Dr Wise Young said: ‘We’re working hard on phase three for a “miracle cord” whatever, whatever’, I won’t go into details’. So, they’re recruiting people in India for phase three clinical trial applications in 2016. In the mean while they have decided to do phase 2-B trials in China, India and US. But When you start a phase 3 trial, you first need to have completed a successful [phase] 2-B trial, right? I did not see any publication about it …. I just want to know from the people who know him in his own country [the US]: Had he passed a successful phase 2 trial, would he have come to India?
Introduction
This chapter examines the ideological dimensions of regulation as safety valve and guarantor of reliable scientific research and clinical interventions against the background of the expanding ‘freedom to choose medicine’ (FTCM) movement as a force against the pharmaceutical industry and the strict regulatory requirements for clinical trials in an international context. From the point of view of an increasing number of health activists in wealthy nations in Europe and North America, the world’s neoliberal economy pits the interests of medical industries against those of patients (e.g., Fisher Reference Fisher2007; Batt Reference Batt2017). In the US, strict FDA regulation for clinical trials and the marketing of medical products would continue the national monopoly of the pharmaceutical industry on the drugs market. It would leave little space for competitors with innovative solutions to serious patient conditions.
The urgency of this situation became manifest when patient movements increased their public visibility in the media in the 1990s (e.g., the film Dallas Buyer’s Club), campaigning for an acceleration of the clinical development of new drugs, ranging from medicine for orphan diseases to intractable conditions such as HIV (Epstein Reference Epstein1996). Critical theory holds that the new health activism no longer passively waits for the hegemonic international life-science industry to produce cures. Instead, health activists demand that patients’ wishes are accommodated and healthcare products are made available earlier (Salter et al. Reference Salter, Zhou and Datta2015). In this view, the redemptive force behind regulation is a myth. Rather than protecting patients, it hinders the development of life-saving drugs and therapies; and rather than enabling the clinical translation of medicine through scientific clinical trials, it slows down the efforts of industries that genuinely support patients by focusing their research on early marketing rather than abstract scientific questions.
In this century, a trickle of patients opting for stem cell therapies abroad grew into a vast global movement of stem cell tourism (Song Reference Song2010; Chen and Gottweis 2011; McMahon Reference McMahon2014; Salter et al. Reference Salter, Zhou and Datta2015). At first, countries in Latin America and Asia became favourite ‘therapeutic’ destinations. Companies of all sizes began to champion the case of hopeful patients and invited them to receive the ‘most advanced’ stem cell interventions, both paid and unpaid (Brown and Micheal Reference Brown and Michael2003; Petersen et al. Reference Petersen, Seear and Munsie2013, Reference Petersen, Munsie, Tanner, MacGregor and Brophy2017; Bianco and Sipp Reference Bianco and Sipp2014; Turner and Knoepfler Reference Turner and Knoepfler2016; Sipp et al. Reference Sipp, Caulfield, Kaye, Barfoot, Blackburn and Chan2017), some even using ‘recruiter patients’ to persuade other patients to spread the word (Patra and Sleeboom-Faulker Reference Patra and Sleeboom-Faulkner2009). Growing access to the internet and global travel (Keck and Sikkink Reference Keck and Sikkink1998) enabled increased numbers of patients to join the swelling flow of medical and stem cell tourism, and the interest in the global provision of drugs and therapies grew exponentially.
Patient organisations began to compare regulation for clinical trials, prices for interventions and the experiences of patients and invested efforts in international activities. These increasingly involved knowledge exchanges and local support for patient movements in LMICs. Thus, the International Organization for Muscular Dystrophy (IOMD, pseudonym) ‘reaches out to countries where limited information about Duchenne Muscular Dystrophy is available’ by facilitating the exchange of information about fundraising and lobbying activities among (associate) member organisations, while the US-based International Organization for Spinal Cord Injury (IOSCI, pseudonym) aims to unite and empower the international spinal cord community and to cure paralysis through advocacy, education and support for research.
Although Western patient movements established international branches, their agendas were underpinned by medical and regulatory goals that suited their societies. This led to frustration, as not all countries share the tendency of Western patient organisations or, as we shall see more aptly, health organisations (HOs) to assert their agency through individual initiatives and activities to gather information aimed at accelerating the translation of science into clinical applications that can cure patients with serious, protractive diseases (Rabeharisao et al. Reference Rabeharisoa, Moreira and Akrich2014). For instance, a representative of the IOMD wondered why Asian organisations do not accept their scientific information, believing that: ‘It would help them understand what science can or cannot do for them’ (Van Kindel, 16/2/2015*). Although this representative found it ‘understandable that people at war, the unemployed, or the abused may not be able to become active’, they found it ‘puzzling that countries do not collaborate with us more closely’ (Van Kindel, 16/2/2015*). Nevertheless, experience told them that, within Europe and the US, there are differences in activism cultures. In Scandinavia, they explained, there are high tax levies, so parents often assume a passive role, expecting the state to provide welfare. By contrast, parents in the US take a more active role, whilst in the Netherlands they emphasise enjoying life in the present. For activism, they concluded, ‘you need an entrepreneurial culture; where it is lacking, such as in Eastern Europe, parents tend to wait for government assistance’ (Van Kindel, 16/2/2015*).
HOs activities have increasingly included efforts around knowledge gathering, taking initiatives for research, managing records and data and looking into scientific research. Health organisations concerned with muscular dystrophy (MD) and spinal cord injuries (SCI) (Rabeharisao Reference Rabeharisoa2006; Sakai Reference Sakai2014; Anderson and McCleary Reference Anderson and McCleary2016; Epstein 2016; also see Brown et al. 2004) are no exception. But international HOs have come to focus on issues relevant to some Western societies only. For instance, various HOs in the US campaign for patient freedom to choose medicine (FCTM) include medicines that have not received permission for clinical trials or marketing by the relevant authorities. Supporters of FCTM, including US right-wing political groups such as the Illinois-based Heartland Institute, propose that, rather than the government, patients and doctors should make their own health-care choices, as it ‘would lower the cost of pharmaceutical drugs while expediting the approval of potentially life-saving treatments’ (Herrin Reference Herrin2020).
A core concept in FTCM is that clinical trial sponsors should be allowed to begin selling investigational products to patients while phase 2 studies are still underway to save the lives of patients (Madden Reference Madden2010). Criticising that ‘prohibitive’ regulation as bureaucratic, lobbyists support ‘deregulation’ as the expression of liberty and responsible health choices. Rather than protecting patient interests, then, in this view clinical trial regulation in the US protects a particular kind of clinical science. For supporters of FTCM, the, ‘deregulation’ will facilitate and expedite the marketing and, therefore, availability of life-saving therapies. From an international perspective, however, this redemptive view of deregulation can be questioned.
Questioning the ‘Redemptive’ Nature of Down-regulation in an International Context
Western individual human-rights discourses defined by the global privileged may lead to the ‘normalisation’ (Canguilhem1989; Foucault Reference Foucault and Davidson2003) of a reality defined by the globally powerful, failing to take into account the position of less powerful groups in the world. Considering the societal roots of disability (Shakespeare Reference Shakespeare1993) and biological reality of impairment, it is not likely that all impairments will be mended and all genetic ‘abnormalities’ prevented. ‘Disability’, therefore, is not something to get rid of as soon as possible but to be accepted and accommodated by society. As such, the identities of local health movements from the South need to be incorporated in order to counter the hegemony of the ‘Northern’ disability discourse (Meekosha and Soldatic Reference Meekosha, Soldatic, Soldatic and Meekosha2014: 24). Identity is not merely a social construction or by-product of power; it is also ‘a way of inhabiting, interpreting and working through an objective social location and group history’ (Alcoff Reference Alcoff2006: 42). Other-ability movement scholars, such as Tobin Siebers (Reference Siebers2011), view other-abledness as an embodied identity and define ‘disability’ as an ontological category. The extent to which improvement can be identified and realised, then, depends on the availability of resources, the socio-political environment and concepts of identity.
In the context of regenerative medicine, we must ask whether stem cell therapies occupy the top of the wish list of patients, as seems to be presumed by life-science industries. Where stem cell therapies are presumed to address patient needs, we must ask if down-regulation is always in their favour. Globally, there is no transcendental authority that can tell patients which countries have adopted the ‘best’ regulation, and which therapies are ‘safe and efficacious’, ‘extortionate’, ‘placebo’ or ‘snake oil’. This chapter aims to recognise the wishes and needs of HOs that represent patients with different possibilities and embodied identities in different parts of the world.
Bringing Health Organisations Together to Discuss Needs, Challenges and Governance
On 18 and 19 May 2015, my colleagues and I convened an international network workshop in Brighton for HOs, UK.Footnote 1 Apart from being an opportunity to network with international HOs, the meeting aimed to identify factors HOs consider when setting priorities in addressing their challenges and needs. As it seems obvious that HOs for different conditions have different needs and challenges, a conversation among the representatives of HOs for conditions such as spinal cord injury (SCI) and muscular dystrophy (MD) from various parts of the world seemed useful for consideration of internationalising particular activities. SCI involves damage to any part of the spinal cord or nerves at the end of the spinal canal – often causing permanent changes in strength, sensation and other body functions below the site of the injury. MD, of which there are many types and subtypes, is a hereditary condition marked by progressive weakening and wasting of the muscles. Delegates from five patient organisations for SCI (South Korea, India, Europe, US, Japan) and six for MD (China, Taiwan, India, Europe) participated in the meeting.
A focus on the conditions of MD and SCI is relevant to the question of the needs of patients and their HOs and how to address them. First, MD and SCI are often mentioned in the scientific literature as urgently requiring biotechnological solutions (Sohn and Gussoni Reference Sohn and Gussoni2004; American Academy of Orthopedic Surgeons 2016). In HICs in particular, significant government funding and scientific efforts are invested into finding a ‘cure’ for these serious and intractable conditions. Second, individuals with MD and SCI have diverging disease histories and treatment, which are likely to affect the aims and activities of HOs. And, third, former research indicates that people with MD in Japan are much less preoccupied with regenerative medicine compared to individuals with SCI and that persons in different parts of the world to a different extent look to science, the state, alternative therapies and society for different ways of managing disability (Kato and Sleeboom-Faulkner Reference Kato and Sleeboom-Faulkner2018).
At the meeting, HOs discussed their main challenges, the governance of their organisations, treatment and research needs and experiences with collaboration with industry and knowledge activism. In break-out groups and plenary sessions, discussions were held both among delegates with the same medical condition from different regions, and among delegates with different medical conditions from the same region. Discussions were recorded, transcribed and analysed in terms of the thematic contents of priorities, the pros and cons of working with industry and the importance attached to scientific innovation and regulatory facilitation. Although delegates emphasised the global nature of problems related to SCI and MD, discussions were usually self-framed in terms of wealthy (HICs) and poor (LMIC) and Asian and Western forms of governance. Significant examples (Timmermans and Tavory Reference Timmermans and Tavory2012) related to the question of how to address needs and challenges were used for this chapter.
During the workshop, ‘patient organisations’ defined themselves variously as patient organisations, halfway houses, projects, associations, foundations, alliances and networks. Referring to them collectively as ‘health organisations’ (HOs) seems appropriate, as they share a focus on activities aimed at supporting, maintaining and improving health conditions. Not all members of HOs can be considered ‘patients’ (also see Rabeharisoa Reference Rabeharisoa2006), in the sense of being under treatment by a medical institution. Even persons who live with serious conditions, such as SCI and MD, often do not self-identify as patients (Kato and Sleeboom-Faulkner Reference Kato and Sleeboom-Faulkner2018), while family, friends and professionals can also be members of HOs and sometimes even dominate them (also see Landzelius Reference Landzelius2006).
The sections below explore whether the wish for down-regulation in the interest of patient movements is universal. The first section asks what HOs view as their main challenges and how they relate to political governance; the second explores the needs of members of HOs per condition and per location and their preferences for treatment and research; the third reflects on collaboration with industry and knowledge activism. The conclusion of this chapter considers the implication of the redemptive ideology of FTCM and down-regulation for DM and SCI in an international context.
Challenges for MD and SCI Organisations and Political Governance
The discussion of the challenges faced by HOs allows us to reflect on the universal benefits and feasibility of Western modes of political activism in different countries. HOs for MD and SCI share various challenges pertaining to treatment: there is a lack of knowledge of patient medication, and symptoms that affect treatment such as pressure sores, numbness, bladder- and bowel-management and long waits for clinical trials. They also share problems related to social infrastructure and information, such as an accurate registration of persons with disabilities to provide adequate care and to decide what kind of research is needed and public awareness of disability. But they also have different priorities. Although all the HOs referred to the need for medication and appropriate equipment that allows for bladder- and bowel-management as a most urgent priority, only MD organisations emphasise the prioritisation of greater awareness of prenatal genetic testing and the need for research into pain management and methods to raise the quality of life, while SCI organisations refer to the need for housing, education and employment after hospitalisation as essential to the return of persons with SCI to society. In other words, the first focuses on the medical condition, while the second aims for ‘return to society’.
Daily life entails different challenges for the two conditions. As MD is diagnosed from an early age and indicates a low life expectancy, many persons with the condition identify with MD and belong to a MD community. Persons with MD tend to be represented by their parents, which is why many MD organisations are parent organisations, such as the Parents of Patients with Muscular Dystrophy Organisation (PPMDO). It is usually parents or carers who enquire about clinical trials. This situation contrasts with the tendency among individuals with SCI, who have a relatively high life expectancy, independence and mobility. However, many persons with SCI avoid belonging to an SCI community. One SCI HO delegate said:
Most people don’t actually perceive themselves as being disabled; it’s an acquired condition. I’m still me; I still support Man[chester] United; and I want to go down to the pub with my mates and scream and shout about football and call the ref a ‘whatever’…. One of my trustees went on holiday and had to source an accessible house. He was horrified when he discovered that in the property next door there also was a SCI person …. He said he hated it.
Often acquired in an active life, SCI necessitates the sudden adoption of a new lifestyle. Many persons with SCI try to maintain their pre-SCI social life and independence.
The HOs for MD and SCI also have different relations with clinicians and scientists. Both SCI and MD HOs prioritise immediate needs. But while SCI organisations generally liaise with scientists directly to find a cure, contacts between MD organisations and the science community are often mediated by family members and involve various kinds of research ranging from improving the quality of life, such as by improving heart and breathing function, to potential life-saving interventions, such as experimental gene-therapy.
The lobbying of HOs can be defined by the kind of ‘workspaces’ they occupy. Workspaces here refers to the geographical spaces in which HOs can exert their influence. The ‘local’ workspaces of HOs are subject to a range of political factors, including public support, competition from other HOs, political status and institutional sources of fund raising, such as subsidies, charity and tax. The transnational workspaces they move in are shaped by the availability of resources both locally and internationally and by the politics that drive HOs and transform them. Some HOs complement each other, while others have similar or overlapping functions. For instance, the SCI-network (SCIN) in the UK focuses on fundraising and scientific research, while other SCI organisations deal with more practical services.
Regional HOs and HOs with similar functions sometimes compete for the same resources. Thus, the UK in 2015 had a congestion of nine different SCI charities, which limited their scope for fundraising and research activities. Some countries just have branches of international organisations, such as Sri Lanka, which has only very small grass-root groups and the PPMDO (pseudonym). The status of HOs as non-political organisations (NPOs), non-governmental organisations (NGOs) or grass-root organisations greatly affects the workspaces of HOs: usually, charitable trusts are tax-exempt and government-supported NPOs prohibited from political lobbying, while NGOs may both receive funds and be allowed to lobby. But definitions of NGOs are highly variable. In both India and China, NGOs experience difficulties when trying to register, and restrictive policies have been put in place to stymie international capital flows associated with Western political influence (L.A.W. 2015; CDB 2016). Conditions for charitable gifts vary, too: taxes on legacies, for example, are low in the US but high in Japan (Yamakoshi Undated). The variability of this institutional landscape, as we will see, is closely related to HOs’ budgets and agendas.
The extent to which HOs enjoy support and authority in a country depends on the socio-political space available to them. Most HO/NGOs in China and India focus their activities on information sharing, advising, and offering practical support. Struggling to acquire state recognition and funding for the operation of their organisations, they are usually vulnerable to government interference and are in a weak position when negotiating with industry. A leader of an Indian MD HO illustrates this:
In our country, I don’t think patient organisations have due recognition by the government. They are not viewed as influential bodies or necessary, which reflects on our voice. So, we have to make the government understand what the patient organisation means first …. The space available to interact with the government is very limited.
Other HOs receive recognition and support from the government and have some authority to make decisions about financial spending. Examples are large government NPOs, such as China’s Disabled Persons Federation (CDPF 2016, 2021). Such organisations can advise the government about disability classification systems on the basis of which financial and material resources are allocated to hospitals and patients but have very little clout and hardly any monitoring power over patient activities and needs. More powerful HOs, usually located in HICs, can spawn into large-scale, international networks. They are able to negotiate funding and to monitor government support, steer scientific research in negotiation with academia and industry and forge their own social identities.
Delegates from HOs for SCI in HICs discussed collaboration with industry in terms of asserting rights, self-empowerment and lobbying with governments. For instance, one American delegate explained:
We’re not necessarily growing ours, but we’re spawning other organisations so people get the bug to advocate. We have a toolkit – you don’t have to even leave the house to advocate – you can work on legislation, you can send letters you can make phone calls. This is important and it gets things done; some of this work, to take up an advocacy stance empowers them.
An Indian MD HO, however, emphasised financial requirements:
In developed countries, the patient groups are capable of funding the research towards finding a cure, whereas in countries like India the patient groups are struggling even to survive on their own operations. Meeting the operation costs itself is a big challenge.
A Korean SCI delegate insisted on the political nature of what he saw as the maturation process of organisations for people with disabilities, characterised by political struggle and practical perseverance:
It required fighting – chaining ourselves to fences and protest: it’s in our minds – and not dependent on money.
In brief, countries’ institutional, financial and political landscapes greatly affect the kinds of workspaces in which HOs can operate. Clearly, HOs’ workspaces are conditioned not just by their competition, recognition as worthy causes, tax systems and charity by donors but also by political and judicial support and the constraints of the political regimes under which they emerge.
Therefore, although both HOs for MD and SCI support scientific research, the HOs for SCI emphasises the search for cures and health activism in favour of the acceleration of translational research. In terms of political governance, however, political lobbying is feasible mainly in HICs; Chinese delegates considered political lobbying as inappropriate, while in India, delegates maintained, HOs are struggling even to gain state recognition.
Locally Situated Needs and Demands for Treatment
Insight into the needs, treatment, collaboration with industry and knowledge activism of HOs will provide us with a better understanding of the locality- and condition-based needs of HOs. This in turn, will allow us to reflect on how regulatory policies of lobbying FTCM could affect HOs outside the West.
Locally Situated Needs
When describing the needs of individuals with MD and SCI, HO delegates made clear that they vary per country, especially regarding disposable income, government support in terms of income, policies of equality and public provisions regard traffic, transport, education and healthcare.
Income support for persons with disabilities in China and India is paltry compared with that in Japan, South Korea, Europe and the US. An Indian delegate expressed this as follows:
I don’t want to talk badly about India but 95 per cent of the Indian population is economically challenged. Poverty becomes more of a challenge than SCI.
According to another delegate from India, some 60 per cent of the government aid for individuals goes to intermediaries, ranging from bureaucrats to bribes for the postman. Delegates emphasised that in rural India and China disability registration is important to get access to income support. Without awareness of the need to register, many families do not receive financial support and struggle to look after the ‘disabled’.
An issue emphasised by delegates from India and China but common to all HOs for SCI was the public provisions necessary for wheelchair users, including traffic regulation and accessibility. Its importance lies both in the enhancement of the quality of life and the prevention of SCI. An increase in traffic safety and safety equipment and the aging of society influence the SCI demography. A delegate from the UK explained:
We’ve seen an immense change in the demographics of SCI. Whereas it used to be young active people, like auto traffic accidents and sports injuries, it’s not anymore. In fact, it’s more an aged population. So, we’re seeing people in their 60s and 70s now being admitted for SCI, but I think that’s a consequence of us all living longer and wanting to live independently at home … 50 per cent of SCIs in the UK are non-traumatic so it’s transverse myelitis, tumours in spines. It’s not something that is preventable.
Apart from requesting measures for traffic safety, HOs for SCI from LMICs emphasised the urgency of education for SCI persons to gain employment and the need to combat discrimination on the basis of disability and gender. Though HOs in other societies face similar issues, they were not considered as important. HOs in HICs focused on issues of legal representation, which privilege the rich and emphasised the need for scientific research.
The diverse social and financial concerns of HOs for MD in different locations pertained especially to medical needs, diagnostic services and social status. HOs in HICs emphasise the need for public awareness, facilities in schools and scientific research. By contrast, MD delegates from India and China pointed to the need for more resources for genetic testing and for more expertise and funding for training volunteers with medical proficiency. While Indian and Chinese MD HOs brought up the shortage of rehabilitation centres, they emphasised the availability of Ayurveda and Traditional Chinese Medicine alongside the use of steroids and other medicine for pain management.
As for social status, it was emphasised that in India women are generally expected to look after the disabled. The MD carrier status of women often leads to mothers being blamed and subsequently to divorce and children being relocated to orphanages (Thomas et al. Reference Thomas, Rajaram and Nalinia2014). A Chinese delegate echoed that mothers are victimised and often blame themselves for their child’s condition. But although some parents end up divorced, other couples deal with the condition as a family (Sui and Sleeboom-Faulkner Reference Sui and Sleeboom-Faulkner2010). Japanese research reveals that the birth of a child with MD can easily escalate into a conflict between the families of the parents, though this may have comparatively less severe financial consequences (Kato and Sleeboom-Faulkner Reference Kato and Sleeboom-Faulkner2018). These gender and family issues greatly impact the kind of care and medical needs HOs must cater for.
Table 7.1 summarises the most urgent needs indicated by the delegates of people with SCI and MD in LMICs and HICs. There is a difference in emphasis, whereby LMICs needs for income support, education, diagnostic facilities and rehabilitation centres seem to be more basic than in HICs, where scientific research, legal representation and support for the elderly (SCI) are emphasised. As was pointed out by several delegates, HOs in HICs often assume the availability of basic healthcare to HOs in LMICs. In addition, the role of the extended family in care, severe social stigma associated with disability and the everyday use of alternative medicine rank lower on the agendas of HOs in HICs. This variation in needs and conditions makes for great differences in HO workspaces.
Table 7.1 Locally variable needs for SCI and MD HOs
| LMIC (SCI) | Income support, fair social security and healthcare systems; socio-political awareness, anti-corruption measures Traffic and general safety and affordable mobility devices Combat discrimination of disability/gender |
| HIC (SCI) | Prepare for elderly persons with SCI Legal representation Scientific research |
| LMIC (MD) | Income support and diagnostic facilities Combat discrimination of disability/gender Rehabilitation centres |
| HIC (MD) | Public awareness Facilities in schools Scientific research |
Locally Situated Demands for Treatment and Research
HOs for SCI and MD desire and support scientific research, but their priorities and reasons for pursuing treatment and scientific research differ. All delegates from HOs for SCI hoped for science-based cures, including regenerative medicine, exon skipping – a therapy option for Duchenne MD (Dzierlega and Yokota Reference Dzierlega and Yokota2020) – and robotic applications. There was some agreement among Western HOs that the pursuit of evidence-based medical cures has its drawbacks. An American delegate explained that, in 2015, there were over 700 clinical trials, but clarity about their quality was lacking. Stem cell trials were not an option for her:
The problem for SCI is that regenerative therapies are not testing function at this point in time; they are safety trials. So, when will we know that they work? We don’t know – it could be 10, 20, 30 years down the road.
An SCI organisation from the UK had also become cynical about the timescale:
I have been with my organisation seventeen years now, and back then I remember it was the first international conference [on olfactory ensheathing cells] and all these scientists from all over the world were saying, ‘the cure is only ten to fifteen years away’. They’re still saying the cure is only ten to fifteen years away.
Persons with SCI in HICs weigh their choices:
There are people in full-time jobs with SCI who cannot afford five, six hours a day to do physiotherapy. That’s just unrealistic. It’s not the real world, and the vast majority of people are saying ‘I’ve got more important things to be getting on with.’
With fewer health and educational facilities, patients in LMICs that can afford to do so pay for medical interventions at stem cell clinics, such as Beike Biotech and the Xishan Institute in China and Neurogen and New World in India (Song Reference Song2011; Bharadwaj Reference Bharadwaj2013; Sleeboom-Faulkner Reference Sleeboom-Faulkner2016). Although delegates indicated a lack of reliable information, the use of the internet and WeChat (weixin) facilitates the exchange of experiences and tips about the reliability of experimental medicine.
Clinical trials for SCI – such as those conducted by the Spinal Cord Injury Network (SCIN) – have moved from the West to China and India. The Indian delegate doubted that the Network would have gone to India, had it been successful in recruiting patients in the US. In this delegate’s view, the threshold to participate in clinical trials in LMICs is lower, due to a lack of access to reliable information sources and suitable healthcare and employment options. Primary motives for participation are improved bowel- and bladder-management and regaining the ability to walk. But the delegate’s organisation currently does not recommend any therapies.
HOs for SCI in South Korea and Japan are informed about new treatments through these scientific contacts at home and HOs abroad. In Japan, homegrown therapies, such as iPSC and stem cell–based clinical interventions, exon-skipping and Hybrid Assistive Limb (HAL®) (Walk Again 2015), have gained much attention. To gauge the reliability of clinical trials the Japanese association for SCI usually looks to the government for advice:
We check papers on the Internet – pubmed – and our CEO has a tight relationship with the Japanese government at a high level, and he has contact with famous professors.
In contrast with other delegates, a Japanese delegate emphasised the trust invested in the Japanese state:
If the Ministry acknowledges a practice … then many patients say, ‘Yeah, you know if Japanese Ministry admits it, I trust it’. So somehow, I feel this trust atmosphere in the government doesn’t mean that the government is trustworthy.
The South Korean delegate explained that his organisation, which used to strongly support a well-known, but maligned, stem cell scientist, Hwang Woo-suk, now distrusts scientific promises:
I had met him and he promised to help. I was very excited every day and the Korean government and people were very excited too about how he was wonderful, is wonderful and will always be wonderful, and Korea’s future will be very bright. When a journal showed [his research] to be fake, it was a real disappointment. Some big private hospitals do some research but we cannot trust them. That’s why severe patients go to China or Japan for treatment.
There was agreement among delegates that most people with SCI in HICs focus on rehabilitation, which is also gradually becoming available in LMICs. Discussion ensued on the SCI patients’ duty to do physical exercise. A US organisation considered it as a responsible step towards clinical trial participation:
With SCI you suffer many complications, so if you have a pressure sore, can you really stay in the clinical trial? Please, stay healthy and do what you can; be strong on the rehab end, continue to rehab, when you’re out of rehab do something every day to stay healthy so you can participate in a clinical trial and not have the investment in your participation be lost because you come up with a health issue that could have been avoided, that just slows down the whole process.
The responsibility to remain healthy before participating in a (usually free) clinical trial contrasts with situations in less wealthy areas, where no rehabilitation is available in areas with little wheelchair access and few employment opportunities.
All delegates had closely observed patients go for experimental treatment – with Indian HOs actually arranging for patients to do so. The exception was the Chinese organisation for MD, which only recommended standard medicines: ‘If patients want anything else, they need to find it and pay for it themselves’ (Li, 18–19/5/2015*). HOs in HICs are usually only prepared to provide information but try to record the experiences of patients that decide to undergo experimental clinical interventions. Most HOs object to commercial charges for experimental medicine and criticise it as ‘medical tourism’. HOs for MD in HICs have researched ‘therapies’ advertised in Asia. Delegates emphasised that MD organisations in HICs now avidly follow developments in exon-skipping, gene therapy and iPSC applications. In most countries, HOs for MD support scientific research with data from patients. In some cases, the state mediates these efforts. For instance, in Japan, the Japan Muscular Dystrophy Association (JMDA) is a member of the national MD-research collective called Remudy (Remudy 2021), organised and sponsored by the Ministry of Health Welfare and Labour.
Although all delegates supported the search for treatment and scientific research, it is clear that the cultural politics of countries make for different workspaces. HIC’s HOs for SCI emphasised the need for scientific medical cures. They usually advise against experimental treatment and are sceptical of the ability of clinical trials to deliver miracle cures. Nevertheless, they valued collaboration with industry and emphasise individual responsibility for body maintenance through self-discipline and exercise. In Japan, China and South Korea, delegates emphasised the importance of the state in the search for therapy. In Japan and China, HOs follow government advice about therapies, while in South Korea, faith in state-supported stem cell therapy was lost after the Hwang scandal. MD-HOs attach importance to research and therapies that can ameliorate the condition. Here we see that most HOs advise against commercial treatment claiming to provide cures and encourage long-term collaborations with industry, although some HOs in the past have tried to mediate such treatments.
Collaboration with Industry and Knowledge Activism
HOs recommended collaboration with industry, with or without the mediation of the state and universities, as industry would be motivated to market therapies and appliances. But working with industry entails both opportunities and risks. Here, HOs see opportunities for knowledge activism in the form of information gathering and agenda-setting. These activities underpin lobbying with the state for support, protection and adequate regulation.
Collaboration with Industry
Collaboration with industry can mean a chance to acquire expert medical advice on treatments, especially important for LMICs, and to access up-to-date information about the availability and costs of new medicines, technologies and interventions. It is also an opportunity to put priorities on the research agenda and ensure that the research concerns translational research, aimed at the market. A successful collaboration with industry can mean gaining credibility as a HO. But as we shall see, HOs in LMICs have less leverage to negotiate compared to HOs in HICs.
Collaboration can entail risks. Mentioned as most worrying were the lack of scientific transparency, not having control over goal-setting and the abuse of trust. All HOs worried about ‘getting into bed with the devil’, as expressed by a UK delegate (Brown, 18–19/5/2015*), that is, collaborating with ‘rogue’ companies. One HO from India raised the example of a pharmaceutical company that offered patients a weekly sum of £250 for providing a blood sample, including clinical and other data, without revealing its exact purpose. HOs also fear losing the ability to steer research priorities if they accept funding. Sometimes researchers use patients to find answers to theoretical issues, ignoring the therapy-oriented agenda of HOs. Coca-Cola, for example, would have funded an exercise programme for the ‘disabled’, mainly to improve the company’s unhealthy image. Another anxiety is that some companies try to use patients to acquire insurance pay-outs for them. For example, a delegate related how one exoskeleton company tried to pressurise SCI HOs to persuade GPs to prescribe expensive suits of approximately £100k.
To diminish the risky aspects of collaboration with industry, the British HO for MD advised the use of a strong Memorandum of Understanding (MOU): ‘It is important to stick to moral standards, as some companies prey on our vulnerability’ (O’Hara, 18–19/5/2015*). While the UK organisation recommended raising funds only after identifying clear targets, HOs in India and China emphasised the difficulty of attracting any funding. One Indian HO followed strategies to make themselves attractive as a business partner. For instance, they promised to introduce buyers to a wheelchair company in exchange for wheelchairs with particular specifications. A less successful example in India was the initiative to negotiate low fees for stem cell ‘treatment’ in exchange for patient introductions (Basu Reference Basu2012). The therapy was not successful, and the HO did not have the resources nor the expertise to sue them. HOs from Europe recommended the involvement of regulators that are open to lay-expert input in clinical trials. For example, the ‘unscientific’ end-points, such as ‘the ability of a boy to scratch his own leg’ (Brown, 18–19/5/2015*) have substituted the formerly standard six-minute walk test, which was of little relevance. In Italy, involvement in clinical trials enables the MD community to get useful feedback on psychological aspects and the placebo effect of clinical trials. This situation contrasts with HOs from China and India, which usually approach scientists individually or rely on symposia for information.
Some HOs in HICs are strong negotiators, as they can set their own targets by mediating between industry and scientists. An American HO brings together companies, government officials, clinicians and scientists at conferences and finances research, making sure that its priorities are supported:
We work to direct funding to the best research and I mentioned this before. It’s oftentimes not going to the best research. So, we provide the tools to make sure that it gets there through peer-review, and we also try to encourage replication studies.
A UK HO for MD was aware of the great value of conferences to pharmaceutical companies: ‘They pay anything to speak with patients face to face’ (O’Hara, 18–19/5/2015*). In Japan, similar conferences are held, but the government – an important research funder – plays a more important role as mediator, while in South Korea, the SCI HO has become weary of working with industry after the data-fabrication scandal (Resnik et al. Reference Resnik, Shamoo and Krimsky2006). Short on financial and state support, Indian HOs expressed vulnerability in negotiations with companies. For instance, initially free/cheap drugs can sometimes suddenly become expensive. Though this can also happen in HICs, poor insurance conditions make the drug markets in LMIC unattractive (WHO 2012). In other cases, however, a tiered payment system is used so that poor patients receive free treatment (Hayden Reference Hayden2014). An Indian HO considered the presence of a scientific advisory board and legal representation to be crucial for minimising risk in dealing with industry. The Chinese MD organisation, however, did not collaborate with companies, as ‘they are motivated by profit and can say anything’ (Li, 18–19/5/2015*), and would rather work with scientists in state institutions and the government.
HOs from HICS and LMIs generally shared perceptions of the risks and opportunities of collaboration with industry (see above and Table 7.2), but in their respective workspaces, delegates from HICs displayed more confidence in their ability to influence research agendas, while delegates from LMICs worried more about their credibility. In dealing with risks, the discourses of delegates from HICs were assertive and those from LMICs timid, conveying a sense of dependency: HIC delegates discussed ways of creating a ‘strong MOU’, sticking to moral standards, involving regulators and making demands of universities point to assertiveness, while LMIC delegates spoke in terms of becoming attractive to companies, aiming for long-term, reliable, collaborations and building relations of trust.
Table 7.2 Collaboration with industry: Opportunities and risks
| Opportunities | All | Information gathering regarding rehabilitation, clinical trials, new technology, clinical treatment Influence the research, e.g., endpoints clinical trials Accelerate marketing |
| HICs | Get priorities, e.g., translational research, on the research agenda | |
| LMICs | Enhance HOs credibility | |
| Risks | All | Reputation loss when ‘getting into bed with the devil’ Lose the ability to steer research priorities when accepting funding Pressure on patients to get the state to pay for drugs Losing access to treatment after clinical trial |
| Dealing with risks | HICs | Create a strong MOU, legal protection Stick to moral standards Direct funding to the research best for you Involve the regulators Bring stakeholders together yourself Demand replication studies and contributions from universities |
| LMICs | Create an MOU Make HO attractive to company partners Negotiate treatment costs Set up long-term connections with reliable scientists and companies Refrain from collaboration |
Knowledge Activism
Health or patient activism refers to health activities engaged in creating critical awareness and political, including regulatory, change. Knowledge activism, which emerged in Western HOs relatively recently, supports health activism. Traditionally, HOs engage in health activities that are not necessarily ‘activist’, as it was not directed at political change or addressing matters of social justice. For this reason, the global spread of ‘knowledge activism’ requires a rethink of issues of identity-making and of governance (Rabeharisao et al. Reference Rabeharisoa, Moreira and Akrich2014). A distinction between HOs activities and knowledge activism highlights difference between activities of HOs for HICs and LMICs and between HOs for different conditions.
The activities of HOs include education in schools, of families and the public through books, films and social media and the training of researchers and nurses. Most also have a newsletter, a website, instructions for emergency posts and a registry. In some countries, health activities are more ‘activist’ than in others. For instance, some HOs spoke of ‘liberating people living with SCI’, ‘spreading awareness’ and ‘empowering patients’, while others described their activities in terms of help, acceptance and sharing. The British SCI HO and the international HOs for MD and SCI work on international services to signpost research, clinical trials, IT and practical information, ranging from housing adaptations to bladder and bowel management. Some HOs in HICs engage in specialist education, such as training people in the newest computer technology, assistive technology training for volunteers, occupation therapists and managers so that new technology, such as for mouth control, can be used by many patients.
HOs in LMICs put much effort into activities related to basic needs. A delegate from an Indian SCI HO clarified that India needs accessible traffic and public provisions before it can begin to have a science focus. The HO liaises with government to set up rehab centres, provides wheelchairs and equipment for members and is in the process of compiling an accident and emergency (A&E) manual and a registry for its members. A delegate from a Chinese SCI HO described how they tried to persuade medical centres and the government to establish rehabilitation centres and how they raise money to train people with SCI to re-enter the labour market and to regain their self-respect.
Of the differences between HOs, those related to knowledge activities are most prominent. Knowledge activism concerns the creation and mobilisation of knowledge, including data-gathering activities related to treatment experiences, exchanges of narratives, medication, and medical data potentially usable in the creation of biomedical knowledge, health technology assessment, public health research and forms of judicial expertise.
In the US, HOs try to influence research policies by lobbying government. For instance, the US SCI HO supported a bill for research, The Dana and Christopher Reeves Act, and a freedom of choice bill for allowing therapy provision outside the FDA (Holbein et al. Reference Holbein, Berglund, Weatherwax, Gerber and Adamo2015). Apart from mediating and steering research, this HO demands that scientists replicate studies and persuades universities to waive overheads and match research funding. The British HO for SCI creates scientific reviews of research strategies, organising conferences and workshops for international lay- and expert audiences. The South Korean HO also organises annual conferences with US nurses, scientists and activists to shape the South Korean research agenda. The Japanese SCI HO, while providing information on clinical trials and health management, also emphasises the need for coordinating with scientists and HOs abroad. The organisation lobbies the government and scientists to develop regenerative medicine, robotics and new approaches to rehabilitation and organises conferences and public lectures to support research.
The delegates from MD HOs maintained that their HOs have all set up registries for various forms of MD. But their use differed per geographical location. In India, registries primarily serve as a tool for patients to enter clinical trials, in Europe, to integrate data for scientific research, and, in Japan, to synchronise drug-development with other countries (also see Kumamoto University 2011). The international MD HO engages in data collection for a registry for clinical trials in Europe, creates documents outlining the pros and cons of clinical trials, campaigns for the reimbursement of clinical trial participation costs and organises conferences, workshops and webinars for patients and researchers internationally.
All HOs engaged in social, informational, social media and educational activities (see Table 7.3), but only HOs in HICs provided training to adopt the newest biotech, and only HOs in non-Asian HICs engaged in international information services. The knowledge activism of HOs in HICs in national and international research activities attests to a much higher involvement with scientific research compared to HOs from resource-limited countries, where scientific activities were mentioned only in the form of participating in or being invited to scientific workshops. And it is this expectation to direct activities to global knowledge activism, which can aggravate dissonance between available resources and demands in the workspace of non-HIC HOs. For when it comes to local health needs, it is clear that for LMICs the priority lies with basic healthcare and policy measures (regarding road traffic, discrimination, education and registration), while in HICs education and funding for research/clinical trials are high on the list. Nevertheless, in LMICs innovative regenerative medicine can also be seen as a way of economically addressing both conditions in the long-term.
Table 7.3 Social, educational and knowledge activities
Especially HOs for SCI prioritise support for finding scientific cures, while many HOs for MD regard this as a desirable, though it is a contested subject. HOs for SCI from HICs found evidence-based medicine too slow and preferred to work with industry-oriented researchers that aim to market medicinal therapies. HOs from LMICs expressed worry about such permissive clinical trials, as they are suspected to be of low quality. But due to high healthcare costs, many patients are interested in them. Most HOs keep tabs on them, but China’s HOs refused to do so as they only recommended authorised clinical trials. In South Korea and Japan, homegrown clinical trials have shown much promise, but due to fabrication scandals, there have been setbacks. In Japan, at the time, the state was hesitant to allow clinical trials for SCI and MD.
Only in the West do HOs have a strong bargaining position and are able to influence the scientific research agenda directly; though in South Korea and Japan, HOs can do this through state mediation. HICs have information, facilities and financial resources that they can use to mobilise activism, in contrast with LMICs, where HOs have trouble attracting funding and where due to poor insurance conditions RCTs are not as attractive to industry. Mainly delegates from European and American HOs used notions of patient rights, individual responsibility and political initiative in explaining their workspaces, indicating that individual-rights discourses inhere to political identities widespread in Europe and the US. Although notions of patient rights are deployed in Asian HICs, local political discourses describe workspaces emphasising state guidelines, long-term collaboration and trust. Delegates from China and India mobilised much less human rights discourses in debate, rather emphasising the role of the state in providing financial support, countering discrimination against women and correcting resource allocation between rural and urban areas.
The individual-rights discourses used by HO delegates from HICs portray an assertive form of activism, expressed in discussions about political negotiations, collaboration with industry, their financial clout, their ability to steer research, their rights to lobby with government and their monitoring of ministerial tasks. It is doubtful that individual rights discourses are helpful in efforts to cover basic grass-root needs of disabled peoples in LMICs’ and in Asian HICs, especially as HOs look to the government for support and mediation in their social and medical endeavours. An HO from India remarked: ‘Poverty is a bigger problem to our government than is disability’ (Ghandi, 18–19/5/2015*). This means that expectations of governments are limited to meeting basic needs. When in pursuit of biomedical cures for SCI, there is no expectation that HOs can steer the development of scientific research by mobilising financial support via the state and charities. In India and China, the political and material contexts require a discourse that has covering basic needs as an endpoint; a pragmatism is adopted that favours a discourse conducive to the political identity of HOs within their political environment. Notions of human needs and trust may be of greater tactical use when requesting government and charity support, while individual-rights discourses may be more relevant to negotiations with industry. HOs in Asian HICs also refrain from individual-rights discourses, as a political culture of trust is more conducive to a relation of state dependency.
Conclusion: The Politics of Regulation and the Embodied Identities of HOs in an International Context
One of the meeting’s concluding remarks by a delegate from a HO for SCI sums up a key ideological problem underpinning global activism:
In the end, we [all] have the same possibilities because the older groups can help the younger ones, so if you don’t have some things that we have, for example in Europe, we can help you in having the same rights, the same access and the same conditions in a co-operative way.
A delegate from India reacted:
That’s exactly the catch. The groups in the developed countries can’t help us, because the needs and possibilities are not the same. When they focus on [globally] shared needs of patients, we focus still on our basic needs.
The global dynamics of capitalism privileges some styles of health activism over those of others, showing us that the ‘counter-hegemony’ of the activism in HICs can be hegemonic in itself. Although HOs have needs in common, the historical embedding of HOs show great variety in patient needs and the workspaces that might address them. This is not well reflected in individual human rights–based discourses of international HOs, which, first, tend to exclude discussions on the different priorities of LMICs, who struggle to fulfill grass-root needs; second, insist on the emancipation and independence of patients and the prioritisation of biomedical solutions – lacking awareness of the importance in many communities of the supportive role of the extended family, and of other modes of coping, such as the everyday use of alternative medicine; and, third, tend to be ethnocentric in their advice: for instance, to ‘take responsibility’ through rehabilitation with the aim of clinical trial participation to patients from LMICs may be expensive, dangerous or impossible and the safety and reliability of ‘clinical trials’ and ‘after-care support’ may differ starkly in different parts of the world.
We also saw that there are fundamental differences between the ways in which individuals with SCI and MD embody and identify their conditions socially and politically (Alcoff Reference Alcoff2006; Siebers Reference Siebers2011), to the extent that the former have formed a sense of community, while the latter has not. It seems obvious that, depending on one’s condition, priorities for treatment and care differ and are contingent upon available financial and material support, cultural views about disease, identity and political governance. While persons with SCI often eschew identity politics and aspire to inhabit a ‘normal’ world through quick-fix technologies, people living with chronic conditions may prefer to consolidate their identities of being other-abled. those that embrace their identity as other-abled tend to accept their condition as ‘normal’ to themselves and look for treatment that can improve the lives they lead. Health activism strategies then, are rooted in everyday life conditions, embodied in socio-political identities and shaped by the constraints and possibilities they bring.
This chapter has shown that wealth, where we live and the kind of conditions we suffer shape our views about healthcare needs and the workspaces of HOs, including ‘knowledge activism’, ‘political lobbying’ and ‘collaborating with industry’. In HICs, some influential HOs criticise the bureaucratic slowing down of regulation, its overprotection of ‘free individuals’ and the monopoly of the pharmaceutical industry and its influence over regulation, even where measures are in place to accelerate clinical translation through regulatory clauses and exemptions. For critics, the promise of stem cell research cannot be fulfilled soon enough, so that down-regulation is for them the only way for seriously ill patients to gain access to life-saving treatment. This redemptive pressure to accelerate therapy access is also advocated by international HOs in LMICs, but HOs make clear that their priorities lie elsewhere. But in LMICs, despite the attraction of quick-fix scientific solution, HOs have been suspicious of permissive regulation, which in their view creates uncertainty about the scientific reliability of the therapies available to them. The redemptive effect of down-regulation lies in the belief that it will save lives in a context of ‘strict’ regulation designed to immunise patients from the risks of experimentation. But in HOs in China, India and South Korea, we saw worry about the safety of clinical trials, their efficacy and the fabrication of data; HOs in India were worried about clinical experimentation but also about ‘foreign’ clinical trials that try their luck among the poor when failing to recruit in wealthy countries. At the same time, the growing middle classes in India and China increasingly view stem cell treatment as an opportunity for extending life. They call for reliable regulation. It is important, not just to know which treatments are vetted by ‘international’ standards but to have a reliable compass for gauging the risks and opportunities of ‘as yet’ unauthorised clinical treatments.
Most HOs try to keep a tab on patients that go for ‘experimental medicine’. Decisions to try unauthorised treatment are not just based on hope but also on calculated risk. Ironically, the HOs in LMICs that are hardly recognised by the state expressed the greatest need for official information on the safety of clinical possibilities, while in countries with detailed and transparent regulation, HOs were much more critical of the state. For instance, HOs from the US express little trust that state regulators can fairly balance their ‘rights’ against the interests of large pharma; the European HOs, however, trusted regulators to take their views into account but also criticised them; in India, HO delegates felt that although they were not sufficiently heard by the state regulators, they had to rely on them for information on clinical trials; the Chinese HOs identified with the state; and, the Japanese and South Korean HOs expressed trust in state regulators and emphasised that their voices were heard.
The variety in regulatory trust among the HO delegates partly reflected the political identities of HOs in different parts of the world. And as no global-level regulatory authority exists that can immunise research regulation against violation, patients who can and who can’t afford it are ‘free to choose medicine’ with misleading guidance regarding its potential to heal, provide temporal reprieve or to deliver expensive death. Though, as we saw in Chapter 4, there have been calls for establishing a strong global regulatory authority, it would need to cater for the world’s diversity in health- and regulatory needs. The suitability of regulation within a jurisdiction is contingent upon the material and organisational resources available to HOs in a jurisdiction. And, as we saw in previous chapters, it is the regulatory gaps that have been exploited by entrepreneurs, industry and the state in both HICs and LMICs.
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