TOPICS IN (NANO) BIOTECHNOLOGY

Lecture V

29th April, 2003

PhD Course

Bringing a drug to market

• Beginning of 21st century – 369 biotechnology medicines in testing fro more than 200 diseases

• To bring a drug to the market need:– 12-15 years– $300-500M

– And with all this might have 1 lucky candidate out of every 5,000-10,000 tested.

Current state of affairs

• Costs about $155-255M

• Lead compound screening• Discovery testing• Scale-up• Stability & formulation.

1. Discovery Research

• Costs about $65-90M

• Uses results from in vitro experiments (cell cultures) and in vivo animal models

• If no effects from long-term chronic use or appearance of carcinogenicity – go to next phase

2. Pre-clinical

• If company/lab decides to pursue human studies, must submit an IND to the Food and Drug Authority– IND is effective 30 days after receipt by the FDA– IND must provide pre-clinical data to justify testing in

humans– About 85% of all IND applications move on to begin

clinical trials

• CLINICAL TRIALS– Testing experimental drugs in humans

3. File an Investigational New Drug application

• Cost about $10M or more

• Examine drug safety, pharmacology, determine drug regimen

• 1 in 2500 compounds get this far• Participants screened for harm caused by the

medicine• Involve 20-100 patients• Phase I shows the drug to be reasonably safe –

further trials

4. Phase I Clinical Trials

• Cost about $25-35M

• Confirm safety, determine efficacy, set up dosage of Phase III trials

• 100-300 patients suffering from the illness are recruited

• Placebo controlled and double-blinded

5. Phase II Clinical Trials

• Cost about $100M or more

• Establishes safety and effectiveness in large group of volunteers over the long term

• Double-blinded, placebo controlled, involve 500-3000 patients overy months-years in wide range of sites

• Minimal risk• Rare side effect of treatment can be identified• Very, very expensive but ESSENTIAL for FDA

approval

6. Phase III Clinical Trials

• FDA reviews all drugs and clears them for marketing

• New Drug Applications – typically 120,000 pages of data

• 12 months for FDA to review• Often, more data is required…sometimes clinical

trials need to be repeated.

…..but are there any shortcuts?

7. File an NDA with the FDA

• Fast track review – investigators work closely with FDA and can

submit the NDA in parts

• Priority review – standard designation – 10 month target date– priority designation – 6 month – typically used for

drugs that address unmet medical needs

• Accelerated review– Drugs that promise a significant advantage over

existing drugs

Speeding things up…

• Compassionate use – lobby for access to experimental medication to save

life of critically ill patient– Request not always accepted– BUT can help with attaining FDA approval– Assesed on a case-by-case basis

• Orphan Drug Act (USA, 1983) – Encourage the developemnt of drugs for rare

diseases– Diseases afflict less than 200,000 patients – Seven year exclusive right – lucrative tax incentives

Speeding things up…

• Avigen• Human Genome Sciences• IDEC pharmaceuticals• ICOS• Milennium Pharmaceuticals• Medimmune

…check them out!

Some biotech companies involved in clinical trials

Intellectual Property Issues for New Technology

Business

Contents

• A Basic Introduction to IP Rights– Patents– Confidential Information– Copyright– Design Right– Trade Marks

• Protecting Developing Technology in the Real World• The Need for a Strategy• Licensing

The purpose of patent law

• A contract between state and inventor• Innovation recognised as a good thing• Monopolies recognised as a bad thing• Why invest in innovation if results freely available to

third parties (and competitors)• The Deal

– Twenty year monopoly in exchange for teaching invention

The Form of a Patent• The form of the contract

– Specification• Describes invention• Instructs ordinary man skilled in the art how to

work the invention• Gives examples

– Claims• Define legal monopoly granted

The Law

• Patents Act 1977 (“PA”)• European Patent Convention • Biotechnology Directive (Council Directive 98/44 on

the Legal Protection of Biotechnological Inventions)• European patent designated for GB treated as GB

patent, governed by PA

Patentability• Section 1(1) PA (1) A patent may be granted only for an invention in

respect of which the following conditions are satisfied, that is to say-

a)the invention is new; b)it involves an inventive step; c)it is capable of industrial application; d)the grant of a patent for it is not excluded by

subsections (2) and (3) below; and references in this Act to a patentable invention

shall be construed accordingly.

Exclusions

Section 1(2) (2) It is hereby declared that the following (among other

things) are not inventions for the purposes of this Act, that is to say, anything which consists of-

(a) a discovery, scientific theory or mathematical method;

…... but the foregoing provision shall prevent anything from

being treated as an invention for the purposes of this Act only to the extent that a patent or application for a patent relates to that thing as such.

Moral Exclusion

• Section 1(3) & (4) (3) A Patent shall not be granted for an invention the

commercial exploitation of which would be contrary to public policy or morality.

(4) For the purposes of subsection (3) above behaviour shall not be regarded as contrary to public policy or morality only because it is prohibited by any law in force in Great Britain or any part of it.

Novelty

• Section 2 PA (1) An invention shall be taken to be new if it does

not form part of the state of the art.

(2) The state of the art in the case of an invention shall be taken to comprise all matter ... which has at any time before the priority date of that invention been made available to the public (whether in the United Kingdom or elsewhere) by written or oral description, by use or in any other way.

Inventive Step

• Section 3 PA An invention shall be taken to involve an inventive step if

it is not obvious to a person skilled in the art, having regard to any matter which forms part of the state of the art ….…

• Who is the “person skilled in the art”?– Historically an individual with no inventive ability– Genentech - team of PhDs with significant inventive

ability– Appears now to vary according to the art

• No need for rocket science?

Industrial Application• Section 4 PA 1) Subject to subsection (2) below, an invention shall be

taken to be capable of industrial application if it can be made or used in any kind of industry, including agriculture.

(2) An invention of a method of treatment of the human or animal body by surgery or therapy or of diagnosis practised on the human or animal body shall not be taken to be capable of industrial application.

(3) Subsection (2) above shall not prevent a product consisting of a substance or composition being treated as capable of industrial application merely because it is invented for use in any such method.

Enabling disclosure and sufficiency • Section 14 PA (3) The specification of an application shall disclose the

invention in a manner which is clear enough and complete enough for the invention to be performed by a person skilled in the art.

(5) The claim or claims shall- (a) define the matter for which the applicant seeks protection; (b) be clear and concise; (c) be supported by the description; and (d) relate to one invention or to a group of inventions which

are so linked as to form a single inventive concept.

Enabling disclosure and sufficiency (2)

• Enabling Disclosure– must enable skilled man to work invention– need not teach every way of working invention

• Sufficiency– specification must fully support the claims– must support every way of achieving what is

claimed• Genentech - claimed recombinant form of natural

t-PA• only taught one way of achieving it

Confidential Information

• No statute - common law right• Protects information which

– is confidential (not in the public domain)– was disclosed under an obligation of confidence

• express or implied– has “the necessary quality”

Copyright

• Copyright Designs and Patents Act 1988 (CDPA)

• Protects original literary, dramatic, musical and artistic works

• Very low threshold for literary or artistic merit• Literary works include

– software– manuals specifications

• Artistic works include– technical drawings

Copyright (2)

• Subsists automatically - no registration• 70 years from death of author• Not an infringement of © in a design drawing to make

an article to that design• First owner

– Author, or– if produced in course of employment - employer.

Trade Marks• Registered or unregistered• Identifying source of origin

– sign capable of distinguishing goods or services of one undertaking from those of other undertakings

• Can be important for new businesses– Microsoft was once a start-up– INEX– Goodwill associated with research establishments

Protecting Developing Technology in the Real World

• Protection v publication– the danger of self-anticipation

• Post - filing– danger of losing priority

• The value of a properly drafted application– avoid substantial amendments– best chance of maintaining priority

• Need for NDAs both pre and post filing • Material transfer agreements

The Need for a Strategy

• The costs of patenting – Cheap (ish) to start– 12 months to PCT– 18 Months to National/Regional phase

• Valuing your patent– Commercial advantage– Access technology v. corale

• Cost/Benefit• Let go what isn’t valuable

Licensing

• Right to use technology

• Exclusive, sole, non-exclusive

• Royalties, licence fee

• Minimum royalties/performance requirements

• Restrictions on fields, territories

• Choosing the right licencee

• Enforcement

Licensing (2)

• Exclusive/non-exclusive– Benefits of exclusive

• higher royalties• control• performance criteria• enforcement support

– Benefits of non-exclusive• more than one licensee• competition• market penetration