1. Patenting in life sciences: general aspects

Key points
  • There are three patentability criteria (novelty, inventive step or non-obviousness, and industrial applicability or usefulness) and two main supporting requirements (sufficiency of disclosure or enablement, and eligibility of subject matter).

  • The patentability criteria and supporting requirements are globally accepted, but with nuanced country-specific differences.

  • It is important to understand how other types of intangible assets such as trade secrets or know-how can help in the protection of life sciences innovations.

  • Patent attorneys rated the inventive step or non-obviousness, illustrated with a diverse set of examples, as the most important elements for post-grant patent strength.

Assessing the particularities of patents within the field of life sciences is important as we all want to have innovations that lead to better healthcare systems and healthier lives. Nevertheless, before doing so we need to understand what a patent is. Patent law provides protection for inventions.

In theory, the purpose of the patent system is to encourage the disclosure of innovation for the progress of our society. An inventor discloses publicly his or her invention and, in return, is rewarded with a period of exclusivity, usually 20 years from the date of filing, during which others are excluded from performing certain specific acts with regard to the invention (as this is defined in the granted claims of the patent). Said acts include making, using, offering for sale, selling or importing the invention without authorization. In other words, it allows the owner to prevent other parties from commercially exploiting the invention. The right conferred by the patent is an exclusionary right; it allows the owner to exclude others from using the invention.

In practice, however, no system is perfect and there is always a debate between the advantages and disadvantages of the patent system, especially for the biomedical sector. (1)Gubby, H. (2020) Is the patent system a barrier to inclusive prosperity? The Biomedical Perspective. Global Policy, 11(1), 46–55. doi:10.1111/1758-5899.12730. The patent does not enable or entitle the owner to practice the invention. For commercial use of most innovations in the healthcare field, regulatory approvals are required. In addition, it is ensured that the use of the invention does not infringe third-party patents. This type of conclusion is only possible in the case of a legally binding decision of non-infringement. In practice, this risk is managed by way of legally informed opinion. This typically involves a comprehensive review of all relevant third-party patents, in combination with written confirmation from a suitably qualified lawyer that any of the acts contemplated (i.e., to practice the invention) would not, if so performed, constitute patent infringement of at least one patent claim issued to one of the third-party patents. This associated legal process is often referred to as “freedom-to-operate” or “patent clearance”. (2)Gubby, H. (2020) Is the patent system a barrier to inclusive prosperity? The Biomedical Perspective. Global Policy, 11(1), 46–55. doi:10.1111/1758-5899.12730.

To obtain a patent for an invention in any technical field, a few essential requirements must be met.

1.1 Patent-eligible subject matter

As with other fields, domains or systems around the world, the patenting system constantly evolves; what can and cannot be patented also changes. What is eligible for patenting also differs between the countries or regions in which an application is filed.

In this report we only cover the most important and relevant patent-eligibility topics for the field of life sciences within Japan, the United States of America and Europe. WIPO published a thorough report on biotechnology patentable subject matter from a global perspective, which provides a thorough analysis of country- and region-specific patent systems. (3)D. Borges Barbosa and K. Grau-Kuntz (2010). 3. Exclusions from patent-eligible subject matter and exceptions and limitations to the rights biotechnology. World Intellectual Property Organization. Available at: https://www.wipo.int/edocs/mdocs/scp/en/scp_15/scp_15_3-annex3.pdf (accessed May 24, 2025).

Japan

The legal requirements for the patent system in Japan are described by the Japan Patent Act (Act 121 of 1959). (4)Japanese Patent Act; 1959. Available at: https://www.japaneselawtranslation.go.jp/en/laws/view/4097 (accessed May 24, 2025). The Japan Patent Office (JPO) has also published guidelines on their website specifically for life sciences, which explain the reasoning of examiners and provide examples for the different scientific areas of life sciences. (5)Examination guidelines in life sciences. Japan Patent Office; 2018. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/lifescience_kijun.html (accessed May 24, 2025). The patentability requirements and the excluded subject matter are described in Articles 29 and 30 of the Patent Act.

In Japan, “a patentable invention is defined as a highly advanced creation of technical ideas utilizing a law of nature.” (6)Examination guidelines in life sciences. Japan Patent Office; 2018. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/lifescience_kijun.html (accessed May 24, 2025). This means that mental models that do not use a law of nature are excluded from patentability. Furthermore, the discovery of an organism or a natural phenomenon is also excluded because it is not created. Additionally, methods of surgery therapy or diagnosis of humans (e.g., methods for administering medicines to humans, methods of surgery on humans, methods for introducing genes to humans using vectors, etc.) are also excluded from patentability, as they are not considered industrially applicable inventions. In contrast, patent-eligible subject matter includes medicines, including combined medicines, vectors, medical materials, medical devices, vectors, methods for manufacturing medicines or medical materials, methods for implanting medical material to mice and methods for collecting information from a human body. (7)Examination guidelines in life sciences. Japan Patent Office; 2018. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/lifescience_kijun.html (accessed May 24, 2025).

United States of America

The legal requirements for the patent system in the United States of America are described by title 35 of the United States Code (USC), and patent matters are dealt with by the United States Patent and Trademark Office (USPTO). (8)Title 35–Patents. United States House of Representatives, United States Code (USC); 1952. Available at: http://uscode.house.gov/view.xhtml?path=/prelim@title35&edition=prelim (accessed May 24, 2025).

The patentable subject matter is described by the 35 USC – section 101. The USPTO website contains guidance on how patent examiners should evaluate claims for patent subject matter eligibility. (9)Subject matter eligibility. United States Patent and Trademark Office; 2020. Available at: https://www.uspto.gov/patents/laws/examination-policy/subject-matter-eligibility (accessed May 24, 2025). The website also includes illustrative examples that are updated regularly and can be used as best-practice guidelines. (10)Subject matter eligibility: life sciences and data processing examples. United States Patent and Trademark Office; 2019. Available at: https://www.uspto.gov/sites/default/files/documents/peg_oct_2019_app1.pdf (accessed May 24, 2025). USPTO has recently published a report with regard to public views on US patent-eligible subject matter, in which stakeholders in life sciences technologies state that the current law is unclear and unpredictable. (11)Vidal, K.K. (2022) Patent eligible subject matter: public views on the current jurisprudence in the United States. United States Patent and Trademark Office; 2022. Available at: https://www.uspto.gov/sites/default/files/documents/USPTO-SubjectMatterEligibility-PublicViews.pdf (accessed May 24, 2025).

Typical exclusions from patent eligibility are related to “laws of nature, natural phenomena and abstract ideas”, which means scientific principles, naturally occurring phenomena, mental processes and mathematical algorithms. (12)35 USC § 101: Statutory Requirements and Four Categories of Invention. United States Patent and Trademark Office; 2015. Available at: https://www.uspto.gov/sites/default/files/101_step1_refresher.pdf (accessed May 24, 2025). Additional considerations are also applied by examiners when deciding whether the invention is eligible, and it is also determined whether the invention is among the judicial exceptions (case law). The most important decisions for life sciences are Myriad: (13)Assoc. for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013). Justia U.S. Supreme Court; 2013. Available at: https://supreme.justia.com/cases/federal/us/569/576/ (accessed May 24, 2025). Assoc. for Molecular Pathology v. Myriad Genetics, Inc. (2013) and Mayo: (14)Mayo Collaborative Services v. Prometheus Laboratories, Inc. 566 U.S. 66 (2012). Justia US Supreme Court; 2012. Available at: https://supreme.justia.com/cases/federal/us/566/66/ (accessed May 24, 2025). Mayo Collaborative Services v. Prometheus Labs Inc. (2012). The common focus of these decisions is to prevent the granting of a patent claim that is defined in terms of a naturally occurring phenomenon, a mental act or an algorithm. Although the underlying principle is acceptable, the magnitude and speed at which the effect of these decisions was distributed through the US legal system meant that tens of thousands of already-issued US patents (mostly relating to clinical diagnostics) became unenforceable overnight.

Despite the decision, equivalent complementary DNA sequences are still considered patentable with the USPTO.

Europe

The body of law for the patent system in Europe is described by the legal texts published by EPO. (15)Legal texts. European Patent Office. Available at: https://www.epo.org/law-practice/legal-texts.html (accessed May 24, 2025). To better understand what comprises patentable subject matter, the texts of EPC and the guidelines for examination according to EPO are the most relevant. The patentability requirements and the excluded subject matter are described by articles 52 and 53 of EPC (16)European Patent Convention - Patentability articles. https://www.epo.org/en/legal/epc/2020/a52.html (accessed July 29, 2025). and part G of the examination guidelines. (17)Patentability requirements. European Patent Office 2025. Available at: https://www.epo.org/en/legal/guidelines-epc/2025/g_i_1.html (accessed July 29, 2025).

Directive 98/44/ec of the European Parliament and the European Council (also known as the Biotechnology Directive) (18)Directive 98/44/EC of the European Parliament and of the Council. Official Journal of the European Communities; 1998. Available at: https://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=CELEX:31998L0044:EN:HTML (accessed May 24, 2025). covers aspects of the patentability requirements for the European Union. These requirements have been incorporated into part G of the examination guidelines from EPC article 52. (19)Patentability requirements. European Patent Office; 2025. Available at: https://www.epo.org/en/legal/guidelines-epc/2025/g_i_1.html (accessed July 29, 2025).

EPO excludes inventions that are contrary to ordre public or morality when exploited commercially (e.g., processes for human cloning, use of human embryos for industrial or commercial purpose, etc.). Scientific discoveries are also excluded from patentability because an invention has to have a technical effect. This EPO exclusion effectively maps on to the USPTO 101 ineligibility provisions, but only to the extent implemented in pre-Myriad or pre-Mayo form. Moreover, EPO does not allow patents for treatment methods “of the human or animal body by surgery or therapy and diagnostic methods practiced on the human or animal body”. (20)European Patent Convention - Patentability articles. https://www.epo.org/en/legal/epc/2020/a52.html (accessed July 29, 2025). This is really a form rather than substance distinction, and does not mean that “methods of [medical treatment]” are not patentable in Europe. In this regard, the preferred EPO format is “Compound X for use in a method of [medical treatment] ….”

1.2 Novelty

Key points

  • Never disclose your invention publicly before filing, as grace periods are limited geographically and in time.

  • Understand the existing prior art, as it will help you to position your novelty and claim strategy.

The novelty aspect requires an invention to never have been previously disclosed in public in any form (verbally, digitally, written, graphically, etc.), place or language around the world. This means that describing your invention to anyone without having a non-disclosure agreement (or a confidentiality disclosure agreement) is considered a public disclosure. (21)Krauß, J. and D. Kuttenkeuler (2021). When to file for a patent? The scientist’s perspective. Nature Biotechnology, 60, 124–9. doi:10.1016/j.nbt.2020.10.006.

The doctrine of inherency or implicit disclosure (USPTO (22)2112 requirements of rejection based on inherency; burden of proof. United States Patent and Trademark Office; 2024. Available at: https://www.uspto.gov/web/offices/pac/mpep/s2112.html (accessed May 24, 2025). or EP (23) Implicit disclosure and parameters. European Patent Office; 2023. Available at: https://new.epo.org/en/legal/guidelines-epc/2023/g_vi_6.html (accessed May 24, 2025).) adds complexity to the analysis of novelty, as patent offices may raise an objection of novelty if the examiner thinks that a prior art inherently or implicitly discloses the invention. Pharmaceutical and biotechnological inventions based on new proprieties of, or new methods of using, existing compositions become difficult to patent. Different case law with regard to the doctrine of inherency is described in detail by Holman (24)Holman, C.M. (2020) Inherency in the patenting of biotechnology and pharmaceutical innovation. Biotechnology Law Report, 39(2), 79–99. doi:10.1089/BLR.2020.29165.CMH and Goldman et al. (25)Goldman, M., G. Evans and A. Zappia. (2015). Inherent Anticipation in the Pharmaceutical and Biotechnology Industries. Cold Spring Harbor Perspectives in Medicine, 5(8), a021006. doi:10.1101/cshperspect.a021006. To avoid objections based on inherency, claim unexpected uses for an uncovered property of a known composition, and be specific in the claims by defining subgroups, subsets of diseases or dosage regimens, and/or a new technical effect.

The most common disclosures of inventions are conference posters or presentations, Master of Science (MSc) or Doctor of Philosophy (PhD) theses, or a simple chat with a friend. A discussion with a qualified patent attorney or with your technology transfer professional is bound by confidentiality and should be the first point of call when realizing an invention.

There seems to be a consensus about the novelty requirement globally. Nevertheless, there are exceptions that are specific to each country or region. (26)Krauß, J. and D. Kuttenkeuler (2021). When to file for a patent? The scientist’s perspective. Nature Biotechnology, 60, 124–9. doi:10.1016/j.nbt.2020.10.006.

The JPO offers a grace period of 12 months. The USPTO grants a similar 12-month grace period for the inventor to file a patent application, even if he or she has disclosed the invention publicly in some way. The grace periods for other countries are described in more detail in a WIPO publication. (27)Grace period: certain aspects of national/regional patent laws. World Intellectual Property Office; 2022. Available at: https://www.wipo.int/export/sites/www/scp/en/national_laws/grace_period.pdf (accessed May 24, 2025).

The EPO offers a grace period of 6 months for only two situations, in which the publication arises from (i) a breach of contractual agreement or (ii) the disclosure of the invention at an officially recognized international exhibition. (28)Gates, C. (2014). Patenting the life sciences at the European Patent Office. Cold Spring Harbor Perspectives in Medicine, 4(12). doi:10.1101/cshperspect.a020792. An officially recognized international exhibition is defined by the terms of the 1928 Paris Convention, last updated in 1972. (29)Bureau International des Expositions. The 1928 Paris Convention; 1972. Available at: https://www.bie-paris.org/site/images/Documents/1928-convention-protocol-en.pdf (accessed May 24, 2025). The EPO published a list of previous exhibitions that fall within the terms of the 1928 Paris Convention. (30)International exhibitions as referred to in Article 55 EPC. European Patent Office; 2016. Available at: https://www.epo.org/law-practice/legal-texts/official-journal/2016/etc/se4/p219.html (accessed May 26, 2025). Taking this into account, applicants planning to file in Europe should assume that there is no grace period.

The EPO recently published a report on the impact of grace periods and how they lead to more unintentional infringement because of the longer period of uncertainty (increasing from 18 to 30 months) regarding freedom to operate. (31)The European Patent System and the grace period - an impact analysis. European Patent Office; 2022. Available at: https://link.epo.org/web/the_european_patent_system_and_the_grace_period_study_en.pdf (accessed May 26, 2025).

1.3 Inventive step or non-obviousness

Key point

  • Highlight all unexpected results as they are more easily accepted as proof of inventive step or non-obviousness.

Discussing inventive step (or non-obviousness) requirements is hard without first understanding the entity referred to as person having ordinary skill in the art (PHOSITA) or simply person skilled in the art, considered in the assessment of inventiveness. PHOSITA is an imaginary person that has average knowledge of a scientific field but zero creativity. For most of the topics of life sciences, a PHOSITA would be someone qualified to MSc or PhD level with an understanding of the science but without any creativity or idea of how to improve a technology or invention. Even though there are well-established methods to assess inventive step or non-obviousness in most jurisdictions to make the process as objective as possible, a certain degree of subjectivity still remains for the examiner, patent attorney and inventor(s), as no person can act 100 percent as PHOSITA.

Inventive step is one of the most important factors in the examination of a patent, and is where most of the arguments are debated between the examiner and the patent attorney. It is also one of the most difficult factors to define, as it always involves a degree of subjectivity from the examiner as well as their level of knowledge and experience.

Japan

Inventive step requirements for Japan are described in Article 29(2) of the Japanese Patent Act and in the examination guidelines published on their website. (32)Examination guidelines for patent and utility model. Japan Patent Office; 2024. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/tukujitu_kijun/index.html (accessed May 26, 2025). The JPO define the person skilled in the art as a person meeting their four conditions, but also mention that a person skilled in the art could sometimes be considered as a team of experts from several technical fields. The JPO examiners begin in the same way as the European examiners with the closest prior art (referred to as the primary prior art), and determine whether the person skilled in the art would easily arrive at the claimed invention by applying the four-step analysis described in the guidelines. (33)Examination guidelines for patent and utility model. Japan Patent Office; 2024. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/tukujitu_kijun/index.html (accessed May 26, 2025). The JPO also articulate two factors that support the inventive step: advantageous effects, an effect of different nature from that of the prior art or the same nature but significantly superior; and obstructive factor, where are other prior art documents obstruct the primary prior art document. The JPO provides several examples to aid understanding. (34)Examination guidelines for patent and utility model. Japan Patent Office; 2024. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/tukujitu_kijun/index.html (accessed May 26, 2025).

United States of America

The non-obviousness condition in the United States of America requires an invention to be not obvious to a PHOSITA. The easiest illustration of the non-obviousness principle is by having an invention combining concepts A+B, where A and B are concepts from two distinct prior art documents. If a PHOSITA read documents A and B, would it seem obvious to combine the two? If yes, then the non-obviousness condition is not fulfilled. The non-obviousness condition is described in section 103 of 35 USC. (35)Title 35–Patents. United States House of Representatives, United States Code (USC); 1952. Available at: http://uscode.house.gov/view.xhtml?path=/prelim@title35&edition=prelim (accessed May 24, 2025). The USPTO has published examiner training material that uses real examples to illustrate in more detail the approach taken by examiners. (36)Examination guidelines and training materials: obviousness. United States Patent and Trademark Office; 2010. Available at: https://www.uspto.gov/patents/laws/examination-policy/examination-guidance-and-training-materials (accessed May 26, 2025).

The factual inquiries that examiners make for the non-obviousness condition include the following. What is the scope and content of the prior art? What are the differences between the prior art and the claimed invention? What is the level of ordinary skill in the pertinent art at the time the invention was made? Does any objective evidence of non-obviousness exist? (37)Examination guidelines for determining obviousness under 35 U.S.C. 103. United States Patent and Trademark Office; 2019. Available at: https://www.uspto.gov/web/offices/pac/mpep/s2141.html (accessed May 26, 2025).

Europe

Inventive step requirements for Europe are described in Article 56 of the EPC and part G, chapter VII of the guidelines for examination. As in the United States of America, an invention in Europe is considered to have an inventive step if it is not obvious to a person skilled in the art. EPO examiners use the problem–solution approach to assess inventive step, which presumes that inventions are solutions to different problems. European examiners analyze inventive step in three main stages: determining the closest prior art, establishing the objective technical problem, and considering if it would have been obvious to a person skilled in the art to solve the objective technical problem in the same way that the patentee now claims to be their inventive contribution to the art. When asking the latter question, EPO examiners look for evidence of an unexpected technical effect associated with the presence of the underlying structural feature(s) that achieves the inventive contribution asserted by the patentee. (38)Part G, Chapter VII, 5. Problem-solution approach: guidelines for examination. Available at: https://www.epo.org/en/legal/guidelines-epc/2025/g_vii_5.html

1.4 Sufficiency of disclosure or enablement

The sufficiency of disclosure or enablement requirement is another element of international consensus with respect to the purpose of patenting. For governments to grant the patent rights, it is necessary that the invention is described sufficiently clearly to enable a person skilled in the art (or optionally, with the benefit of common general knowledge) to reproduce the invention as claimed (across the full scope of each patent claim). Certain types of biotechnology inventions are not workable (or reproducible, to a lesser extent) without access to a particular source or form of living biological material (e.g., a new bacterial strain, or a delivery vector charged with exogenous nucleic acid). In many cases, this problem is exacerbated when the biological material in question cannot be readily and reliably replicated by a skilled person. In such scenarios, the patentee should make a parallel Biological Deposit in accordance with the governing Budapest treaty provisions.

The enablement requirement in the United States of America is described in section 112 of the 35 USC, where it is deemed obligatory that an invention is described in a manner that enables a PHOSITA to make or use it as defined by the claims and the patent application. (39)2164: The enablement requirement. United States Patent and Trademark Office; 2013. Available at: https://www.uspto.gov/web/offices/pac/mpep/s2164.html (accessed May 26, 2025). The enablement requirement has become in recent years one of the hardest requirements for the patenting of life sciences invention in the country.

For Japan, the enablement requirement is described in Article 36(4)(i) of its Patent Act, (40)Japanese Patent Act; 1959. Available at: https://www.japaneselawtranslation.go.jp/en/laws/view/4097 (accessed May 24, 2025). and requires the description of the patent application to be clear and sufficient for the person skilled in the art to apply it. The JPO provide several examples illustrating this requirement. (41)Enablement requirement. Japan Patent Office. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/tukujitu_kijun/document/index/02_0101_e.pdf (accessed May 26, 2025). As for the other requirements, the USPTO provides free training materials. (42)Examiner training materials. United States Patent and Trademark Office. Available at: https://www.uspto.gov/learning-and-resources/examiner-training-materials (accessed May 26, 2025). The disclosure of invention requirements for Europe are described in Article 83 of the EPC (43)Article 83: disclosure of the invention. European Patent Convention. Available at: https://www.epo.org/law-practice/legal-texts/html/epc/2020/e/ar83.html (accessed May 26, 2025). and part F, chapter III of the guidelines for examination. (44)Part F, Chapter III. Sufficiency of disclosure: guidelines for examination. European Patent Office. Available at: https://www.epo.org/en/legal/guidelines-epc/2025/f_iii_1.html (accessed May 26, 2025). The patent application must disclose the invention (especially its essential features) sufficiently clearly and completely for a person skilled in the art to be able to put it into practice.

WIPO recently published a study on the sufficiency of disclosure requirements, which emphasizes the specific requirements relating to the biological, chemical and artificial intelligence technical fields. (45)Further study on the sufficiency of disclosure (Part I). World Intellectual Property Organization; 2022. Available at: https://www.wipo.int/meetings/en/doc_details.jsp?doc_id=582853 (accessed May 26, 2025). A comprehensive analysis relating to the disclosure requirements for genetic resources has also been published by WIPO. (46)Key questions on patent disclosure requirements for genetic resources and traditional knowledge. World Intellectual Property Organization; 2020. Available at: https://www.wipo.int/publications/en/details.jsp?id=4498 (accessed May 26, 2025).

1.5 Usefulness or industrial applicability

As for the novelty and sufficiency of disclosure requirements, the usefulness or industrial applicability is a factor applied by most patent offices worldwide. This requirement is rarely (if at all) the main objection of examiners regarding the patentability of an invention. The JPO describes the industrial applicability criteria in Article 29(1) of its patent act, (47)Japanese Patent Act; 1959. Available at: https://www.japaneselawtranslation.go.jp/en/laws/view/4097 (accessed May 24, 2025). and states that the word “industry” is similarly to be interpreted in a broad sense. (48)Eligibility for patent and industrial applicability. Japan Patent Office. Available at: https://www.jpo.go.jp/e/system/laws/rule/guideline/patent/tukujitu_kijun/document/index/03_0100_e.pdf (accessed May 26, 2025). The USPTO mentions the usefulness criteria in 35 USC 101. (49)Vidal, K.K. (2022) Patent eligible subject matter: public views on the current jurisprudence in the United States. United States Patent and Trademark Office; 2022. Available at: https://www.uspto.gov/sites/default/files/documents/USPTO-SubjectMatterEligibility-PublicViews.pdf (accessed May 24, 2025). The EPO will deem an invention “susceptible of industrial application if it can be made or used in any kind of industry” in accordance with Article 57 EPC (50)Article 57: industrial application. European Patent Office. Available at: https://www.epo.org/law-practice/legal-texts/html/epc/2020/e/ar57.html (accessed May 26, 2025). and part G, chapter III of the examination guidelines. In this context, “industry” is understood to encompass the biotechnology and pharmaceutical fields. (51)F II, 4.9 Industrial application. European Patent Office. Available at: https://www.epo.org/law-practice/legal-texts/html/guidelines/e/f_ii_4_9.htm https://www.epo.org/en/legal/guidelines-epc/2025/g_iii_1.html (accessed July 29, 2025).

1.6 Patent law vs. know-how or trade secret

The protection of intangible assets is a crucial aspect of modern businesses, with intangible assets accounting for 90 percent of the total value of the Standard and Poor’s 500 (S&P 500) index (the leading 500 companies in the United States of America) in 2020. (52)The soaring value of intangible assets in the S&P 500. Visual Capitalist; 2020. Available at: https://www.visualcapitalist.com/the-soaring-value-of-intangible-assets-in-the-sp-500/ (accessed May 26, 2025). Intangible assets are considered to fall within three different categories: registered IP rights (patents, trademarks, designs), unregistered IP rights (trade secrets, customer database, know-how, etc.) and goodwill. The advantages and disadvantages of each intangible asset type need to be considered when deciding the best protection for any creation. For most pharmaceutical and biotechnological innovations, the choice of the best form of protection will be either (i) patents or (ii) know-how or trade secrets.

Know-how

Know-how is defined in many ways by different bodies, but a legal definition provided by the EPO and European Union Intellectual Property Office defines know-how as “industrial information or technique likely to assist in the manufacture or processing of goods and materials.” (53)IP Teaching Kit Advanced II. European Union Intellectual Property Office. Available at: https://euipo.europa.eu/knowledge/course/view.php?id=1922 (accessed May 26, 2025). A more detailed definition of know-how from case law (54)Mycalex Corporation v. Pemco Corporation, 64 F. Supp. 420 (D. Md. 1946). Justia U.S. Law; 1946. Available at: https://law.justia.com/cases/federal/district-courts/FSupp/64/420/1952852/ (accessed May 26, 2025). is “factual knowledge not capable of precise, separate description. However, when used in an accumulated form, after being acquired as the result of trial and error, gives to the one acquiring it an ability to produce something which otherwise would not have known how to produce with the same accuracy or precision found necessary for commercial success.”

An accepted example of know-how can be a specific supplier list for certain raw materials. Negative information (e.g., experimental conditions that produce poor results) can also be considered as know-how. As another example, assume that it is generally known that using a particular fungal strain produces protein A at a certain range of temperature and humidity. Company One can make protein A using the fungi at a much lower cost compared with its competitors, because it has found through historic trial and error that the best yield is at a very specific temperature and humidity. This knowledge is part of company One’s know-how. (55)IP Teaching Kit Advanced II. European Union Intellectual Property Office. Available at: https://euipo.europa.eu/knowledge/course/view.php?id=1922 (accessed May 26, 2025).

Trade secrets

Trade secrets are defined as “confidential business information which provides an enterprise a competitive edge.” To be able to state that an intangible asset is a trade secret, it needs to meet the criteria of the general standards from Article 39 (56)Part II. Standards concerning the availability, scope and use of intellectual property right. World Intellectual Property Organization. Available at: https://www.wto.org/english/docs_e/legal_e/27-trips_04d_e.htm (accessed May 26, 2025). of the Trade-Related Aspects of Intellectual Property Rights agreement (TRIPS), which are listed below.

  • “It is a secret”: the information must be not generally known or easily discovered.

  • “It has commercial value because it is a secret”: loss of the trade secret would cause a financial loss to the owner.

  • “Reasonable steps under the circumstances, by the person lawfully in control of the information, to keep it secret”: an owner must be able to demonstrate the practical steps taken to keep the information secret. (57)Nealey, T., R.M. Daignault and Y. Cai (2015). Trade secrets in life science and pharmaceutical companies. Cold Spring Harbor Perspectives in Medicine, 5(4), a020982. doi:10.1101/CSHPERSPECT.A020982.

Well-known examples of trade secrets are the recipes for Coca-Cola and Colonel Sanders’ Kentucky Fried Chicken. In life sciences, trade secrets can be a useful approach at the very early stages of the drug discovery process or in protecting complex information, such as the particular sequence of conditions and activities used to produce a biological product. (58)Nealey, T., R.M. Daignault and Y. Cai (2015). Trade secrets in life science and pharmaceutical companies. Cold Spring Harbor Perspectives in Medicine, 5(4), a020982. doi:10.1101/CSHPERSPECT.A020982.

The Unfair Competition Prevention Act (UCPA) (59)Unfair competition prevention act. Japanese Law Translation; 1993. Available at: https://www.japaneselawtranslation.go.jp/en/laws/view/2803/en (accessed May 26, 2025). governs trade secret protection in Japan. The UCPA provides civil remedies and criminal penalties for trade secret misappropriation (UCPA, Articles 2(6) and 21).

In the United States of America, trade secrets are protected under both federal and state laws. The Defend Trade Secrets Act (DTSA) (60)Defend Trade Secrets Act of 2016. United States Congress; 2016. Available at: https://www.congress.gov/bill/114th-congress/senate-bill/1890 (accessed May 26, 2025). of 2016 provides a civil cause of action for trade secret misappropriation (18 USC section 1836). (61)18 U.S. Code § 1836 - Civil proceedings. Cornell Law School, United States Code. Available at: https://www.law.cornell.edu/uscode/text/18/1836 (accessed May 26, 2025). There are examples of prosecution for life sciences trade secret theft in this country, for example, Pacesetter Inc. v. Nervicon Co. or the Eli Lilly scientists trying to steal information for Jiangsu Hengrui Medicine Co. Ltd. (62)Nealey, T., R.M. Daignault and Y. Cai (2015). Trade secrets in life science and pharmaceutical companies. Cold Spring Harbor Perspectives in Medicine, 5(4), a020982. doi:10.1101/CSHPERSPECT.A020982.

In Europe, the European Union Trade Secrets Directive (2016/943) (63)EUR-Lex - 32016L0943. European Union; 2016. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX%3A32016L0943 (Accessed May 26, 2025). harmonizes trade secret protection across EU member states. Each member state has implemented this directive into their national laws, ensuring a consistent approach to trade secret protection.

Patents vs. know-how or trade secrets

The choice of whether to use either (i) patent protection or (ii) know-how or trade secrets depends on various factors, including the nature of the invention or innovation, the resources available to acquire and maintain protection, and the ease of enforcing the protection. Understanding the different benefits offered by these two approaches is crucial when advising innovators or making strategic decisions about the best ways to protect their intangible assets.

Patents are the most frequently used method of protection in the life sciences, as they provide commercial confidence to both investors and shareholders. Furthermore, regulatory requirements around the therapeutics field mean that simply not disclosing details of your new product or treatment may not be a viable approach to protecting your innovation. However, if circumstances allow, the use of know-how or trade secrets may have certain benefits, as outlined below.

  • Duration: Patents provide a limited term of protection (20 years), but know-how or trade secrets can last indefinitely, as long as the information is kept confidential.

  • Disclosure: Patents require the disclosure of the invention to the public, which can promote further innovation. However, know-how or trade secrets do not require disclosure, which may limit the dissemination of valuable knowledge.

  • Cost: Patent protection can be the most expensive aspect of IP, requiring application fees, attorney fees and ongoing maintenance fees. In addition, there are opportunity costs and impacts on the ability to outsource, cooperate, license etc. Know-how or trade secrets do not incur these legal costs; instead, high investment in security measures and regular staff training to maintain confidentiality are required.

  • Scope: Patent protection is conferred only by the scope of claims in view of the patent description, whereas know-how or trade secrets can cover a much broader range of information.

  • Territoriality: Patent protection is territorial, requiring separate applications and fees for each jurisdiction. However, know-how or trade secrets do not have territorial limitations, although the level of protection may vary between jurisdictions.

As well as the above benefits, the use of know-how or trade secrets has the risk of a third party being able to reverse-engineer your innovation and use it.

If trade secrets are the chosen method of protection, knowledge of prior use rights is important. If an entity files a patent on your trade secret innovation, you cannot challenge it as a result of its secrecy but you may defend yourself against patent infringement through prior use rights. Prior use rights are limited territorially and require the acts to have been performed in good faith. The USPTO published a report that details the particularities of prior use rights in Japan, the United States of America and Europe (including Denmark, France, Germany and the United Kingdom). (64)Report on prior user rights. United States Patent and Trademark Office. Available at: https://www.uspto.gov/sites/default/files/ip/global/prior_user_rights.pdf (accessed May26, 2025).

1.7 Impact of key patenting requirements on patent strength

Understanding the relative importance of these different factors for patentability, and the way in which they can impact upon strengths of any patent granted, can be useful when considering how to prioritize a patent budget or assess shortcomings in potential filings. As shown in Figure 2, experienced patent attorneys responding to our poll considered inventive step as having the greatest impact on sustainability of a granted patent. The issue of novelty based on prior art, and data-based issue of sufficiency of disclosure or enablement, were viewed as joint second, while patentable subject matter and industrial applicability came third and fourth, respectively. Bearing these relative weightings in mind may be useful when assessing the strengths or weaknesses of patents in your portfolio, or when deciding whether a particular innovation is ready for filing.

1.8 The role and sources of experimental data

Key points

  • There is always a compromise between the amount of data gathered to support the claims across their whole breadth, and the incentive of the first-to-file system.

  • WIPO Standard 26 (ST.26) of reporting biological sequences allows for consistent reporting across the different bodies.

1.8.1 Main data sources

In ideal circumstances, the same guidelines that are used for the publication of life sciences research should be used for life sciences patenting. Nature Publishing Group outlines a non-exhaustive list of the elements to be reported for life sciences research to improve the transparency and reproducibility of published results. The list includes experimental design reporting (sample size, randomization, blinding, etc.), statistics reporting (t-tests, P-values, mean, median, standard errors, etc.), description of reagents (cell lines, antibodies, catalogue numbers, primary citations, etc.), description of methods or protocols, and data deposition policies. The same document has links to guidelines regarding the reporting for biomarker studies, molecular structure determination, chemical compound characterization and microscopy. (65)Reporting life sciences research. Nature; 2015. Available at: https://www.nature.com/documents/nr-reporting-life-sciences-research.pdf (accessed May 26, 2025). The Proceedings of the National Academy of Sciences journal also published the MDAR (materials, design, analysis and reporting) framework for transparent reporting in the life sciences. (66)Macleod, M., A.M. Collings, C. Graf, V. Kiermer, D. Mellor, S. Swaminathan, et al. (2021). The MDAR (materials design analysis reporting) framework for transparent reporting in the life sciences. Proceedings of the National Academy of Sciences, USA, 118(17), e2103238118. doi:10.1073/pnas.2103238118. The MDAR author checklist provides guidance regarding all the details to be reported for a transparent and solid data set in life sciences. Because of the pressures of the first-to-file system, in practice the data required for a patent filing can be more preliminary than the level required for a peer-reviewed article. Some jurisdictions allow for post-filing data to be used during prosecution to support more limited data present in the application (e.g., recent decision of the EPO Enlarged Board of Appeal). (67)Communications from the Board of Appeal. European Patent Office. Available at: https://www.epo.org/law-practice/case-law-appeals/communications/2023/20230323.html (accessed May 26, 2025). Usually, post-filing data for the sufficiency of disclosure or enablement requirement are more easily accepted by examiners than new data in support of the inventive step. New data in support of the inventive step are accepted only in very specific circumstances (e.g., comparing the invention with the closest prior art to show unexpected advantageous results). (68)Altman, D., C. Sweeney and T. Yasui (2014). Preparing effective experimental data for pharmaceutical patent applications from US and Japanese perspectives. Pharmaceutical Patent Analyst, 3(5), 469–73. doi:10.4155/ppa.14.38.

Given the broad range of inventions encompassed within life sciences, it is very hard to outline all the potential data sources that can be used as exemplification for patent applications. Nevertheless, the data can be categorized into a few types of classes: in vitro, in vivo and ex vivo. There are other types of inventions in the life sciences field that are not a drug but are a system, device or method; data to support the scope of such claims have to be linked to what is considered relevant for that specific subfield.

Most of the inventions in the life sciences field are relevant to a part of the drug development process pathway, and the experimental data that are required to meet certain criteria along the pathway will also be used when patenting. Additional data might sometimes be required for patent applications to fully support the desired scope of claims. Since this report is focused on the disclosure and filing of new patent applications, more emphasis is given to data required in the earlier stages of drug discovery. A high-level overview of the drug development process is provided on the FDA website, (69)The Drug Development Process. United States Food and Drug Administration; 2018. Available at: https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process (accessed May 26, 2025). with a more detailed overview presented by Hughes et al. (70)Hughes, J.P., S.S. Rees, S.B. Kalindjian and K.L. Philpott (2011). Principles of early drug discovery. British Journal of Pharmacology, 162(6), 1239–49. doi:10.1111/j.1476-5381.2010.01127.x. in the British Journal of Pharmacology.

Some of the most used data sources in life sciences patents are sequences of nucleotides and amino acids. The sequences are reported according to the global sequence listing standard managed by WIPO. Sequence listings are required for patent application that mention DNA or RNA sequences of 10 or more nucleotides, or peptide sequences of four or more amino acids (irrespective of whether such sequences are mentioned in the claims). To address the drawbacks of WIPO Standard 25 (ST.25), which did not comply with the International Nucleotide Sequence Database Collaboration (INSDC) requirements, did not account for nucleotide analogues or D amino acids or branched sequences, and allowed for an inconsistent enforcement across patent offices, the sequences reporting standard was updated to ST.26 in July 2022. In comparison to the sequence listing being submitted in text format, ST.26 requires the sequence listing in an XML electronic format. (71)WIPO Standard ST.26 goes live! World Intellectual Property Organization; 2022. Available at: https://www.wipo.int/pct/en/news/2022/news_0039.html (accessed May 26, 2024).

A further feature of the sufficiency of disclosure or enablement requirement is that the data source used to support a patent application must show “possession” of the invention at the time of filing. This means that if the invention involves biological data this must be deposited or, if certain properties of compounds are important for the claims, they have to be measured across the entire scope of the claims (e.g., melting point). The possession condition is applied depending on the nature of the invention and state of knowledge. (72)Liberto, M. and P.J. Cuomo (2021). Don’t be caught without possession (of your invention): what you need to know about the written description requirement. IP Litigator, 27(2). Available at: https://www.mintz.com/sites/default/files/media/documents/2021-05-12/Liberto%20Cuomo%20Print.pdf (accessed May 26, 2025).

In vitro

In vitro refers to experiments performed outside of a living organism in a controlled environment such as a petri dish, centrifuge tubes, cell culture flasks, etc. In vitro assays are used significantly in the early drug discovery and preclinical research stages to establish profiles of both (i) drug metabolism and pharmacokinetics and (ii) absorption, distribution, metabolism and excretion. A non-exhaustive list of in vitro experimental data sources includes cell line assays, patient isolates assays and analysis, receptor binding tests, solubility tests, hERG binding assay, HepG2 cytotoxicity assay, P450s assays, microsomal assays to understand metabolic stabilities, drug metabolism assays, immunological assays (immunogenicity, immunophenotyping, cellular immune response) and genomics assays.

In vivo

In vivo refers to experiments performed in a living organism. Although important during the preclinical stage onward, in vivo studies are more costly and time-consuming compared with in vitro and ex vivo experiments, and are therefore used less often during the earlier drug discovery stages. In vivo experimental data sources often utilize animal models as primary disease models to obtain data demonstrating the effectiveness of the therapy at the preclinical stage, and to establish pharmacokinetic and pharmacodynamic profiles. The animal model of choice will vary for each of these applications. Once suitable data have been gathered to indicate effectiveness and safety, the drug candidate will progress to clinical phases where in vivo studies are conducted in humans. (73)Romero, L. and J.M. Vela (2014). Alternative models in drug discovery and development part II: in vivo nonmammalian and exploratory/experimental human models. In: In Vivo Models for Drug Discovery. Wiley, 59–90. doi:10.1002/9783527679348.ch03.

Ex vivo

Ex vivo refers to experiments performed on a tissue taken from a living organism. This type of model is considered to be a compromise between in vitro and in vivo models. Ex vivo assays have been developed for a wide range of applications; Xu et al.(74)Xu, Y., N. Shrestha, V. Préat and A. Beloqui (2021). An overview of in vitro, ex vivo and in vivo models for studying the transport of drugs across intestinal barriers. Advanced Drug Delivery Reviews, 175, 113795. doi:10.1016/j.addr.2021.05.005. describe the different types of ex vivo assays used for studying the transport of drugs across intestinal barriers, and Meijer et al. (75)Meijer, T.G., K.A. Naipal, A. Jager and D.C. van Gent (2017). Ex vivo tumor culture systems for functional drug testing and therapy response prediction. Future Science OA, 3(2). doi:10.4155/fsoa-2017-0003. describe different ex vivo models for use in the development of cancer therapies. Ex vivo models can also be presented for other types of innovations such as medical devices or training devices or systems (e.g., ex vivo testing of flexor tendon model to improve surgical training). (76)Papavasiliou, T., R. Nicholas, L. Cooper, J.C.Y. Chan, J. Ibanez, C.J. Bain, et al. (2022). Utilisation of a 3D printed ex vivo flexor tendon model to improve surgical training. Journal of Plastic, Reconstructive and Aesthetic Surgery, 75(3), 1255–60. doi:10.1016/j.bjps.2021.11.027.

1.8.2 Support of claims and patentability

Understanding the strength of a patent and its claims requires an understanding of the usual structure of a patent. The typical parts of a patent application are the description, which is split into different sections (title of the invention, technical field, background art, summary of the invention, brief description of the drawings and detailed description), claims, drawings and an abstract. These parts are described in detail in WIPO’s patent drafting manual. (77)WIPO patent drafting manual, second edition. World Intellectual Property Organization; 2023. Available at: https://tind.wipo.int/record/44657?v=pdf (accessed May 26, 2025).

The claims define the legal protection provided by the patent, and are interpreted by reference to the description supported by examples. The latter provide specific embodiments of the invention, and collectively provide a data support package illustrating the key technical effects of the invention. The description should envision as many of the potential variations of the invention as possible in order to provide the necessary written basis required to support amendment of claims, should this be required during the prosecution of the patent (some jurisdictions do not allow amendments that are not covered in the detailed description). As an absolute minimum, a patent application must include enough technical details (typically working examples and experimental data) to support any technical effect that is later cited.

The examples section typically begins with a materials and methods section, after which the different examples of embodiments of the invention are provided. Data are usually presented from the proof of the concept or underlying mechanism to ex vivo data, in vivo (animal model) data and clinical trial data (if available). When presenting the data, each significant result should be given its dedicated example, that is: Example 1, proof of concept; Example 2, in vitro data for certain subpopulation; Example 3, in vitro data for another subpopulation; Example 4, animal model experiments etc. Presenting the data in a story-like manner may help the examiner comprehend their relevance and the way in which they support the claimed invention. Making use of tables, graphs and figures is another strategy to highlight and summarize the key results in an easily understandable manner. The figures and graphs are usually presented separately from the text, normally at the end of the patent specification. A summary of the figures is usually presented in a “Brief description of the figures” section, and their relevance discussed in more detail in the Examples. When preparing the figures for the patent application, contrasting colors and different markers and legends should be used, as they will need to be presented in greyscale to satisfy the requirements of most patent offices.

When filing the patent application, the preferred embodiments should be covered in the examples section and at different levels of generalization to demonstrate that the invention works across the full area defined by the claims. To enhance visualization of the concept, the claims could be considered as a tool to describe a map of the patent landscape. For example, consider a patent application claiming Switzerland as a space in the patent landscape of planet Earth. If the patent application shows examples across the whole area of Switzerland, it would be difficult for the examiner to constrain the land to something else; however, if the examples covered only the Geneva and Lausanne area, then the examiner may decide to constrain the land to the French-speaking area only. On other words, the examples should demonstrate operability of the invention across the entire scope of claims (as eventually granted). As another example, if a claim cites an essential technical feature in terms of a range of 1–100 units, a sweet spot of preferred examples within the subrange of 40–60 units may be observed. However, to justify the broader range, additional exemplification (of the technical effect relied upon) within the 1–10 and 90–100 subranges would be required. The nature of the invention is also a factor of influence on the examiner’s restrictiveness, with pioneering inventions being allowed a broader scope.

In some cases, prophetic examples can be used to describe experiments that have not been performed but are planned. Such examples can describe predicted or simulated results, but examiners in the life sciences do usually not assign much importance to these types of examples (unless they are substantiated by experimental data). To have a strong position for the inventive step or non-obviousness requirement, any unexpected or surprising result or effect compared with the prior art or the knowledge in the field should be highlighted within the examples section.

Understanding the relevant prior art for an invention is crucial for a well-drafted patent application and claims. The claims are usually drafted to protect the invention, and to prevent any envisaged potential workarounds by competitors. The independent claims start from the essential elements of the invention with dependent claims adding other elements that, during prosecution, form alternative claims in case the examiner does not accept the independent claims as filed (onion-layer strategy). In exchange for a fee, several jurisdictions offer the possibility of conducting a search for prior art and a written opinion on the patent application. Many patent attorneys use this search and/or written opinion to refine the claims prior to the international application.

There is always a fine balance between how early to file a patent application and how much exemplification data to gather. Ideally, an application is filed as soon as the inventive concept is proven and exemplified. However, in very competitive fields, an application should be filed as soon as the inventive concept is clear, with filing updates used to incorporate more data as acquired.

How data are used within a patent application is an important consideration to how life science patent attorneys who participated in our survey rank different elements of drafting and data use for the patent defensibility. A diverse set of examples is considered to be the most important element, followed by a detailed description of and a strong example set for the preferred embodiments. Utilizing multiple data sources in examples and highlighting the problem that the invention solves is also seen as important. Less significance is given to the figures, and even less to the prior art discussion section. The prior art discussion section is avoided by some patent attorneys, as it may be used by the examiner in the arguments against the application during prosecution.

Some jurisdictions (most notably the United States of America) impose a duty on patent applicants, inventors and/or representing patent attorneys to disclose all known prior art information that ”a reasonable examiner would be substantially likely to consider important in deciding whether to allow an application to issue as a patent.” (78)Akron Polymer Container Corp. v. Exxel Container, Inc., 148 F.3d 1380. Cornell University Law School; 1998. Available at: https://www.law.cornell.edu/patent/comments/97_1438.htm (accessed May 26, 2025). This duty of disclosure is met by filing a list of references through an information disclosure statement (IDS). The IDS is usually filed at the same time as filing the patent application, or within 3 months of the filing the application for no fee. Additional fees are only applicable if filing more than one IDS, which is usually done for prior art documents identified in other jurisdictions. Failure to comply with this duty may lead to the unenforceability of the patent for “inequitable conduct.” The EPO does not impose this duty but, in some circumstances, it might ask for prior art cited by other offices. The JPO also applies a duty of disclosure, but the failure to comply does not affect the validity of the patent.