Surgical innovation as sui generis surgical research

Successful innovative ‘leaps’ in surgical technique can contribute exponentially to surgical advancement and, thereby, to improved health outcomes for patients. Such leaps often occur relatively spontaneously, without substantial forethought, planning, or preparation. This feature of surgical innovation raises special challenges for ensuring sufficient evaluation and regulatory oversight of new interventions that have not been the subject of controlled investigatory explorations. It is this feature that is generally thought to make early-stage surgical innovation resistant to classification as ‘research’, given the attendant methodological and ethical obligations—of planning, regulation, monitoring, reporting, and publication—associated with that classification.

Yet, there are compelling grounds—both conceptual and ethical—for proposing that innovation in surgical technique might, in fact, be usefully classified as a form of surgical ‘research’, albeit of a sui generis kind. This paper considers the foundations and implications of adopting a ‘sui generis research’ classification for surgical innovation. While my initial goal is purely conceptual, the arguments in this paper provide the necessary foundation on which specific forms and mechanisms of ethical review and oversight can in future be developed, so as to be amenable to the complexities of innovation in surgical practice and compatible with the needs and expectations of surgeon innovators.

This paper responds to the problem recently reiterated by Angelique Reitsma and Jonathan Moreno, that ‘the current system of definitions, ethical theories, and voluntary professional guidelines may be inadequate to meet the challenge of surgical innovation’ [1, p. 797]. A particularly vexing aspect of the difficulty is encapsulated by what has become known in the surgical innovation literature as ‘Buxton’s Law’, which holds that in regards to the challenge of identifying an appropriate point at which formal regulatory review of some investigative process is required, ‘it is always too early [for rigorous evaluation] until unfortunately it is suddenly too late’ [2].¹

There are a number of possible respects in which ‘too-late’ regulatory oversight of surgical innovation can be unfortunate. Clearly, as explained by Jane Johnson et al., significant ethical risks are associated with surgical innovation, not least, including risks of harm to patients such as have arisen with innovations like routine episiotomy and gastric freezing for peptic ulcer disease [4, p. 1113]. Yet, while innovation by its very nature imposes some degree of risk, the specific concerns of interest to me in this paper pertain not exclusively to direct harm to patients caused by unsuccessful attempts at innovation but to both successful and unsuccessful innovations. On the one hand, successful innovations sometimes receive uptake in spite of not having been subjected to thorough, controlled investigation. Where that happens, knowledge dissemination is ad hoc and contingent upon surgeons’ professional networks and word-of-mouth, rather than systematic, guaranteed, and wide distribution. On the other hand, unsuccessful early innovations (or attempted innovations) are commonly not reported or published at all, hence inhibiting the knowledge transfer essential for preventing future similarly fated attempts by other surgeons unaware of the infeasibility of the intervention. Potentially significant knowledge dissemination deficits therefore occur in both domains.²

As a mechanism specifically aimed at avoiding some of these knowledge-transfer deficits or ‘misfortunes’, I want to consider whether even early-stage surgical innovation could plausibly and fruitfully be conceptualized—essentially if not comprehensively—as a type of ‘research’ activity, where the express goal of conceiving of it as such would be to improve knowledge transfer and thereby ultimately benefit patients. While the argument in this paper is primarily conceptual, a proposal such as this has potentially significant practical implications, which I cannot fully consider here. More specifically, my aim is to propose a way of conceiving of innovative surgical procedures (such as in novel applications of, for example, laparoscopic or robotic surgery or off-pump cardiac surgery) that might assist with clarifying certain specific knowledge transfer-related obligations that ought, but currently do not, automatically accompany early-stage innovation. Conceiving of early-stage surgical innovation as ‘research’ of a special, limited kind can also go some way towards mitigating the potential anxiety about the stifling of innovation that is likely to greet a proposition that even early-stage innovation ought to be understood as a research-type activity.

Framing the reasons for pursuing this line of enquiry as I have done highlights something important about my goal and method in this paper. In particular, the definitional proposal I offer here is explicitly (and fairly narrowly) purposive, and my purpose is essentially practical rather than theoretical. Specifically, what I am advocating is what Walter Sinnott-Armstrong refers to as a precising definition [6, p. 187]. Starting from the observation that definitions comprise different types, Sinnott-Armstrong notes that ‘different kinds of definitions serve different goals and meet different standards’. According to his taxonomy, definitions can be lexical (basing themselves on empirical findings about actual usage of terms); they can be stipulative (informing us of how the term is intended to be used); they can be theoretical (specifying a use to meet a purely theoretical purpose); and, crucial for my purposes here, definitions can be precising (that is, specifying ‘a precise use within the vague limits of common language in order to serve some practical purpose’ ([6, p. 188], italics added). In the case of precising definitions in particular, Sinnott-Armstrong points out that it is not truth or accuracy that will determine their appropriateness. Instead, these definitions are subject to a pragmatic test, namely, of how well the precised definition serves relevant particular interests. Of course, any definition will need to lie within the loose limits of common language relevant to that which is being defined, but many alternative definitions may do so. The only way to pick out one definition as superior is in terms of its usefulness as determined by reference to the specific goals and interests at stake.

For my purposes here, the specific practical goal in question is that of minimizing the failures of knowledge transfer and dissemination that sometimes occur due to uncertainty about the point at which a new surgical technique ought to come within the purview of ethical and regulatory oversight—failures that occur, that is, as a result of the playing out of Buxton’s Law. Accordingly, the merit of adopting a precised definition that classifies early-stage surgical innovation as sui generis research will need to be judged in terms of whether doing so will indeed minimize these failures of knowledge transfer and without imposing other unreasonable and avoidable costs.

As noted, whether or not surgical innovation is construed as a form of surgical ‘research’, even in its incipient stages, will be a matter of significant potential consequence, since the determination of an activity as a research-type activity standardly imposes a range of obligations, specifically in terms of regulation and monitoring of the intervention, as well as of reporting and disseminating results. Given this, it is interesting to observe something of a ‘disconnect’: on the one hand, there is the apparent willingness of many surgeons and commentators—reflectively, at least—to characterize attempted surgical innovation in terms akin to those used for defining ‘research’. On the other hand, there is what has been referred to as the continuing ‘substantial under-representation’ of the gold-standard for research in medicine, namely, randomized controlled trials, or RCTs [1]. As has been pointed out elsewhere, RCTs in surgical research have been reported to constitute only 9% of surgical reports in 1993 and 8% in 2006 [7, 8]. There are of course, many features of surgical innovation that are difficult to translate into RCTs [1, 7]. Nevertheless, both the literature and empirical evidence indicate widespread acknowledgement that surgical innovations are significantly ‘experimental’, even if that acknowledgement does not, on its own, entail automatic designation as ‘research’. Back in 1978, the Belmont Report recommended that ‘major departures’ from standard or accepted practice—so-called significant innovations—ought, at an early stage, to be made the object of formalized research studies, though what constitutes a ‘major’ or ‘significant’ innovation was not elucidated [9, pp. 3–4]. Also evident from the more recent surgical innovation literature is the fact that commentators and surgeons consistently express concern about the lack of a clear mandatory regulatory framework for introducing new surgical procedures and the extent to which surgical innovation remains significantly self-regulated, unlike for the introduction of new devices and pharmaceuticals [10]. Accordingly, it is claimed that surgical innovations routinely go unreported and regularly achieve uptake without trial completion, full ethical review, or routine extra-institutional reporting. Commonly referred-to examples of surgical innovations that are alleged to have been largely unregulated (or highly ‘variably’ regulated) include adult-to-adult live donor liver transplants and laparoscopic cholecystectomies carried out during the mid-1980s and 1990s [1].

In relation to early-stage surgical innovation in particular, the realities of practice thus appear to fall short of endorsed ideals and aspirations.

A plausible explanation for this ‘disconnect’ is a lack of clarity concerning precisely when a change in surgical procedure truly constitutes an innovation. The difficulty of demarcating routine variations in practice from surgical innovations is well documented [10, 12, 13]. Martin McNeally and Abdallah Daar note the challenges that surgeons confront in judging whether what they are doing is ‘an evolutionary variation on a standard procedure, a unique departure from accepted standards, or the first stage of what should become recognized as a formal surgery research project’ [13, p. 930]. Dan Riskin et al. point out that such demarcation is greatly complicated by the fact that the practice of surgery is ‘steeped in innovation’ and that, according to at least some conceptions of innovation, ‘most surgeons innovate on a daily basis, tailoring therapies and operations to the intrinsic uniqueness of every patient and their disease’ [14, p. 688].

Interestingly, the primary problem thus appears to be with identifying specific activities as instances of innovation, rather than with acknowledging that innovation, where it occurs, is essentially experimental, and perhaps even an embryonic form of research. Of course, reticence in acknowledging that a particular surgical intervention is an innovation may also be grounded in assumptions about the potentially burdensome ethical and regulatory requirements associated with surgical research. Such assumptions may incline surgeons towards an assessment that what they are doing is a mere ‘variation in practice’ of a kind that is routine and integral to surgery, and unencumbered by the need for full-scale peer review, ethics approval, and reporting requirements. This indicates a need for greater conceptual clarity and guidance both about what properly constitutes an innovation and about what ought to be the proper regulatory requirements for early-stage innovation.

One definition, adopted in a series of recent Lancet papers from the Balliol Collaboration, describes an innovative procedure in surgery as ‘a new or modified surgical procedure that differs from currently accepted local practice, the outcomes of which have not been described and which may entail risk to the patient’ [3, p. 1089]. Notably, however, this definition requires only a divergence from currently accepted local practice with undescribed outcomes and some potential risk. In its omission of the favoured modifiers requiring that the change in practice be ‘major’, ‘significant’, or ‘substantial’, and that the risks be ‘increased’ or ‘unknown’ as compared with those of the standard practice, this definition seems too broad and blunt. It looks likely to capture some minor and fairly routine variations along with the really significant departures from standard practice, and could therefore trigger unnecessary and overly burdensome levels of regulatory oversight.

Notwithstanding a level of uncertainty, once a surgeon recognizes that a planned intervention will indeed be innovative, there is generally a willingness to accept that the activity is experimental and, to that extent, akin to surgical research. This recognition in turn prompts attention to be directed to the established procedures and protocols of ethical approval and regulatory oversight.

However, in a notable subset of cases, the innovative nature of a procedure or technique is appreciated by a surgeon only in retrospect. That recognition depends substantially upon a crucial subjective change within the investigator’s consciousness, described by Barkun et al. as ‘the belief that the innovation represents a novel approach and a desire to communicate the innovation and to assess its usefulness’ [3, p. 1109]. Since it is only once that conscious determination has been made that there is acknowledgement of the need for formal scientific and ethical review and regulation, this amounts to a significantly subjectivist foundation for the attribution of research-related obligations. The concern thereby raised is that such a determination is overly discretionary and self-regulated, may occur too late in the innovative process, and/or may result in significant innovation-related activity going unacknowledged and unregulated. ‘Thus’, the Balliol Collaboration says, ‘in many cases, between innovation and harm to the patient lies little more than a surgeon’s sense of responsibility, dedication, and fear of medico-legal consequences’ [3, p. 1091].

The IDEAL paradigm does not explicitly take a stand on the underlying conceptual question of whether surgical innovation, even in its early stages, ought to be conceived of as a research-type activity. What it makes clear, however, is that regarding interventions involving human subjects at least, innovation becomes subject to regulatory review only at the Development phase, a point at which the experimental intervention has been deemed sufficiently promising to warrant its extension to 10–30 patients.³

It is clear that the characteristic non-reviewability of Stage 1 experimentation—at least with human subjects—results from a range of factors, many of which may constitute insurmountable obstacles to meeting the requirements of full regulatory review. As noted, innovation at this early stage may be entirely spontaneous and unplanned or may involve only minimal advance planning. Outcomes will by definition be uncertain, potentially even highly so, involving not just unknown but, in some cases, essentially unknowable risks. Furthermore, and largely as a result of these factors, early-stage innovation is also unlikely to be able to meet the full requirements that standardly apply for attainment of fully informed patient consent.

These factors certainly impose significant obstacles to full prospective review, especially in cases of unplanned innovation. Setting aside unplanned innovation, however, the question is: does the impossibility of full prospective review of early stage innovation entail that review of any scale or scope is likewise impossible or unfeasible? In the following section, I present considerations against such a conclusion.

I have already noted that for innovation at Stage 1 (on the IDEAL model) ex ante research-related obligations will typically not arise, and this is an immediate function of the relative lack of planning and prospective design involved. Furthermore, while surgeons are of course subject to all of the usual professional and legal requirements of patient care (including consenting patients fully and treating them with respect), the absence of prospective approval means that research specific in situ obligations are also not applicable for early-stage innovation.

Matters differ somewhat, however, with respect to ex post obligations. Unlike in the case of ex ante and in situ obligations, the absence of ex post obligations in early-stage innovation—and, in particular, those pertaining to dissemination of the intervention results—creates a significant opportunity cost, and this opportunity cost consists in reduced knowledge transfer. As is well understood, knowledge transfer is highly likely in cases of successful early attempts to innovate, as surgeons will seek to extend the benefits of the improvement to other patients and will be happy to report their achievements—perhaps in case notes or series and informally. Yet, such knowledge transfer is clearly highly selective: only successful innovations will reliably be communicated in ways that can advance knowledge in the surgical discipline. Early-stage unsuccessful innovations are, by contrast, far less likely to be reported.

Where information is reliably communicated in relation to both successful and unsuccessful innovations, the outcome can be referred to as inclusive knowledge transfer. Only inclusive knowledge transfer optimizes the realization of potential benefits to patients, by ensuring both uptake of successful innovations and avoidance of unsuccessful ones. Yet, the absence of review and regulation of early-stage innovation clearly prevents inclusive knowledge transfer. As a result, significant opportunities—both to minimize the incidence of unnecessary interventions and to optimize efficiency in the continued development of successful innovations—are foregone. That loss of knowledge transfer can potentially contribute to significant avoidable harm to patients.

In view of this not-insignificant opportunity cost, is there good reason to consider that the imposition of any research-related obligation is appropriate only where all three domains of research-related obligation can be imposed? I would suggest not. Specifically, I would argue that it is perfectly feasible for ex post obligations to be imposed even where ex ante and in situ obligations cannot be. One way in which to conceptually facilitate this is precisely by designating even early-stage innovation as essentially a research-type activity, albeit of a special or sui generis kind.

I need to get clearer, however, about the conceptual grounds on which this might be done. Answering this question requires an exploration of what it is that, most centrally, defines research.

More specific to contexts of surgical innovation, however, early-stage innovation can conceptually be captured within the scope of a definition of ‘research’ via identification of what is common to the core activities involved in both surgical research and surgical innovation, including at their earliest stages. Specifically, we can understand the core of both activities to involve, centrally, the experimental pursuit of skills, techniques, and knowledge aimed at benefitting current and future patients. At the earliest stage, the intended beneficiary of an attempted innovation will exclusively be the patient in the surgeon’s immediate care. However, whether consciously articulated or not, the intention to apply and generalize the knowledge and skills gained from that individual intervention remains central to a surgeon’s motivation, even at the very inception stage of the innovation. There will almost certainly be an intended extension of any improvement beyond the initial patient; no credible surgeon would refrain from seeking to generalize an improvement to other patients and contexts where initial results suggest feasibility and efficacy. Reflecting this combination of therapeutic goals with knowledge-enhancement goals—a combination that lies at the heart of surgical innovation—Lisa Bortolotti and Bert Heinrichs note that ‘knowledge in biomedicine always has a long-term therapeutic function’.⁴ Accordingly, they acknowledge the difficulty of determining—with respect to activities like the application of unproven therapeutic interventions in medicine—whether or not such activities constitute research activities, noting that ‘[it] is perfectly possible to conceive of circumstances in which an activity is aimed at both providing therapy for a patient or a group of patients and extending a body of knowledge’. Early-stage surgical innovation seems to constitute precisely the type of activity they have in mind when they say that in some cases ‘there are no sharp boundaries between research and therapy’ [11, p. 167].

Understood in this way, even a very early-stage innovation can be conceived as a research-type activity, to the extent that even where it initially aims primarily to be clinically therapeutic, it cannot help but embody goals of advancing and generalizing knowledge more broadly. To be sure, it is not research in the full-blown sense, to which apply the full battery of requirements of formal prospective review and regulatory oversight. It is research in a special, more limited sense. And while not sufficient to trigger the full suite of ex ante, in situ, and ex post research-related obligations, even early-stage innovation can and should incur at least the knowledge transfer-related ex post obligations of research. Such obligations can be fulfilled even where innovations occur in an unplanned, spontaneous fashion—that is, even where it can only be retrospectively determined that a change in surgical practice amounts to a surgical innovation.

Importantly, the focus on ex post obligations for early stage innovations is not intended to preclude ex ante obligations. Where an innovation is planned, at least some standard ex ante research-related obligations will also be incurred (the number and type varying with the extent of the planning). These include (but are not limited to) obligations to inform patients of the proposed new intervention and the grounds for its introduction; to provide an accurate indication of the surgeon’s level of inexperience with the particular procedure but also her proposed competence to undertake it (based on related experience and expertise); to provide an accurate indication of any anticipated risks or issues likely to arise from attempting the innovation; and to obtain the voluntary informed consent of the patient to undergo what he or she understands to be an innovative procedure (which may require significant patient counselling and consultation).

Each of the obligations in Table 3 warrants further discussion than is here possible, and many additional questions and objections will arise, including in regards to the implementation of such requirements. Particularly challenging will be questions concerning how a culture of online reporting to early-stage innovation registries can be developed and supported within surgical practice.⁵ I believe that to be effective, such reporting needs to become routinized and part of surgical culture, rather than enforced as mandatory. Yet, while not without its challenges, surgery can certainly learn from other sectors and industries how to mainstream these kinds of processes in ways that minimize the compliance burdens while maximizing the benefits they potentially afford.⁶

I have proposed conceptual grounds for designating early-stage surgical innovation as essentially a type of research activity. In addition, I have argued that the fact that unsuccessful surgical interventions (not only successful ones) generate knowledge that can benefit present and future patients (or at least prevent avoidable harm to them) means that clear ethical grounds exist for a more robust requirement of inclusive knowledge transfer than is currently demanded of early-stage surgical innovation.

Furthermore, there is a widely acknowledged need to reduce the level of dependence that knowledge transfer-related obligations have on surgeons’ own subjective determinations of whether what they are doing constitutes innovation and is hence reportable. Strategically encouraging a professional culture in which surgeons are trained to err, from the outset, on the side of thinking of novel approaches and technique changes as essentially research-type activities would contribute significantly to this. Designating early-stage innovation as sui generis research provides the needed conceptual foundation for triggering ex post obligations, in particular, those designed to facilitate inclusive knowledge transfer. It is with this objective, rather than any purely theoretical goal, that I have advanced here the proposal to construe innovation as a research-like activity.

Having cleared the conceptual pathway for such a designation, the next task will be to determine the specific means and mechanisms by which to convert conceptual foundations and moral justifications into practical feasibility and manageability, and by which to enculturate a transformed conceptualization of early-stage innovation so as to maximize health outcomes for current and future patients.