Framing the Wicked Problem of Orphan Drugs

The rare disease field is host to many issues, but people disagree on what the problem is exactly. Such an unclear problem definition, and absence of clear-cut solutions, are characteristics of wicked problems. Schon and Rein, two influential scholars, argued three decades ago that if you want to solve a complex issue, you should look at how people frame it (Schon and Rein, 1994). A frame is a coherent set of facets of a problem, typically including a definition, cause, and possible solutions (Entman, 1993). Charting how people frame a wicked problem differently might help resolve it.

Lenalidomide, brand name Revlimid, is a drug that serves several orphan indications. It is also a blockbuster medicine for specific cancers. Its market exclusivity has recently expired, and, as such, it has gone through the whole drug research and development cycle. Van Lonkhuyzen and Hordijk, two Dutch journalists, researched the lenalidomide case in depth. In their recent article, they describe several issues the drug raised on the way, many of which are by no means exclusive to lenalidomide (van Lonkhuyzen & Hordijk, 2022).

For my BSc thesis in Science & Innovation Management, I set out to discover how Dutch stakeholders framed the problem(s) involved in the lenalidomide case, and orphan drugs generally. I interviewed different stakeholders at the Dutch Ministry of Health, Welfare, and Sports, the Dutch National Health Care Institute, the Dutch Medicines Evaluation Board, the Dutch Healthcare Authority, the Antonie van Leeuwenhoek Hospital in Amsterdam, a Dutch health care insurer, Bristol Myers Squibb, and the Dutch Association of Innovative Medicines.

During these interviews, I encountered two primary frames. The industry frames the problem of orphan drugs as one of data uncertainty caused by low numbers of patients. Industry representatives argue that this high uncertainty makes the development process increasingly risky. They suggest more basic research on these diseases to decrease this uncertainty and further incentivising the development of drugs for rare diseases.

All others I spoke with frame the problem as one of uncertainty, too, but this time in terms of the cost-effectiveness of developed medicines. In this frame, this high uncertainty causes difficulties in evaluating treatments, which is problematic because accepting relatively high prices means risking the out-crowding of other forms of health care. Proponents of this frame blame regulatory privileges such as market exclusivities for enabling companies to ask for such high prices.

In our analysis, the problem framing differs primarily on the issue of profit. The core question is, what price is justified when, and what aspects do we consider when answering this question?

Notably, all interviewees agreed on one potential solution. Both groups suggested that, as a society, we must engage in serious conversations on how to decrease health care costs. Perhaps we could start by discussing what kind of profit margins we find justified under which circumstances.

These findings have been published as Gengler, R. (2022) The Wicked Problem of Orphan Drugs - Lenalidomide as a case study. Utrecht University (BSc thesis)



Entman, R. M. (1993). Framing: Towards clarification of a fractured paradigm. McQuail's reader in mass communication theory, 390, 397.

Rein, M., & Schön, D. (1996). Frame-critical policy analysis and frame-reflective policy practice. Knowledge and policy, 9(1), 85-104.

Van Lonkhuyzen, L. & Hordijk, L. (2022). Now that the patent has expired, this cancer drug is suddenly 99% cheaper. Accessed on 20 September 2022 via

Photo credit: Pine Watt (2017). Person Holding Rectangular White Frame [Image].


Written by: Remke Gengler (BSc) and Tineke Kleinhout-Vliek (PhD)

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