Together for the greater goods: legitimising social innovation in the pharmaceutical field

Figures & data

Table 1. Case descriptions.

Case name	Case description
Case 1: Novel reimbursement route for eculizumab	The breakthrough medicine eculizumab (Soliris®) is currently licenced for two rare diseases. After the responsible health care authority, Zorginstituut Nederland (ZIN), issued negative reimbursement advice for the first indication Paroxysmal Nocturnal Hemoglobinuria (PNH), clinicians at the Radboud University Nijmegen drafted a research protocol for the second indication, Atypical Hemolytic Uremic Syndrome (aHUS), which aimed to establish whether a ‘restrictive treatment regimen’ would be feasible (giving patients less medicine, resulting in lower spending). This resulted in ZIN issuing positive reimbursement advice for aHUS patients, as long as the eculizumab was provided under the CUREiHUS research protocol. Afterwards, the protocol was repeatedly called exemplary: it was the ‘first ever’ orphan drug arrangement and granted the first prestigious Dutch ‘Pearl’ prize for projects with particularly relevant results, excellent collaboration, or extra care for diversity, patients, or innovation.
Case 2: Hospital-based manufacturing of CAR T cell therapy	Chimeric Antigen Receptor (CAR) T cell therapy is a personalised treatment for a rare, aggressive cancer, whereby immune cells naturally present in the body are ‘harvested’, modified, and given to the patient, where these modified cells recognise and attack the cancer cells. In May 2020, the Dutch Minister for Health admitted Yescarta® or axi-cel, a CAR T cell therapy, to the benefits package after successful price negotiations. Researchers at the University Medical Center Groningen (UMCG), University of Groningen, sought funding to investigate the comparison of ‘in house’ generated CAR T cells with CAR T cells produced by the pharmaceutical company. They received a 30 million euro ‘Potentially Promising Care’ subsidy, and the six-year project started on 1 December 2020.

Table 2. Interviews.

Interviewees	Case 1: Eculizumab	Case 2: CAR T cell therapy
Clinicians	n = 2	n = 1
Patient representatives	n = 3	n = 2
Other patient representatives	n = 1 across both cases
ZIN employees	n = 2	Contacted three interviewees, all declined to be interviewed (citing time constraints, referring to the ‘Potentially Promising Care’ subsidy website instead)
Other policy makers	n = 2 across both cases
Pharmaceutical company employees	Contacted three interviewees, all declined to be interviewed (not commenting on individual cases)	n = 3
Pharmaceutical representatives	n = 2 across both cases

Table

Case 1: Eculizumab	Title	Source, year
Policy documents	Pearl project – aHUS: treatment with eculizumab can be much more targeted	ZonMw (2018)
	Optimalization of Eculizumab/Soliris® treatment in patients with atypical hemolytic uremic syndrome by means of individually-tailored, personalised therapy	ZonMw (2021)
	Report Meeting Appraisal Committee (18 March 2016)	Zorginstituut Nederland (2016a)
	Report Meeting Appraisal Committee (14 October 2016)	Zorginstituut Nederland (2016b)
	Package recommendation eculizumab (Soliris®) in treatment of aHUS patients	Zorginstituut Nederland (2016c)
	Monitor Orphan Drugs in Practice 2018	Zorginstituut Nederland (2018a)
	Monitor Orphan Drugs in Practice 2019	Zorginstituut Nederland (2019)
	Monitor Orphan Drugs in Practice 2020	Zorginstituut Nederland (2020a)
News articles	Saving lives sometimes too expensive, Thijs Rösken, De Telegraaf (19 March 2016)	Rösken (2016)
	Dosage of expensive drug may be lower than manufacturer says, Karel Berkhout, NRC Handelsblad (24 June 2017)	Berkhout (2017a)
	Swallow or choke? There is another way, Karel Berkhout, NRC Handelsblad (24 June 2017)	Berkhout (2017b)
	Lower dose of expensive drug also works, Ellen de Visser, de Volkskrant (7 March 2019)	De Visser (2019)
	Kidney patient need not take drug for life, Lineke Nieber, NRC Handelsblad (31 October 2022)	Nieber (2022)
	Tailored usage advice for expensive drugs – ZonMw, Pearls for rare disease research	ZonMw (2018)
	Monitor orphan drugs: spending up 45% in 5 years	Zorginstituut Nederland (2018b)
Other websites	CUREiHUS protocol	Radboud Universiteit Nijmegen (no date)
	aHUS Conference: The Dutch Approach	aHUS alliance (2018)
Policy documents	Promising care – Care site-produced CD19 CAR T-cell therapy in patients with relapsed or refractory DLBCL	Zorginstituut Nederland (2020b)
	Decision request policy on unregistered medicines (19 November 2021)	Rijksoverheid (2021)
News articles	Groningen boosts cancer drug, Ellen de Visser, de Volkskrant (24 September 2020)	De Visser, 2020
	€ 30 million grant for UMCG research into promising cancer treatment	Zorginstituut Nederland (2020c)
	Zorginstituut Magazine: At the desk and at the bedside	Zorginstituut Nederland (2020d)

Documents analysed for the two case studies.

Data availability statement

Anonymised interview data will be made available upon request to protect the identity of the interviewees. All analysed documents are available online.