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Review of 'International Drug Repurposing Patent Landscaping, Part 1: Rare Diseases'

Reviewer: Donald Lo

Inviter role(s): EDITOR

Publication date of review: 2024-05-16

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Donald Lo5

International Drug Repurposing Patent Landscaping, Part 1: Rare DiseasesCrossref ScienceOpen

This interesting and important review on DR as viewed through the lens of patent filings

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International Drug Repurposing Patent Landscaping, Part 1: Rare Diseases

Hermann Mucke (2024)

In this first installment of a series of papers on landscaping of drug repurposing patents we present an analysis in the context of rare diseases. Using the RepoScope drug repurposing database, developed as part of the REPO4EU Horizon Europe project, we identified 190 documents that were published through the international PCT patent system from 2010 to 2023. These documents claimed utility in a total of 98 rare diseases. We provide detailed tabulated summaries for two representative disease classes: lysosomal storage diseases and muscular dystrophies. High variability in active pharmaceutical ingredients indicated high levels of innovation. Seventy-five percent of rare disease drug repurposing patents were filed by small/medium pharmaceutical companies or universities, often university spin-offs. Big pharmaceutical companies show limited interest in this area, as reflected in their low representation. Approximately one-third of all patent disclosures came from the United States and the European Union. The EU demonstrated a stronger position in patenting for rare diseases than in general pharmaceutical patenting. Patentees from China, Korea, and Japan, who are notable in the general pharmaceutical field, showed limited interest in rare disease drug repurposing patents, in contrast to their publication record in this area.

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Review information

DOI:: 10.14293/S2199-1006.1.SOR-LIFE.AZBS0J.v1.RBGEMR

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This work has been published open access under Creative Commons Attribution License CC BY 4.0, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Conditions, terms of use and publishing policy can be found at www.scienceopen.com.

ScienceOpen disciplines: Medicine,Life sciences

Keywords: drug repurposing,rare diseases,patenting

Review text

This interesting and important review by Mucke highlights the use of international PCT patent disclosure data to understand and analyse drug repurposing activities across a broad range of filing institution types. The article outlines why this is a different and complementary approach to analysing only peer-reviewed publications, and nicely provides a primer on the patent filing process.

Major comments:

p. 5: The sentence “Rare diseases would mostly not provide sufficient economic incentive for pharmaceutical companies to develop and market drug treatments”, while true, deserves more comment. In fact, there a numerous rare diseases that are quite viable for large pharma companies, e.g. Huntington’s disease which is mentioned elsewhere. Typically, a prevalence of >10-30K patients in Europe or US is deemed viable markets by most companies. Moreover, with premium pricing clearly new drugs are quite profitable even for ultra-rare diseases. This may also be the case for at least some repurposed drugs where there is potential for a commercial monopoly position.

p. 6: “A remarkable 10% came directly from national authorities and institutions…” The US NIH has an annual budget of >2B USD so perhaps not so unexpected?

p. 6: "At 17%, Asian applicants (almost evenly distributed between China, Korea, and Japan) were comparatively underrepresented.” Underrepresented by what measure, perhaps by annual R&D spend? This should be clarified.

p. 9: Similarly, some basis for the conclusion “This is not in line with their peer-reviewed manuscripts in this field” should be provided.

Finally, there should be mention of drug repositioning (i.e., repurposing of clinical stage drug candidates to different indications other than originally targeted) should be discussed. These would still appear at some point in PCT disclosures, though likely difficult to know if as originally positioned or repositioned. Nevertheless, this should be discussed at least as a stream of drug drug repurposing that is challenging to quantify.

In addition, it would be good to have an Appendix Table, say, where the remainder of the 190 PCTs could be listed and annotated as they are in Table 1.

Minor comments:

P. 5: “broader” is misspelled as “briader”

P. 9: “European and American” should be “European and US”

Comments

Hermann Mucke wrote:

Hi David,

Thx for your valuable comments. V2 incorporates them as follows:

# p. 5: The sentence “Rare diseases would mostly not provide sufficient economic incentive for pharmaceutical companies to develop and market drug treatments” -- the following sentence has been added: "Unless premium prices can be charged (which is more difficult for repurposed agents than for new chemical entities), a prevalence of >10-30,000 patients in Europe or US is deemed viable from a business perspective by most companies."

# p. 6: “A remarkable 10% came directly from national authorities and institutions…” The following sentence has been added: "These agencies have large budgets that put few if any limitations on their patenting activities. However, large pharmaceutical companies."

# p. 6: "At 17%, Asian applicants (almost evenly distributed between China, Korea, and Japan) were comparatively underrepresented.” added: ..."in terms of PCT disclosure numbers."

p. 9: “This is not in line with their peer-reviewed manuscripts in this field” has been expanded: "This is not in line with their much larger fraction of peer-reviewed manuscripts in this field.The question why Asian researchers translate a smaller fraction of their repurposing innovation to the international patent space would warrant closer investigation."

Mentioning of drug repositioning: the Methods section now has the sentence, "RepoScope does not differentiate between repurposing of an active pharmaceutical ingredient and drug repositioning, i.e., repurposing of clinical stage drug candidates or approved drugs to indications other than originally targeted), as both types of new uses will appear in PCT documents in the same way."

2024-06-10 19:21 UTC

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Reviewed by Donald Lo