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这个讨论涉及到所谓“医疗创新”（medical innovation）问题，引用ISSCR干细胞研究标准建议里的相关内容（其实这从科学的角度是“不得已”的事情，因为这是可能发生的情况，提出一个标准建议，是某种规范这类行为的尝试）。这里的medical innovation与纯商业利益的行为完全不同，也与标准的临床试验模式有些区别。
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7. Stem Cell-Based Medical Innovation
" m- H5 O0 |) S" c7 k0 s" eHistorically, many medical innovations have been introduced into clinical practice without a formal clinical trials process. Some innovations have resulted in significant and long-lasting improvements in clinical care, while others have been ineffective or harmful. In contrast to the commercial purveyance of unproven stem cell interventions noted in Section 2, the ISSCR acknowledges that in some very limited cases, clinicians may be justified in attempting medically innovative stem-cell based interventions in a small number of their seriously ill patients.
% x) Z& c c0 ^. zIn the case of medical innovations using stem cells and their direct derivatives, unique considerations justify a heightened level of caution. The diseases which potentially could be targeted by stem cell-based interventions are some of the most intractable diseases confronting clinicians – and interest in stem cell research has resulted in the organization of patient communities with high hopes for the prospect of future stem cell treatments. Due to their relative novelty in science, stem cells and their direct derivatives could behave more unpredictably when delivered to patients than either drugs used off-label or modified surgical techniques. Some attempts at medical innovation using stem cells and their direct derivatives may inadvertently violate physicians’ ethical obligation to “do no harm,” by producing more injury than benefit.
2 H( @) }6 g# n; C' r: \Innovative medical care and clinical research aim at different goals. The mere fact that a procedure is medically innovative does not qualify it as clinical research. Clinical research aims to produce generalizable knowledge about new cellular or drug treatments, or new approaches to surgery. Notably, the individual patient’s benefit is not the focus of clinical research, nor is the individual patient’s benefit the primary focus of the human subjects research committees overseeing clinical research. In contrast, medical innovations do not aim to produce generalizable knowledge but are aimed primarily at providing new forms of clinical care that have a reasonable chance of success for individual patients with few or no acceptable medical alternatives. Unlike clinical research, then, the main goal of innovative care is to improve an individual patient’s condition.
/ v6 p* @/ G1 Z5 Z# RAlthough attempting medically innovative care is not research per se, it should still be subject to scientific and ethical review and proper patient protections. This is especially true for stem cell-based medical innovation.
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* n- z6 |' P' N1 a, Q7 tGiven the many uncertainties surrounding the infusion of cells in ectopic locations and the significant challenges to the processing and manufacture of cellular products, only in exceptional circumstances does the ISSCR believe it would be acceptable to attempt medical innovations involving stem cells and their direct derivatives. The ISSCR anticipates that the following recommendation will be applicable primarily for seriously ill patients who lack good medical alternatives.
8 H' z- ]9 h8 R2 O/ s8 LRecommendation 34: Clinician-scientists may provide unproven stem cell-based interventions to at most a very small number of patients outside the context of a formal clinical trial, provided that:
|4 U: U$ e* d(a) there is a written plan for the procedure that includes:
% Y3 J5 \ [7 \" E7 H5 Hi. scientific rationale and justification
$ f; D0 ~/ }* X2 x; u+ ]explaining why the procedure has a
3 P* K& | g% ` d' Sreasonable chance of success, including# Y4 n, M* b9 g! \& U( C: f
any preclinical evidence of proof-of-
& Q+ t: R9 }2 d8 u( V2 h1 w$ Rprinciple for efficacy and safety;+ A+ o2 a$ Q& D, s R
ii. explanation of why the proposed stem
# f0 g5 @" b) s9 @' Ycell-based intervention should be P9 E4 d, Z S* A9 q$ c
attempted compared to existing* L, I! q, R' F& F; z( p/ \* o6 \% D
treatments;
& G% \9 q# i ~1 miii. full characterization of the types of cells being transplanted and their$ L5 {2 l( |/ K$ ?, C5 |# t2 ]& K- ?
characteristics as discussed in Section 4, Cell Processing and Manufacture;
) }: j" x9 u1 ?$ Xiv. description of how the cells will be$ X: A6 P C ]5 u- O; s0 c
administered, including adjuvant drugs, agents, and surgical procedures; and
+ ]! L9 }/ Y! E# q& K4 K5 h) Pv. plan for clinical follow-up and data
2 r& p3 B3 @# ]! t7 u3 A# h# T) @- Pcollection to assess the effectiveness and adverse effects of the cell therapy;6 S# d% x1 |$ L2 b9 z* ?( V3 i
(b) the written plan is approved through a peer review process by appropriate experts who have no vested interest in the proposed procedure;) }0 n* _4 s* Y# S
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(c) the clinical and administrative leadership supports the decision to attempt the medical innovation and the institution is held accountable for the innovative procedure;. T- q: c5 ~6 H
(d) all personnel have appropriate qualifications and the institution where the procedure will be carried out has appropriate facilities and processes of peer review and clinical quality control monitoring;
) E7 I6 V' o4 E# u$ T(e) voluntary informed consent is provided by patients who appreciate that the intervention is unproven and who demonstrate their understanding of the risks and benefits of the procedure;
( ]7 c) M& c7 {5 ?% @6 X% J" X(f) there is an action plan for adverse events that includes timely and adequate medical care and if necessary psychological support services;
/ x! {- S- W: J% I(g) insurance coverage or other appropriate financial or medical resources are available to patients to cover any complications arising from the procedure; and
0 {6 a+ U. f1 u5 Q0 n/ G(h) there is a commitment by clinician-scientists to use their experience with individual patients to contribute to generalizable knowledge. This includes:
8 m. \9 a! {; Y& b- li. ascertaining outcomes in a systematic: a% W9 x* @( ~+ ~+ r' a! S
and objective manner;( w) Y3 y5 Q1 b) x' |
ii. a plan for communicating outcomes,( |- l1 U5 t* }& `7 N& G6 `1 M- Y
including negative outcomes and. I+ A9 _( P- I" r7 ]& g
adverse events, to the scientific/ Q* Z- G8 I) i* k
community to enable critical review
6 g, z/ t$ d. z7 [. I(for example, as abstracts to
! r" e! j ]% @, X- Iprofessional meetings or publications in peer-reviewed journals); and
, @/ a- j5 P( X9 niii. moving to a formal clinical trial in a" S% P0 E/ `. E. l# R/ q
timely manner after experience with at# w# J0 \$ B0 B) r7 W
most a few patients.) g7 P% x: S/ e( {4 Y8 C% W
Not following such standards may exploit desperate patients, undermine public trust in stem cell research, and unnecessarily delay better designed clinical trials. Many who provide stem cell-based therapies may claim that they offer innovative medical care not available in
+ ^" f, R8 e9 N# k- G/ W! Cother medical institutions because of the conservative nature of medical care. Strict application of the above criteria to many clinical interventions offered outside of a formal clinical trial will identify significant shortcomings that should call into question the legitimacy of the purported attempts at medical innovation. |
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发表于 2011-1-19 21:29
