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Long abstract:

This paper focuses on drug discovery initiatives hosted by organisations outside of the commercial pharmaceutical sector. We draws on 18 interviews with staff from eight universities and research institutes, hosting the drug discovery activities at different scales (from small teams to whole dedicated institutes) where support mechanisms have facilitated the establishment of these dedicated groups. These groups have been selected for study as a representative sample of a wider population, comprised of around 30 groups across the UK. Qualitative thematic analysis has been used to identify institutional support mechanisms available to these groups, as well as the types of activities undertaken in non-commercial settings.

Over the last 20 years, many drug discovery groups have sprung up outside of the commercial sector in the UK. Most have grown and persisted by accessing state or NGO funding to undertake novel early-stage drug discovery that is generally considered too high-risk for industry to undertake. Despite the opportunities afforded by working outside of the industry the studied organisations relied very much on former industry staff, who they employed.

Being a part of academic research organisations, those engaged in drug discovery are generally afforded very few special conditions for their activities, other than start-up funding (e.g. to set up labs). Staff may also be diverted from drug discovery by traditional academic duties such as teaching. Where groups have enjoyed some autonomy, these have tended to organise themselves more like small firms. Most seem focused on delivering projects that meet the needs of the established industry.

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Short abstract:

Drawing from empirical data collected during fieldwork on oncology trials with immunotherapy, in this paper I will discuss the conflicting moral dilemmas around Advanced Therapy Medicinal Products and present future lines of research on this topic.

Long abstract:

One of the major transformations of the pharmaceutical industry in recent years has been a shift towards personalized living drugs, also called biologics, or biopharmaceuticals, which are medicines based on biological substances and/or biological process. At the front of this paradigm shift stand Advanced Therapy Medicinal Products (ATMPs) -medicines based on cells, genes and tissue-, being Chimeric Antigen Receptor (CAR) T-cell therapy one the first and most frequent treatment to be adopted by healthcare systems worldwide.

Hailed as a breakthrough in cancer research and treatment, CAR T-cell therapy aims to enhance the potential of the immune system to cure cancer by genetically engineering the patient´s T-cells to target the specific antigen of the patient´s cancer cells. In contrast to conventional oncology treatment such as chemotherapy or radiotherapy this therapy is portrayed to be more efficient and less harmful to the patient. However, its implementation engenders challenges and changes in scientific and biomedical practices, knowledge and skills, which in turn transforms the lived experience of patients and their caregivers. Beyond the organisational and epistemic changes, in this paper I will focus on the new moral challenges that face scientists, healthcare professionals, patients and their caregivers when producing, delivering or receiving CAR T-cell therapy. I will present several ethnographic vignettes collected during fieldwork on oncology trials with this treatment to illustrate the conflicting moral dilemmas that arise around CAR T-cell therapy. Then, I will present current work in progress that aims to develop future lines of research on the topic.

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Historically, pharmacy preparation (compounding) has been used to prepare drugs, dosages, or formulations that industry cannot provide. However, recently, it has been proposed to use compounding, instead of marketing authorization, as an alternative medicine-to-patient route for newly developed personalized medicines for orphan indications. The barriers for bringing orphan drugs to the market are often high, especially when these drugs are developed in hospital or academia. This results in decreased patient access. Additionally, the current regulatory system provides barriers for personalized medicines, which need to be produced locally and adapted to individual patients. Pharmacy compounding could provide a solution.

Currently, a device (Nanospresso) is being developed with the intention of enabling the local bedside production of personalized nucleic acid therapies, potentially achieved via compounding. The development of the Nanospresso device gives rise to several questions regarding the boundaries of pharmacy compounding as an alternative route towards patient access. This paper explores the feasibility and desirability of the local pharmacy preparation of personalized complex pharmaceuticals.

We aim to provide an overview of the advantages and disadvantages of using this alternative access pathway, focusing on technical, practical, legal, ethical, regulatory, social and monetary aspects. To create this overview, we will analyze scientific literature, newspaper articles, and government documentation about recent Dutch compounding initiatives. In addition, we will conduct interviews with stakeholders in the Dutch compounding debate where we present people with different scenarios of a compounding device for the bedside production of individualized medicines (Nanospresso). At EASST we will present our preliminary results.

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Technological advancements in understanding and modifying the human genome have enabled the emergence of the gene and cell therapies (GCTs) field. GCTs hold the potential to treat rare genetic diseases, cancers and infections, many of which were previously hardly treatable or untreatable. Fairly early on in the emergence of the field, gene therapies started to be regulated as pharmaceutical products. Consequently, gene therapies have been developed within the traditional linear regulatory and access frameworks based on traditional business models in the sector. However, these traditional models are failing to deliver the quantity of therapies needed for rare genetic diseases, as well as access to the ones that are developed. Initiatives have emerged in the GCT field which aim to challenge traditional regulatory frameworks, access conditions and business models. Examples include patent opposition filed by NGOs against the patent of gene therapy Kymriah or academic hospitals using the hospital exemption in direct competition with a licensed product. These initiatives often involve public actors (academia, hospitals, civil society) and reflect public values (not-for-profit, transparency, equity). They create frictions with dominant regulatory and access frameworks that reflect the values of the traditional pharmaceutical model (for-profit, IP protection, economies of scale). In this paper we study how we can understand the transformative nature of these initiatives and associated value orientations of actors. We identify initiatives based on a qualitative event history analysis of the GCT field and specifically examine how these initiatives can be transformative.

co-author: Lourens Bloem (Utrecht Institute for Pharmaceutical Sciences)

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Drug repurposing refers to the investigation of existing pharmaceuticals for new therapeutic purposes. While drug repurposing is acknowledged as a strategy for therapeutic innovation, particularly in rare and neglected diseases, the practical application of this strategy raises a number of challenges. In this paper, we a) describe the current drug repurposing ecosystem in Europe, b) identify main systemic barriers in moving repurposed drugs from the clinic to the market in a sustainable fashion, and c) discuss how the approval, launch and reimbursement of repurposed pharmaceuticals might be better supported. Building on analysis of policy reports and stakeholder interviews, we identify barriers relating to the cost of late-stage clinical development, liability at market authorisation, lacking knowledge about regulatory processes among potential sponsors, and issues of reimbursement. Further, there is no proven business model to ensure that innovation centred on drug repurposing will be economically viable or commercially attractive without ongoing support from research funders and philanthropy. Drawing on work in innovation studies and the idea of a sociotechnical niche, we propose that a more sustainable alternative could be to create a circular drug repurposing economy with profits reinvested in R&D. Further, we propose that intermediary institutions (or market translators) can play an important role by mimicking the activities of large, integrated pharmaceutical companies in project finance, clinical trials design, portfolio management, and regulatory affairs. Such intermediary institutions could take different organisational forms (public, private, hybrid) and create new models for commercial or non-profit drug development.

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Brazil’s Partnership for Productive Development (PDP) policy has facilitated the local production of affordable generic drugs to meet national demands. This article explores the PDP’s role in transferring biosimilars revealing the intricate challenges in establishing local production in Brazil.

Long abstract:

The policies of Partnership for Productive Development (PDP) have permitted the rapid replication of antiretroviral drugs within both private and public Brazilian pharmaceutical laboratories, ensuring access within the national health system. This was achieved through ‘innovation through copy’ – i.e. reverse-engineering of original products, aimed at circumventing patents and enhancing production processes (Cassier and Correa 2003). However, the PDPs aimed at transferring the capacity for biosimilar production – copies of biopharmaceuticals derived from cell cultures – have failed to replicate the success of chemical generics. Only a few manufacturing capacities have been established, still reliant on importing key components. Additionally, there is a minimal price difference between the originator and the biosimilar. Our on-going fieldwork on the local production of biopharmaceuticals in Brazil sheds light on the challenges faced by the local industry in producing biosimilars and highlight how those differ from the generic sector. Specifically, we highlight the near absence of innovation-through-copy in biosimilar production. First, PDP policies governing biopharmaceuticals have led to a sluggish transfer process, including a mandated 10-years delay for foreign companies to transfer technology while building a monopoly on the Brazilian public market. Second, compliance with biosimilar regulations necessitates more stringent comparability studies compared to generics. Lastly, Brazilian public laboratories prioritize vaccine production, diverting attention from biosimilar development. Overall, we argue that biosimilars produced in Brazil rely on foreign key components, processes, and knowledge, prompting critical reflexion on the way a ‘copy’ is made and regulated in specific places (Greene 2014; Hayden 2007; Pollock 2019).

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This paper investigates novel forms of antibiotic innovation that aim to overcome the biomedical and commercial sustainability constraints posed by antimicrobial resistance (AMR). Modern antibiotics have underpinned public health and medicine for decades. However, the rapid spread of AMR progressively diminishes the efficacy of existing antibiotics. The search for novel antimicrobial treatments has become a top policy priority but is confronted with intricate scientific and commercial challenges. The growing threat of AMR, where bacteria rapidly develop resistance against drugs used to treat infections, has cast doubts on the effectiveness and sustainability of the prevailing 'biomedical arms race' model of public health. This model involves developing ever-more 'super-drugs' effective against an ever-growing population of multi-drug-resistant 'superbugs.'

This paper scrutinizes efforts and approaches within the leading global AMR-related product development partnerships that explicitly strive to break with conventional models of antibiotic research, development, and commercialization. Their aspiration is to radically rebuild pharmaceutical innovation systems in more sustainable ways.

Integrating new materialist approaches to the study of microbes and AMR with political-economic approaches to the study of pharmaceuticals, the paper analyzes an emergent approach to post-antibiotic innovation here tentatively termed 'eu-biotic.' This eu-biotic innovation shifts the focus in at least two significant ways: on the one hand, it seeks to reduce antibiotic use by decoupling investment incentives from economies of scale; on the other, it shifts the underlying clinical model from its default aim of 'combatting bugs' towards the aim of modulating virulence and decreasing pathogenicity.

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Despite concerns about rare disease patient organisations' involvement biasing evaluation processes about market access and pricing of orphan medicinal products (OMPs), their involvement can work as a catalyst for optimising the use of OMPs in clinical practice. This paper investigates the reimbursement evaluation processes for eculizumab for the treatment of atypical Haemolytic Uraemic Syndrome in England, Scotland, and the Netherlands. Using the analytical lens of valuation studies, the paper asks: how did patient organisations frame the issues at stake in these processes, how did their framing come to reshape the approach taken in these specific cases, and what are potential implications for future reimbursement evaluation processes? The paper shows how, in the face of highly unfavourable cost-effectiveness and looming non-reimbursement, patient organisations together with clinicians mobilised the potential for dose tapering and treatment withdrawal in patients treated with eculizumab, leading to significant cost reductions, better cost-effectiveness, and greater patient safety and comfort. Analysing the different ways in which patient organisations were able to shape the issues at stake in the evaluation processes in the countries studied, the paper argues these cases can be exemplary in moving away from a heavy reliance on industry evidence submissions in reimbursement decision-making. Similar studies are now emerging in the treatment of patients with Paroxysmal Nocturnal Haemoglobinuria, showing their potential value in reframing reimbursement questions. Finally, the paper reflects on patient organisations' differing abilities and opportunities to shape issues at stake in reimbursement processes, and obstacles to the types of 'real-world' research studied here.

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Social pharmaceutical innovation (or SPIN) refers to modes of innovation that advance novel forms of collaboration with a wide range of actors, which breaks with conventional pharmaceutical innovation practices in an institutional way to produce more treatments that are more affordable and more equally accessed. Over the course of four years an international consortium of STS researchers from Brazil, Canada, France and the Netherlands have been examining cases of SPIN: what are they, how are they working, what challenges are the coming up against? This paper provides a brief report on that work. We show how SPINs are re-organizing partnerships in innovation processes and describe key features of how innovation occurs in this space. We also describe how SPINs are changing the way data is gathered and how evidence is constructed, as well as providing new routes for patients to access treatments in more sustainable ways. We conclude with some recommendations for supporting SPIN as an alternative to the dominant model of pharmaceutical innovation, and some STS insights in the boundaries that are blurred in the innovation process by way of SPIN.

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During the Covid-19 pandemic the Africa CDC gained prominence as a technical agency committed to articulating and protecting the public health needs of the continent worst affected by the globally inequitable distribution evocatively decried as “vaccine apartheid”. Informed by this experience, it is now creating technical, regulatory and financing infrastructures to support sustainable R&D into, and local production of, pharmaceuticals and other health products and technologies that prioritise continental public health priorities – during and outside of health emergencies. This paper explores the transformative potential of these efforts in relation to three questions. First, what geopolitical imaginaries and conceptions of sovereignty, international cooperation and solidarity are being mobilised by Africa CDC in promoting investment in local innovation and manufacturing in the context of the “New Public Health Order” it is calling for? Second, how do these imaginaries articulate the role of African R&D and pharmaceuticals in promoting wellbeing on the continent in ways that nonmedical technologies cannot? Third, how do histories of exploitation, medical extractivism, and the conjoined experiences of subjugation/tutelage and abandonment at the hands of the “international community” during past public health emergencies and epidemics shape the organisation’s work in promoting R&D and local manufacturing? Drawing on key informant interviews and publications (academic, grey literature and policy and legislative documents), this paper seeks to develop some preliminary answers to these questions in order to map Africa CDC’s articulation of its potential in transforming the regional and global pharmaceutical landscape.

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Long abstract:

We consider the question of whether the ‘global health era’ starting around the turn of the century, in effect, led to a harmonisation of pharmaceutical policies across the globe. Beginning in the early 2000s, we will examine several examples, such as the WHO essential medicines list, clinical trial and patenting legislation, and health technology assessment, to consider the interplay between different institutional layers at the supra-national level. We then move on to the crucial period of 2019-2022 and the system shock of the COVID-19 pandemic, which has led to global controversy around stark vaccine inequalities, and significant but as-yet unfulfilled institutional efforts towards intellectual property waivers, patent pools, and knowledge transfers. The COVID-19 period has arguably shown the failure of the global harmonisation era to the benefit of regional coordination efforts. Whilst the Pandemic Accord negotiations and a redrafting of the International Health Regulations (IHR) continue to aim for global harmonisation, this regionalisation trend has continued in the European HERA initiative and the ‘local’ production of vaccines. We also briefly consider the likely implications of heightened scholarly, policy, and advocacy interest in intersectionality and current high-tech medical advances (gene therapies, etc).

Concluding, we analyse a dual movement over time. On the one hand, the move towards supra-national harmonisation on specific policy topics has varying causes and vastly differing effects. On the other hand, we see increased awareness of and efforts to actively combat inequities deriving from these differentiated harmonisations in a contextually appropriate manner.