Pharma

'Repositioning for Rare Diseases – Too Much, Too Little or Just Right?' by Jakob Wested and John Liddicoat in (2021) NIR: Nordiskt Immateriellt Rättsskydd / Nordic Intellectual Property Law Review comments 

“Drug repositioning” refers to the idea of expanding the use of an already authorised drug, for the treatment of new patient categories with the same diseases as well as the treatment of new diseases. Repositioning is considered an important innovation modality to increase treatments for patients with rare diseases. Yet, two recent EU studies have raised concerns that developers who reposition drugs are financially overcompensated to the detriment of national healthcare systems and patients and, therefore, suggest limiting the benefits provided by the orphan drug regulation. In this study, we show that the number of drugs repositioned for rare diseases (22%) is more modest than predicated and argue this indicates that developers may not be overcompensated, otherwise we would expect a much higher number. Furthermore, we argue that changing the incentives provided by the orphan drug regulation to address overcompensation is not the option if policymakers want to realise the benefits of repositioning. Instead, we suggest policymakers consider other legal and regulatory tools to address overcompensation issues, such as competition law and novel mechanisms emerging in Canada.

Disruption

'Silicon Valley, disruption, and the end of uncertainty' by Susi Geiger in (2020) 13(2) Journal of Cultural Economy 169-184 states 

This paper reflects on the relationship between high-tech disruption narratives and uncertainty. My main argument is that an economic sociology of the future is incomplete without addressing the ‘demonic’ or rather eschatological elements apparent in the promissory twin rhetoric of disruption and inevitability that a number of contemporary technology firms employ. The conjuring up of liberatory high-tech futures implicates a political-philosophical perspective of the end game. It utilizes at once the productive power of uncertainty to create visions of ‘absolute riches’ and societal gain but at the same time narrows these futures down to one inevitable alternative to the status quo. Through the examples of two Silicon Valley disruptor firms, I argue that these eschatological narratives need to be opened to social scientific critique in order to examine their potential societal consequences above and beyond the narrow geographic confines of ‘the Valley.’

 Geiger comments 

 The historian of economics and science Philip Mirowski once said, when talking about the 2008 Recession, that ‘In the neoliberal land of make-believe, if you can sell it, then it must a fortiori be legitimate’ (Mirowski 2012, p. 290). Indeed, the relationship between fictionality and the economy has been subject to much scrutiny in recent years (e.g. Esposito 2013, 2015, Beckert 2016, Geiger and Finch 2016, Meyers and van Hoyweghen 2018). This literature has, by and large, argued that economic expectations, as projections made under conditions of uncertainty, are necessarily fictional – they have a ‘broken relationship to reality’ (Beckert 2016, p. 62). Where fictional expectations around future markets or technologies are circulated, these expectations act as cognitive reference points for stakeholders to buy-into the fiction over time (Brown 2003). These expectations can be strongly affectively laden – they can be taken over by ‘irrational exuberance’ (Shiller 2000, Geiger and Gross 2017). But do expectation-based theories account for the ‘demonic’ undertones that have been diagnosed in the discourses of disruption found in contemporary technology circles (Beckert 2016, p. 285)? 

In this paper, I examine the political-philosophical and quasi-religious colourings of contemporary high-tech disruption narratives, as circulated and perpetuated by many Silicon Valley-type (bio)-technology firms. Where Hogarth (2017) sees these firms suspended in a liminal space between the ‘regime of hope’ and the ‘regime of truth,’ I argue that their disruption narratives work as political technologies, conjuring up a ‘theological unconscious’ (Muniesa 2017), where futures are envisaged as liberatory and essentially faith-based alternatives to the current status quo. Recent works in the sociology of expectations see capitalist futures as multiple and entrepreneurs productively utilizing this multiplicity (Esposito 2013, 2018). In my reading of high-tech disruption narratives, this multiplicity is often paradoxically combined with a narrowing down of all potential futures to one. This political or philosophical techno-vision is elevated to be the only possible alternative to the status quo; it signals the necessary destruction of the old and the creation of a world that clearly parts with whatever has preceded it. In other words, I propose that through advocating ‘the end of uncer- tainty’ disruption narratives gain an eschatological character. 

In its secular usage eschatology signals both the fulfilment of time and the opening up of a new and better future (Pannenberg 2008, Loureiro 2010). In contrast to an expectation-driven rendering of economic futures, eschatologies of disruption thus start with an end state and work backwards, using a metaphorical future perfect rather than the ‘future present’ (Esposito 2018) of imaginaries and expectations: future worlds are wished into existence in a quasi-messianic manner. 

I investigate this proposition by tracing the evolution and narratives of two organizations that, like many other Silicon Valley-type firms, have played heavily on a future-oriented disruptor narrative: the consumer genomics testing firm 23andMe and the blood-testing firm Theranos. My investigation of these two firms’ narratives is based on extensive secondary data and complemented with broader insights from an ethnographic study in the Silicon Valley digital health ecosystem. I empha- size that I am not drawing any factual parallels between Theranos’ alleged fraudulent behaviour and a firm – 23andMe – that operates in the regulatory sunlight. My interest rather revolves around the political-philosophical narratives which these firms build and the effects these may have in conjuring up future markets-to-be. Overall, then, this paper reflects on the relationship between expectations, uncertainty, and high-tech eschatologies that the omnipresent concept of disruption creates.

Charities

Two items in today's The Sunday Age (Melbourne) discuss questions about trusts, charity and the 'purple dollar'. 

The Mormon Church has been accused of engaging in significant tax evasion in Australia, allowing its adherents to collect hundreds of millions of dollars in tax exemptions that are not lawfully available to followers of other religions. Some of its tax activities are run through a shell company, with no paid employees, that could be in breach of Australian tax laws, an investigation by The Sunday Age and The Sun-Herald has found. ...

The investigation has uncovered how the Utah-based Church of Jesus Christ of Latter-Day Saints (commonly called Mormons) appears to have structured itself internationally to minimise tax for its adherents and maximise benefits for itself, in particular in Australia and Canada. In Australia, the church has ensured that donations and tithing – which are not tax-deductible – are routed through a charitable trust to gain 100 per cent tax deductibility. 

Mormons are required to pay 10 per cent of their gross income in tithing, a significant financial impost on followers. Australia is unusual among English-speaking countries in that it does not allow tax deductions for tithing or church donations. But it does allow generous deductibility for charitable giving. The Mormon Church has structured itself to maximise that tax benefit, and reports that it spends up to 70 per cent of its Australian income on charity. 

Globally, the church spends less than 1 per cent of its income on charity. “It’s all a ... smoke-and-mirrors thing,” claims barrister Dr Neville Rochow, QC, who had senior roles in the church, including working as its representative to the European Union until 2018, and previously in pastoral and public affairs roles. He claims the church is “taking it off the back of taxpaying Australians”. Dr Rochow said the church spent little on charity. “They don’t set up schools, don’t set up hospitals, don’t have homeless shelters, don’t have places for women to escape when they have bad marriages.” 

The tax structure has meant that since 2015, Australian adherents have been able to draw on $400 million in tax deductions not available to followers of other religions or denominations. A Mormon Church spokesman did not respond to written questions about this discrepancy, but said the church operated in accordance with tax laws. 

In the mid-2010s, Dr Rochow was involved in a Mormon-instigated econometric study used to justify its tax status and to ensure that religious trust properties of other churches were not taxed. Soon after, it started to direct substantial donations and tithing through its charitable trust – a decision he was not involved in. Before that, in a submission it made to Treasury, the church said it spent just $36,000 a year, on average, on charitable donations between 1985 and 2010. 

There has been little to no growth in the 61,600 Mormon adherents in Australia over the past decade that could explain the reported 2000-fold increase in charitable giving. Each year, funds are transferred from the church’s charitable trust to a separate entity, Sydney-based LDS Charities Australia. From that entity, about $70 million a year is distributed to global charitable causes. The church spokesman said after LDS Charities Australia was created in 2012, “there was a desire and the ability to significantly increase the support of global humanitarian and development projects”. That $70 million a year is close to the amount the global church – whose strongest following is in the United States – itself says it gives each year on average throughout the world, according to its own annual reports. 

LDS Charities Australia has no paid staff, Australian website, expenses or infrastructure to run what purports to be one of the country’s major charities, collecting more in individual donations than Oxfam, Beyond Blue or Caritas Australia, the Catholic Church’s international aid charity. It runs in parallel with the Utah-based Latter-Day Saints Charities, which apparently directs the church’s charitable spending throughout the world and includes all its senior staff, management and infrastructure. ... 

University of Tampa professor of sociology Ryan Cragun, an expert on Mormonism, said it was inconceivable that the church was making significant global charitable decisions from Australia. “It’s a stunningly hierarchical religion,” Professor Cragun said. “So, any big decisions like that are going to be made in Salt Lake City.” 

In 2019, the Australian Tax Office made a binding ruling that for an Australian charity to have deductible gift recipient (DGR) status – which allows tax write-offs for donors – it must have Australia as “the focal point of the DGR in a legal or organisational sense”. Krystian Seibert, a charities expert at Swinburne University’s Centre for Social Impact, speaking in general terms, said the DGR rule meant the operations and management decisions of a charity needed to be made from Australia. “It can, of course, engage and consult with partner organisations outside Australia, but if it effectively outsources operational and management decision-making to individuals or organisations that are outside Australia, that could raise issues under our tax laws.” 

Mormonism is a US religious movement that began in the 19th century and has become incredibly wealthy. A Washington Post investigation in 2020 revealed the church was secretly running a $US100 billion investment fund, Ensign Peak Advisors, that was accumulating vast tax-free wealth by investing in hedge funds, Chevron, Visa, Apple and some of the biggest landholdings in the US. 

Professor Cragun said the church, for a time, disclosed its charitable giving, which equated to less than 1 per cent of its revenue. “It is shocking how little they give,” he suggested. ”But on the flipside, this is the part that’s really interesting: they advertise it every single time. And that’s where the ex-Mormon in me is like, ‘That’s exactly what Jesus said not to do’.“ ... The international church, he said, was run like a multinational corporation, with practices all but identical around the world. According to Cragun, the church is likely to “milk the system for every tax deduction they can... And they’re very, very good at it”.

In another item Cragun is reported as commenting 

... Church leaders live comfortably but all this wealth - the $US100 billion investment fund, the tax minimisation - is not about personal enrichment. Rather, he suspects, it’s about long-term corporate planning. Many of its current leaders - called apostles - have backgrounds in senior positions from the corporate world and run the Church as a business, he says. 

“My sense is they know what’s coming. As far as secularisation goes, they’re losing young people in droves. They’re not really getting any converts in Australia or New Zealand, or most of Western Europe at this point. Even Eastern Europe, they’re not getting anybody, Africa, OK, they’re doing OK there. But even in South America, they’ve kind of mined that,” he says. ”My sense is they’re reading the tea leaves saying… that donating members are going to begin to shrink. How are we going to maintain all the properties that we own, all the stuff that we’re doing?“ He surmises that the “massive war chest” is designed to allow them to “just live off the revenue from that indefinitely into the future, that’s my best interpretation”.

Evidence

Waratah Coal Pty Ltd v Youth Verdict Ltd & Ors (No 5) [2022] QLC 4 addresses an application to take on country evidence from First Nations witnesses ... 

[4] YV and TBA propose the Court take on country evidence from four of the First Nations witnesses and conduct site inspections on Yidinji Nation (Cairns), Erub and Poruma (Torres Strait). 

[5] Site inspections are a routine feature of mining objection hearings. Taking on country evidence is not; but it is a familiar process for a court hearing native title and cultural heritage claims. 

[6] YV and TBA seek the following orders:

Group Evidence 

Athe Kapua Gutchen, Arke Florence Gutchen and Lala Gutchen are granted leave to give evidence in chief, cross-examination and re-examination together as a group. Site Inspection, On Country evidence and evidence in chief 

Evidence will be taken by the Court from Athe Kapua Gutchen, Arke Florence Gutchen, Lala Gutchen and Jiritju Fourmile at the places and on the topics specified in the Proposed Site Inspection and On-Country Evidence Plan as follows: a. Athe Kapua Gutchen on Erub (Darnley Island); b. Arke Florence Gutchen on Erub (Darnley Island) and Poruma (Coconut Island); c. Lala Gutchen on Erub (Darnley Island); d. Jiritju Fourmile in Gimuy (Cairns region). 

Athe Kapua Gutchen, Arke Florence Gutchen, Lala Gutchen and Jiritju Fourmile are permitted to give oral evidence in chief to explain the matters covered in their written statements of evidence. Their evidence in chief is in addition to, and does not replace, their written statements of evidence.

[7] Waratah opposes on country evidence as unnecessary and involving disproportionate costs. It does not wish to cross-examine the witnesses on their statements. 

[8] It opposes leave being granted for further evidence in chief, but, if that were to occur, say it could be given in Brisbane or by video conference. YV and TBA say those arrangements would be entirely inadequate. 

[9] The consequence, then, of not making the orders is that the witnesses’ evidence would be confined to their written statements. 

[10] The statutory party says on country evidence is appropriate and consistent with the Court’s function in this hearing. Mr Brinnand strongly endorsed the orders. Mr Harris, the solicitor for Mr and Mrs Brown, was concerned about the cost. If they could ask to be excused from attending, that would protect his clients’ interests. ... 

[18] The decision about arrangements for taking evidence from First Nations witnesses engages the right protected by s 28(2)(a) of the HRA: to enjoy, maintain, control, protect and develop their identity and cultural heritage, including their traditional knowledge, distinctive spiritual practices, observances, beliefs and teachings. 

[19] Ms Rose deposed to the cultural practices about imparting traditional knowledge and culturally important matters: Kapua Gutchen, Lala Gutchen and Florence Gutchen explained to her that Traditional Knowledge is best spoken about on Country at the place that is being discussed. Jiritju Fourmile said that under Yidinji Lore, the Elders conduct a ceremony when evidence is given where the senior Lore man explains to the Court’s Law people how the Knowledge has and will be transmitted. Those four witnesses say that to comply with cultural protocols: (a) evidence should be given orally at the place which is being discussed given the level of cultural sensitivity and importance of the topic; (b) it is best to be discussed in the company of other members of the community who are knowledge or Lore keepers for particular topics due to the way that knowledge is held collectively; (c) it is best given in the presence of Elders; and (d) a proper explanation of particular topics cannot be done without showing or demonstrating a particular place or impact or landscape on Country and this explanation can’t be done any other way. 

[20] Further, Ms Rose deposed to a particular difficulty for Lula Gutchen giving evidence about certain matters that the parties have agreed will be redacted from her written statement. Ms Gutchen said that under Erub Customary Lore she cannot give evidence about those matters. Certain Elders are Knowledge Keepers for their land before Traditional Knowledge is transmitted. Some of them have recently passed. Ms Gutchen cannot seek permission about these matters at this time of sorry business. She would have to defer to her father Athe Kapua Gutchen, who is a Senior Elder in the Meuram tribe. She proposes to give evidence with her father, who will invite the Elders to be present to ensure an accurate account is given to the Court in accordance with Customary Lore. 

[21] Waratah says that the evidence given by Ms Rose is insufficiently specific. It does not descend to the detail of what protocol applies to what evidence. It argues Ms Gutchen must have received permission to give the written evidence that will be redacted and there is a process for her to get that permission in the future. That may be so, but that argument addresses necessity, not whether a protected right is engaged by the application. 

[22] The witnesses have described the cultural protocols they would be expected to observe in giving evidence of traditional knowledge and culture. Evidence about those matters is central to the objection their evidence relates to. Refusing the witnesses’ request would limit their ability to enjoy and maintain their cultural heritage, specifically about how traditional knowledge is imparted. If they are confined to their written statements they cannot observe those cultural protocols. 

[23] Section 13 of the HRA defines when a limitation of a protected right is lawful.  

Human rights may be limited 

(1) A human right may be subject under law only to reasonable limits that can be demonstrably justified in a free and democratic society based on human dignity, equality and freedom. 

(2) In deciding whether a limit on a human right is reasonable and justifiable as mentioned in subsection (1), the following factors may be relevant – (a) the nature of the human right; (b) the nature of the purpose of the limitation, including whether it is consistent with a free and democratic society based on human dignity, equality and freedom; (c) the relationship between the limitation and its purpose, including whether the limitation helps to achieve the purpose; (d) whether there are any less restrictive and reasonably available ways to achieve the purpose; (e) the importance of the purpose of the limitation; (f) the importance of preserving the human right, taking into account the nature and extent of the limitation on the human right; (g) the balance between the matters mentioned in paragraphs (e) and (f). 

[24] Respectfully, I adopt Martin J’s interpretation of s 13 in Owen D’Arcy v QCS. His Honour found that section was enacted with the intention of embodying a proportionality test. He also followed the reasoning of Warren CJ in Re Application under the Major Crimes (Investigative Powers) Act 2004 in interpreting an equivalent provision in the Victorian Charter of Human Rights:

“[145] A free and democratic society is the fundamental hallmark of our system of governance and way of life. Notions of the ‘public interest’ stem from notions of what is best for a free and democratic society. I find I am assisted by the remarks of Dickson CJ in Oakes: ‘The Court must be guided by the values and principles essential to a free and democratic society which I believe embody, to name but a few, respect for the inherent dignity of the human person, commitment to social justice and equality, accommodation of a wide variety of beliefs, respect for cultural and group identity, and fair in social and political institutions which enhance the participation of individuals and groups in society. The underlying values and principles of a free and democratic society are the genesis of the rights and freedoms guaranteed by the Charter and the ultimate standard against which a limit on a right or freedom must be shown, despite its effect, to be reasonable and demonstrably justified.”

[25] The onus of demonstrably justifying a limitation rests with the party seeking to uphold it. The issue for the Court is balancing the competing interests of society, including the public interest. 

[26] Putting aside the question of necessity, which Waratah accepts is not the test, Waratah says the cost is disproportionate and would not assist the Court because it does not contest the witnesses’ evidence. 

[27] The nature and the purpose of the limitation would be to avoid the commitment of time and resources in taking the evidence on country. This involves some days of hearing time and expense to the parties and the Court. 

[28] Time and expense are relevant considerations. A court should be prudent in making directions about the conduct of proceedings. It has a responsibility to wisely use public resources to discharge its functions fairly and efficiently. It should not impose an unjustified burden on litigants. Declining the proposed orders would help to achieve that purpose. 

[29] Waratah estimates their costs would be in the order of $80,000, which it says is prohibitive. It is possible, as Waratah suggested, for the witnesses to give evidence on country using videoconferencing technology, but that would limit the witnesses’ ability to fully observe the ceremonial aspect of imparting traditional knowledge. 

[30] Waratah did not lead evidence of financial incapacity and it might employ a leaner legal team than routinely appears before me in Brisbane. The cost estimate made by YV and TBA is more modest and draws on their recent experience of travelling to these places to take statements from the witnesses. They have offered to assist Waratah to raise funds, a matter I leave with the parties. Importantly, they offer to take the lead on logistics and have done some advance work in organising the visits. This would reduce the inconvenience and, likely, the costs. 

[31] Turning to whether evidence taken as proposed would assist the Court, Waratah draws a distinction between this case and one involving a native title claim where the Court must make factual findings about cultural matters. Waratah does not contest the factual matters the First Nations witnesses have deposed to. It accepts they have a very strong connection with the sea and the land, and the flora and fauna. It does not dispute there will be sea level rise, that the climate will warm, that the fish may not be as plentiful, or that different aspects of the witnesses’ traditional way of life will change. 

[32] That does not mean there will be no contest about their evidence. 

[33] Evidence is led from these witnesses to establish how physical climate change impacts will affect their cultural rights. If YV and TBA establish the mine would limit the ability of First Nations peoples of Queensland to exercise their cultural rights, I will need to weigh that in the balance with other relevant factors in deciding whether the limit is reasonable and demonstrably justified. That is an evaluative, not a fact-finding process. 

[34] There is another dimension to this that Waratah did not address. 

[35] YV and TBA propose the witnesses give evidence in the presence of the people who have the collective authority to speak about matters of place and culture. Section 28 of the HRA frames these cultural rights in collective terms. It protects the rights of Aboriginal and Torres Strait Islander peoples to do specified things with other members of their community. 

[36] The High Court has long accepted the communal nature of the rights, and recently recognised as a compensable loss a group’s sense of failed responsibility, under the traditional laws and customs, to have cared for and looked after land. 

[37] The witnesses will be giving evidence about the impact climate change will have on their community’s ability to enjoy and maintain their cultural rights. The Court will be assisted in its evaluative function by seeing and hearing this evidence being given in that community. 

[38] Further, as the statutory party observed, this is a specialist Court. In my experience as a lawyer and judicial officer, written evidence from a First Nations witness is a poor substitute for oral evidence given on country and in the company of those with cultural authority. 

[39] Finally, I have been assisted by site inspections, even where factual matters are not in dispute. A site inspection can aid understanding. Importantly for an evaluative hearing, it can provide context and proportionality. 

Conclusion 

[40] To return to whether the limit is demonstrably justified in a free and democratic society, refusing the witnesses’ request to give evidence in this way orders does not respect their cultural and group identity or accommodate their beliefs. 

[41] There is utility in the evidence being given in the way YV and TBA propose. It will not impose an unreasonable and disproportionate burden on the parties or this Court. 

[42] If the orders are not made, the First Nations witnesses will be confined to their written statements. If the orders are made, they will be able to explain that evidence in the presence of those they are speaking for and Elders who can ensure the Court receives an accurate account. 

[43] The orders will ensure I receive the best evidence from the First Nations witnesses. That, and the site inspections, will assist me in discharging my administrative and evaluative function in this hearing. 

[44] In deciding this application, I have balanced the collective right to enjoy and maintain culture against the public and private interests in minimising the inconvenience and cost of litigation. Confining the First Nations witnesses to the written statements is a limit to their right, and that of their community, to maintain their culture about how they transmit traditional knowledge. I am not persuaded that limit is reasonable and demonstrably justifiable in the circumstances of this case. 

[45] The same considerations are relevant in deciding that it is in the interests of justice to make the orders sought. ...