Artist Bjørn Bjørnholt

Patents on Medical Uses Knowing your opportunities

October 2011

Lars Hallander M.Sc., Ph.D. Molecular Biology Senior Patent Attorney T +45 33 63 95 60 E lah@pv.eu

Overview • Introduction

• Requirements for patentability

• European patent law and practice

• Patents on medical use: Limitations and escapes

• A hypothetical case

• Possible patent claims and consequences

• Issues to discuss with your patent attorney

• Short case studies

• Conclusions and questions

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Introduction

Patentability requires:

• Novelty ”An invention shall be considered to be new if it does not form part of the state of the art.”

• Inventive step (non-obviousness) ”An invention shall be considered to involve an inventive step if, having regard to the state of the art, it is not obvious to a person skilled in the art.”

The invention must solve a technical problem.

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European patent law and practice

Limitations and escapes:

• For ethical reasons methods for treatment of the human or animal body by therapy are exempt from patenting.

Therapy includes prophylactic and therapeutic treatment directed to maintaining or improving the health of the body.

Cosmetic treatment, termination of pregnancy, sterilization, treatment for experimental purpose etc.

• The exclusion does not apply to products, in particular substances or compositions, for use in therapy.

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European patent law and practice

Patentabilty of novel medical use:

• Even if a substance or composition is comprised in the state of the art:

The compound or composition for use in a method for treatment of the human or animal body by therapy may be patentable if no such use is disclosed in the state of the art.

• Even if the use of a substance or composition in a therapeutic method is comprised in the state of the art:

The substance or composition for any specific use in a therapeutic method may be patentable, provided that this specific use is not comprised in the state of the art.

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European patent law and practice

1st medical use claim: Compound of formula (1) for use as a medicament.

2nd and subsequent medical use claims: Compound of formula (1) for use in treatment of disease X.

Compound of formula (1) for use in treatment of disease Y.

…

The product is chracterised by its intended use; i.e. the purpose for which the product is marketed!

The intended use can be determined by inspection of the product label and the marketing authorization.

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European patent law and practice

What constitutes a ”specific” medical use?

• Treatment of a new disease or condition. e.g. a compound for use in treatment of diabetic retinopathy where the same compound for use in treatment of Crohn’s disease is known.

• Treatment of a novel group of subjects. e.g. a compound for use in controlling bleeding in non-haemophilic mammals may be patentable, even if the same compound for controlling bleeding in haemophilic mammals is known.

• A new route of administration. e.g. a compound for subcutaneous injection may be patentable, even if intramuscular injection of the same compound is known.

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European patent law and practice

• A new dosage regime. e.g. a compound for intermittent administration may be patentable, even if continuous administration of the same compound is known.

• Different technical effect leading to new application. e.g. a salt solution for use in removing plaque from teeth may be patentable even if use of the same solution for depressing solubility of tooth enamel in organic acid is known.

• Possibly other technical features relating to the mode of use???

Does this definition create complete legal certainty?

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European patent law and practice

Requirements for experimental support:

• Clinical trials are not required to support medical use claims.

• In vivo data from relevant animal models are accepted.

• Even in vitro data may suffice, where: ” …there is a clear and accepted established relationship between the shown physiological activities and the disease.”

• Post-filed data are accepted. If the patent application as filed contains proof of concept data, the applicant may later, during prosecution of the patent application, file evidence to back up the initial findings in the patent application.

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A hypothetical case

“Company A” is developing a known compound (“substance X”) as a drug for treatment of several CNS indications and has supportive preclinical data.

• Which medical use claims can the company possibly make?

Possible medical use claims under European patent law assuming that “substance X” has not previously been used for a medical purpose:

1. Substance X for use as a medicament.

2. Substance X for use in treatment of Alzheimer’s, Huntington’s, Parkinson’s…etc.

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A hypothetical case

The same company generates convincing data suggesting an effect in a new, not previously claimed, CNS condition. The company intends to perform a clinical trial in this condition.

• Does the existing patent cover this new condition?

Yes, by way of the 1st medical use claim.

• Is it possible to obtain a patent for use in this new condition?

Yes, in the form of 2nd medical use claims, depending on what is comprised in the state of the art – including any previous patent applications from Company A.

• What is the benefit of filing a new patent application?

It prolongs the patent protection and prevents the grant of 2nd medical use claims to a third party will be dominated by the 1st medical use claim granted to Company A.

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Issues to discuss with your patent attorney

Is your invention based on a valid rationale and do you have sufficient proof of concept when filing your application?

Potential implications if not?

Example: Use of thalidomide in treatment of cancer. European Patent was granted to Children’s Medical Center Corporation, Boston, in 2005 – Plougmann & Vingtoft opposed on behalf of a client.

Patenting was based on the rationale that angiogenesis (formation of new blood vessels) is a prerequisite for tumor growth.

Thalidomide was effective in an in vivo model of angiogenesis: The rabbit cornea angiogenesis assay.

Eventually, thalidomide turned out to be effective only in treatment of multiple myeloma – a cancer of plasma cells in the bone marrow. Multiple myeloma was not mentioned in the patent application.

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Issues to discuss with your patent attorney

Do you have sufficient knowledge of the state of the art?

If not, what are the potential implications?

Example:

Use of thalidomide in treatment of cancer. State of the art included an old study in 21 cancer patients (Olsson et al.) showing “subjective improvement”, “possible arrest of tumor growth” and palliative effects!

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Issues to discuss with your patent attorney

How do your patent claims define the disease/medical indication and how is it defined in your application for marketing approval?

Example:

Use of thalidomide in treatment of cancer Claim 1 reads:

“1. Thalidomide for use in the treatment of a solid or blood-borne tumour.”

What is a blood-borne tumour?

Does “a solid or blood-borne tumour” include multiple myeloma?

During prosecution, applicant argued that ”blood borne tumour” referred to ”malformed cells in the bone marrow”.

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Issues to discuss with your patent attorney

How is your product marketed?

Medical use claims protect the way the final product is marketed. Consider the following situations:

• Bed-side applications – e.g. use of stem cells for regenerative therapy.

The final product is prepared by the medical practitioner.

• Products which do not require regulatory approval - e.g. salt solution ”to remove plaque from teeth”.

The salt solution may alternatively be marketed for the purpose of ”improving dental hygiene”.

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Conclusion

• Coordinate strategies for research, regulatory approval, establishment of patent protection and marketing.

• When applying for patent protection, provide the best possible disclosure of your technology in terms of description and experimental support.

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After having developed the new, revolutionary cancer therapy, I spent 9 years writing patent applications and letters to the patent office… …and eventually, I was rewarded with the Nobel Prize!

Wow, the Nobel prize in medicine!?!

No, in literature!!!