[pp.int.general] Fwd: [SPICY IP] Application of Orphan Drug Act to section 3(d)?

Amelia Andersdotter teirdes at gmail.com
Mon Jul 18 14:39:22 CEST 2011

the reasoning about section3(d) and its application should be included 
in the suggested, near-time reforms of the patent system as proposed by 
Pirate Parties. spicyip has written quite a lot about the patent law 
section3(d) in india and its application is all but sensible (for 
instance, it is not extended to chemicals that are not pharmaceuticals, 
probably because the indian industry for chemicals is closer to being an 
originator industry than not - same controversy existsaround data 
exclusivity in the india/EU FTA, if one believes present india business 

well. the basis for the exception is sound, and it could very well be 
applicable within the EPC-zone (for instance). the last evaluation is 
biochemical patents in sweden was 2006, so there may be another one 


-------- Original Message --------
Subject: 	[SPICY IP] Application of Orphan Drug Act to section 3(d)?
Date: 	Mon, 18 Jul 2011 00:08:12 -0700 (PDT)
From: 	Rajiv Kr. Choudhry <rajiv.choudhry at gmail.com>
Reply-To: 	rajiv.choudhry at gmail.com
To: 	spicyip at googlegroups.com

Long post follows:
In an earlier post <http://goo.gl/h4PD3>, I had discussed providing for 
a numerical quantifier for section 3(d) of our patents act and in 
another <http://goo.gl/52S67> discussed of the possibility using an 
exclusionary definition of the term efficacy as relates to the act.This 
post provides a summary of definitions used in US Orphan Drug Act (ODA) 
to determine whether a drug is same or similar to another.This 
discussion is helpful to the Indian context because of two reasons: (1) 
the definitions as provided by the ODA consider similar issues as faced 
by our Controllers/Courts regarding defining efficacy for same or 
similar drugs, and (2) they have been applied and tested to specific 
cases and hence their impact and relative certainty can be measured.

The ODA was enacted by US Congress to help a relatively small number of 
consumers who suffer from a rare disease as there was not enough 
incentive for research and development (“R&D”) for those small number of 
consumers suffering from rare diseases.The ODA provided an incentive to 
pharmaceutical company manufacturing drug to help those few individuals 
affected by any one (rare) disease or condition to overcome the 
financial losses.The incentive was a seven year market exclusivity for 
discovery of new orphan drugs.Specifically, a drug is an "orphan drug" 
if it is for a "rare disease or condition" that effects fewer than 
200,000 patients in the United States or for which there is no 
reasonable expectation that the cost of developing the drug for a 
disease will be recovered from sales in the United States.FDA definition 
A little bit of science and history:Prior to the development of 
recombinant proteins by the biotechnology industry, simple (and smaller) 
chemical structures provided a basis for most drugs and two drugs were 
considered the same if they had the same active moiety.See definitions 
<http://goo.gl/ezF64>. “Active moiety means the molecule or ion, 
excluding those appended portions of the molecule that cause the drug to 
be an ester, salt (including a salt with hydrogen or coordination 
bonds), or other noncovalent derivative (such as a complex, chelate, or 
clathrate) of the molecule, responsible for the physiological or 
pharmacological action of the drug substance.”This approach of 
designating two structures worked well with small molecules because the 
chemical structure was a good indicator of a drug’s chemical 
syntheses.Therefore, changes to the chemical structure (of the active 
moiety) would most likely result in large pharmacologic differences for 
small molecules produced by chemical syntheses.
This definition, however, does not work for large molecules such as 
recombinant proteins, polysaccharides or nucleic acids, or genes, or 
glycoproteins.Living organisms contain genes, a unit of heredity.Genes 
consist of deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). DNA 
consists of two long polymers of "nucleotides." Nucleotides are arranged 
in a DNA molecule and a specific arrangement of the nucleotides is a 
"DNA sequence."DNA encodes the information that living cells use to 
build proteins and other RNA molecules.Proteins are essential parts of 
organisms and participate in virtually every process within 
cells.Functions include food digestion, metabolism, muscle formation, 
cell signaling, immune responses, etc.Proteins are made from amino acids 
using information encoded in genes. Each protein has its own unique 
amino acid sequence that is specified by the nucleotide sequence of the 
gene encoding this protein. The genetic code is a set of 
three-nucleotide sets called codons and each three-nucleotide 
combination designates an amino acid. Because DNA contains four 
nucleotides, the total number of possible codons is 64.Genes encoded in 
DNA are first transcribed into pre-messenger RNA (mRNA) by proteins such 
as RNA polymerase. Most organisms then process the pre-mRNA (also known 
as a primary transcript) using various forms of posttranscriptional 
modification to form the mature mRNA, which is then used as a template 
for protein synthesis by the ribosome.(Definitions of gene, protein, 
nucleic acids, DNA taken and combined from Wikipedia).
Without a proper definition of "sameness," any market exclusivity for a 
gene, a protein, nucleotide, or a glycoprotein could be completely 
valueless, because competitors can easily substitute nucleotides to 
create a "different" gene coding for an identical protein.In proteins, 
there is a specific problem, because certain individual amino acids are 
generally structurally interchangeable (because of their similar basic 
structure and net charge) without producing a noticeable functional effect.
To address the problems associated with these macromolecules, the ODA 
restricted the use of active moiety to small drug molecules and provided 
that large drug molecules cannot contain the "same principal molecular 
Specifically, the definitions <http://goo.gl/P98kM> provide:
(13) Same drug means:
(i) If it is a drug composed of small molecules, a drug that contains 
the same active moiety as a previously approved drug and is intended for 
the same use as the previously approved drug, even if the particular 
ester or salt (including a salt with hydrogen or coordination bonds) or 
other noncovalent derivative such as a complex, chelate or clathrate has 
not been previously approved, except that if the subsequent drug can be 
shown to be clinically superior to the first drug, it will not be 
considered to be the same drug.
(ii) If it is a drug composed of large molecules (macromolecules), a 
drug that contains the same principal molecular structural features (but 
not necessarily all of the same structural features) and is intended for 
the same use as a previously approved drug, except that, if the 
subsequent drug can be shown to be clinically superior, it will not be 
considered to be the same drug. This criterion will be applied as 
follows to different kinds of macromolecules:
(A) Two protein drugs would be considered the same if the only 
differences in structure between them were due to post-translational 
events or infidelity of translation or transcription or were minor 
differences in amino acid sequence; other potentially important 
differences, such as different glycosylation patterns or different 
tertiary structures, would not cause the drugs to be considered 
different unless the differences were shown to be clinically superior.
(B) Two polysaccharide drugs would be considered the same if they had 
identical saccharide repeating units, even if the number of units were 
to vary and even if there were postpolymerization modifications, unless 
the subsequent drug could be shown to be clinically superior.
(C) Two polynucleotide drugs consisting of two or more distinct 
nucleotides would be considered the same if they had an identical 
sequence of purine and pyrimidine bases (or their derivatives) bound to 
an identical sugar backbone (ribose, deoxyribose, or modifications of 
these sugars), unless the subsequent drug were shown to be clinically 
(D) Closely related, complex partly definable drugs with similar 
therapeutic intent, such as two live viral vaccines for the same 
indication, would be considered the same unless the subsequent drug was 
shown to be clinically superior.
FDA regulations define <http://goo.gl/P98kM> a "clinically superior" 
drug as:
(3)Clinically superior means that a drug is shown to provide a 
significant therapeutic advantage over and above that provided by an 
approved orphan drug (that is otherwise the same drug) in one or more of 
the following ways:
(i) Greater effectiveness than an approved orphan drug (as assessed by 
effect on a clinically meaningful endpoint in adequate and well 
controlled clinical trials). Generally, this would represent the same 
kind of evidence needed to support a comparative effectiveness claim for 
two different drugs; in most cases, direct comparative clinical trials 
would be necessary; or
(ii) Greater safety in a substantial portion of the target populations, 
for example, by the elimination of an ingredient or contaminant that is 
associated with relatively frequent adverse effects. In some cases, 
direct comparative clinical trials will be necessary; or
(iii) In unusual cases, where neither greater safety nor greater 
effectiveness has been shown, a demonstration that the drug otherwise 
makes a major contribution to patient care.

It can be seen that the *clinical superiority test is based on patient 
care rather than basing the test on structural similarity*.
Hence under our patent act, section 3(d) may also be interpreted based 
on similar guidelines and if appropriate, similar guidelines may be used 
by our courts to interpret patent claims for new compounds.
Our readers may wonder why I keep providing multiple definitions for 
section 3(d):The reason is simple-currently there are no guidelines that 
Assistant Controllers follow while rejecting or allowing patent claims 
under section 3(d).Each Asstt. Controller applies section 3(d) 
differently and just about anything is fair game when rejecting 
applications.The result is a mess.With no clear guideline, both the 
patentee and public suffer.Rejection of claims under section 3(d) 
differently leads to insufficient incentive to applicants.

Image from: http://en.wikipedia.org/wiki/Spot_the_difference

Posted By Rajiv Kr. Choudhry to SPICY IP 
at 7/18/2011 12:38:00 PM --
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