.
- Modification
of standard surgical or diagnostic techniques for unusual
or unique situations (e.g., new application of intrauterine
therapeutic device to a new, remediable fetal anomaly
which might allow pregnancy to proceed).
- Diagnostic
use of a research laboratory test for a very rare familial
disorder when no commercial or other research laboratory
routinely performing the test clinically is available.
Recognizing
that the distinction between innovation and research is
clearer on paper than in practice, the following guidelines
and procedures for review and approval of innovative therapies
have been adopted by the Human Research Committee.
Submission requirements (forward to the HRC offices at
relevant institution):
-
Letter from treating physician co-signed by division
chair or other knowledgeable physician peer. The letter
submitted should include the following:
- Description
of the clinical situation and the planned innovative
diagnostic or therapeutic approach to treating or
diagnosing the disease or condition
- Why
other standard therapies are not appropriate
- Rationale
for determining that the approach is "innovative
therapy or diagnosis" rather than a "pilot
study" or widely accepted "off-label"
use of a drug or device
- Supporting
documentation (attachments), if relevant or helpful,
e.g., case report from another institution, abstract,
or other data supporting your proposal
- Consent
form requirements: A formal MGH/BWH research consent
form should not be submitted as these submissions are
describing clinical diagnosis or treatment and not research.
You may use a standard clinical procedure consent form
or a customized PHRC consent form that includes the
standard sections of a research consent form (purpose,
procedures, contacts, costs, risks, benefits and alternatives)
without any reference to "research." The PHRC
administrative staff are available to assist in generating
customized PHRC consent forms for innovative therapy
or innovative diagnosis.
- Adverse
event reporting: Adverse events related to innovative
therapeutic or diagnostic approaches must be reported
in accordance with the PHRC Adverse Event Reporting Policy. Serious
adverse events must be reported (within 24 hrs of event)
by telephone, fax or e-mail followed by a full written
report submitted within the Humans module of Insight within 10 working days/14 calendar
days.
-
Follow-up reporting on the outcome: A report on
the outcome of the innovative therapeutic or diagnostic
approach must be submitted to the HRC offices within
one year of PHRC approval. The follow-up report
may be submitted as soon as the patient returns to routine
clinical follow-up. Once the follow-up report is reviewed
and accepted by the PHRC, the file will be closed.
PHRC
processes: Submissions will be reviewed and approval
given administratively by one of the chairpersons of the
Human Research Committees. Approval will be given initially
for one patient; thereafter the HRC will consider written
requests to treat up to two additional patients using
the same innovative approach. Situations may rarely arise
in which it is not possible to obtain prospective approval;
approval may occasionally be granted retrospectively,
if appropriate. After treatment of a third patient has
been completed, a final report on the experience with
the innovative approach and further plans should be submitted
to the Human Research Committee. Since the primary goal
is clinical diagnosis or therapy, up to 10 total patients
may then be allowed at the discretion of the PHRC.
If the investigator wishes to evaluate the innovative
approach systematically to develop or contribute to generalizable
knowledge, a formal research protocol must be submitted
for PHRC review.
If
the physician wishes to continue to treat patients using
this innovative therapeutic or diagnostic approach, he/she
must file a request through the Chief Medical Officer,
the Innovative Diagnostics and Therapeutics Committee
(MGH) or another relevant departmental practice oversight
committee to have the innovative approach adopted as a
standard or acceptable therapy.
Use
of Data: Data obtained as a result of these approved
uses may be considered approved by the PHRC and used in
grants, publications, or future PHRC protocol submissions
with the following notation: "This procedure/test/diagnostic
evaluation/treatment was reviewed by the Human Research
Committee (IRB) at MGH/BWH, and in accordance with institutional
policy approved as an acceptable innovative approach to
clinical diagnosis or therapy." Investigators
are reminded that they must comply with the Partners Confidentiality
Policies and Guidelines when using patient data in these
settings.
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