Having justified an individual case study design focused on a unit of analysis that is process-based and relational in nature, we can now articulate the reasons for the selection of the
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specific case. Stake (1998:236) points to this as the hallmark of any case study research design: “As a form of research, case study is defined by interest in individual cases, not by methods of inquiry used”. The interest in this individual case was based on three pieces of rationale, which I draw from different methodological and sociological scholars. First, the case fulfilled key operational criteria of the phenomenon in question (Yin 1994:91). Second, the case “looms large” over debates in pharmaceutical pricing and innovation (King and Sznajder 2006:767). Third, the case presented an “opportunity to learn” with uncommon access to data and social networks involved in the innovation process (Stake 2005:451). Taken together, these reasons align with my aim of
explaining outcomes in a single case rather than testing theories in a set of ‘representative’ cases. Sofosbuvir-based treatments posed two key operational criteria of the larger phenomenon under question: (1) sofosbuvir represented a true clinical breakthrough with public health
implications and (2) their prices became the subject of intense social and political contestation. Sofosbuvir-based treatments created a drastic shift in treatment possibilities for hepatitis C patients: while prior regimens required six to twelve months of treatment and offered only 50% response rates with high rates of side effects and toxicities, sofosbuvir-based regimens have offered nearly 100% cure rates with few side effects after only three months of treatment (Hoofnagle and Sherker 2014; Ward and Mermin 2015). This clinical advance required scientific and technological advances over a significant period of time, meaning an innovation process existed which could be investigated. This trait is unlike many medicines that have prices deemed to be high but are the result of gaming the intellectual property system and not new scientific and technological labor, such as the prominent case of insulin in which prices continue to rise even though insulin’s discovery was decades ago (Greene and Riggs 2015).
The prices of sofosbuvir-based treatments also ignited a wide-ranging social struggle over treatment access and valuation. Unlike other prominent cases, Gilead’s pricing could not be explained away by maneuvers of a single business unit in acquiring previously approved or off- patented medicines (Armour and Rockoff 2016).44 Nor could criminality be used as a central explanation. As a staff member of a US Senator shared with me, “you won’t find any thing orange jump-suit worthy here”, suspecting that I had been looking for incriminating evidence when
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Two cases of price gouging which drew public attention and indignation in 2015 and 2016, Turing
Pharmaceuticals and Valeant Pharmaceuticals, have been subject to US Congressional scrutiny, with senior leadership in both companies coming under scrutiny. See “Valeant, Turing Boosted Drug Prices to Fuel Preset Profits” in Wall Street Journal on February 2, 2016 (Armour and Rockoff 2016).
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examining the data generated by the US Senate finance committee investigation into Gilead.45 Rather, what I found most interesting was the absence of an overt criminal explanation and rather the ostensible presence of a multi-layered and contested social process requiring the tools of sociological and political economy analysis.
The second reason for selecting this case, beyond fulling my two core ‘operational criteria’ - the pricing over sofosbuvir-based treatments ‘looms large’, meaning novel research findings have the potential of making important contributions to the debate over hepatitis C and potentially the broader discussions over pharmaceutical pricing and innovation. The medicines reflected not only a clinical marvel, but also the most profitable drug launch in pharmaceutical history. They became the subject of political, policy, and academic debates. In the arena of politics, they were raised by candidate Clinton on the campaign trail (DuVall 2016) and galvanized a US Senate investigation (Loftus 2015). In space of policy discussions, health agencies from around the world, policy institutes, and think tanks have wondered about how to interpret and respond to the challenge of hepatitis C medicines and drug affordability (Chahal et al. 2015; Reinhardt 2015; J. Walker 2015; Ward and Mermin 2015). At the sole forum on drug pricing organized by the Obama administration, sofosbuvir was cited as a paradigmatic of the drug affordability problems
challenging many health systems (Pear 2015; US Health and Human Services 2015) Scholarly analysis of the pharmaceutical sector, ranging from medicine to industrial and health economics, has weighed in on the issues raised by sofosbuvir as well (Brennan and Shrank 2014; Chahal et al. 2016; Kesselheim et al. i 2016; Leidner et al. 2015). Yet these analyzes typically have obscured the innovation process behind sofosbuvir, thereby only providing partial explanations. A more complete explanation would be a boon for this disputed domain: given the large numbers of hepatitis C patients, but also the potential for breakthroughs in other disease areas with sizeable patient populations such as Alzheimer’s and certain cancers, the hepatitis C and sofosbuvir innovation process holds potential for pivotal lessons that will be closely examined by multiple stakeholders.
Third, the case of sofosbuvir-based treatments offers what Stake (2005:451) frames as an “opportunity to learn”, which in this case comes in two forms: its unique features as well as the practical access to data. The case of sofosbuvir may be considered to be an outlier by some
analysts, based on the large numbers of patients with an infectious disease in hepatitis C as well as
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the curative and time-bounded nature of sofosbuvir-based treatments (Flier 2017). Indeed, many medicines with high prices are for diseases with smaller populations, and many of these
medicines require long-term or even life-long treatment (Kesselheim et al. 2016; Montazerhodjat, Weinstock, and Lo 2016). However, as Stake (2005:451) has noted, “some times it is better to learn a lot from an atypical case than little from a seemingly typical case”. To heed Stake’s advice, I take inspiration from Mauss (1985:10), who noted that such cases possess "an excessiveness which allows us better to perceive the facts than in those places where, although no less essential, they still remain small-scale and involuted." Indeed, the tensions that sofosbuvir’s pricing created for health systems - and the debates this instigated - were an important reason for why a large array of evidence became available for analysis over time. For example, the appendices in the US Senate investigation provided approximately 1,500 pages of internal corporate documents with detailed information on the valuation of sofosbuvir by Pharmasset, Gilead’s acquisition of Pharmasset, and their subsequent pricing of their treatment regimens. Interviews would not have yielded such detailed internal information about business strategies. Given the timeliness of the drug’s
approval with the start of my PhD, I was also able to follow a slew of media accounts, new journal articles, and in-person events through which I could identify potential interviewees and gather more data. The case thus offered rare opportunities to gain access to sources of data pivotal towards answering research questions on drug pricing and innovation.
In sum, the innovation process behind sofosbuvir-based treatments possessed three compelling reasons to motivate my investigation: key operational criteria as a high and contested price for a breakthrough drug, a case which looms large over debates on the issues I am interested in, and finally an opportunity to learn with uncommon availability of data. Armed with an
intriguing case, I set out to collect data that could answer the questions posed in this dissertation.