ARLINGTON, Va. _ The U.S. Patent and Trademark Office (PTO)released new draft guidelines Thursday instructing biotechnologypatent examiners to accept "credible" scientific evidence _ and notrequire clinical trial data _ as proof of utility for compounds thataim to treat human disease.

"These guidelines will shift our approach in reviewing theseapplications from one which doubts whether an invention works toone where we assume it does unless there are sound reasons tosuspect otherwise," said Commissioner and Assistant Secretary ofCommerce Bruce Lehman at a press conference. "The examiner isnot going to be the inquisitor or act as an FDA official." (For moreon the guidelines, see BioWorld Today, Dec. 22, 1994, p. 1.)

The interim guidelines, although subject to public comment untilFeb. 24, 1995, are to take effect immediately, said Lehman. Therules instruct examiners to assess the credibility of utility claimsfrom the perspective of "one of ordinary skill in the art" and using"any evidence of record" (e.g., data, statements, opinions, references,etc.). In most cases, Lehman said, standard screening tests thatindustry uses to gauge a drug's potential will satisfy PTO utilityrequirements.

Examiners must now accept "as true, credible statements made by anapplicant." According to the guidelines, federal courts have rarelysustained PTO rejections on the basis of utility. In the rare caseswhere a utility rejection was sustained, it was because applicantsasserted what is known as "incredible utility," a utility that couldonly be true if it "violated a scientific principle, such as the secondlaw of thermodynamics, or a law of nature, or was whollyinconsistent with contemporary knowledge." The PTO guidelinesremind examiners that "incredible utility" is one possible conclusionafter extensive review of an application, not "a starting point foranalysis."

The new guidelines further raise the evidentiary hurdle for examinerswho rule that a claim should be rejected for lack of utility. Utilityrejections must now include "a prima facie showing" that theclaimed invention has no utility. In order to prove that rejection isself-evident, examiners must "set forth the reasoning used," provide"factual findings" in support of it and make clear why the utilityclaims "would not be considered persuasive to a person of ordinaryskill in the art."

Once a prima facie showing of no utility has been "properlyestablished," the patent applicant bears the burden of rebutting it.Applicants can amend the claims, provide reasoning or arguments, orprovide new evidence (such as a printed publication) that rebuts theprima facie showing.

Following are several direct excerpts from the section of theguidelines entitled, "Special considerations for asserted therapeuticor pharmacological utilities" (all underlining is from original PTOdocument):

* Correlation Between Evidence And Asserted Utility

As a general matter, evidence of pharmacological or other biologicalactivity of a compound will be relevant to an asserted therapeutic useif there is a reasonable correlation between the activity in questionand the asserted utility. The applicant does not have to prove thatthere is a statistically proven correlation between characteristics of acompound and the asserted use, nor does he or she have to provideactual evidence of success in treating humans where such utility isasserted.

* In Vitro And Animal Data

Data generated using in vitro assays and testing in animals almostinvariably will be sufficient to support an asserted therapeutic orpharmacological utility. In no case has a federal court required anapplicant to support an asserted utility with data from human clinicaltrials . . . If one skilled in the art would accept the animal tests asbeing reasonably predictive of utility in humans, they should beconsidered appropriate to support the credibility of the assertedutility. Examiners should be careful not to find evidenceunpersuasive simply because no animal model for the human diseasecondition had been established prior to the filing of the application.

* Human Clinical Data

There is no decisional law that requires an applicant to provide datafrom human clinical trials to establish utility for an invention relatedto treatment of human disorders, even with respect to situationswhere no art-recognized animal models existed for the humandisease encompassed by the claims . . . As a general rule, if anapplicant has initiated human clinical trials for a product or processused for treating an indication, the subject of that trial has met theburden of being reasonable predictive of utility.

* Safety and Efficacy Considerations

Other agencies of the government have been assigned theresponsibility of ensuring conformance to standards established bystatute for the advertisement, use, sale or distribution of drugs. Thus .. . it is improper for an Examiner to request evidence of safety in thetreatment of humans, or regarding the degree of effectiveness.

* Treatment Of "Incurable" Diseases

Claims directed to a method of treating or curing a disease for whichthere are no successful treatments available may warrant carefulreview . . . For many of these "incurable" diseases there may be noart-recognized animal models available for testing the efficacy oftherapeutic agents which are predictive of results in humans. Themere fact that there is no known cure for a disease, however, shouldnot serve as the basis of an Examiner's conclusion that such aninvention lacks utility . . . a greater amount of evidence may berequired to support utility where the applicant has claimed that theinvention will cure the disease, where less evidence would berequired to establish that a claimed invention would be useful inalleviating one or more symptoms of the disease. n

-- Lisa Piercey Washington Editor

(c) 1997 American Health Consultants. All rights reserved.