A common feature of modern human clinical trials is a double-blind placebo test. In such a trial, neither the doctor administering the drug nor the patient knows who is receiving the active drug and who is receiving a placebo. The placebo effect is well documented and commonly understood as a mental expectation response to being told that you are receiving a powerful drug. Patients taking a placebo report a variety of responses: some patients on the placebo report feeling better; some report no response and no side effects; others report that they are feeling worse or only experiencing side effects. Some patients, who are not experiencing side effects, believe they are receiving the placebo tend to drop out of studies. Patient drop out is a serious problem and in response, the “active placebo” was developed.

Active Placebos

An active placebo is a placebo that causes the same side effects as the active drug, but does not treat the patient’s disease. This helps to convince patients that they are receiving the active drug in the trial, and makes them less likely to drop out (but more likely to report side effects and curative effects of the drug).  Active placebos are commonly used in pain trials, giving people symptoms like drowsiness, nausea, or dry mouth.

Patentability under §101

35 U.S.C. §101 requires that a patent be given for any “new and useful process, machine, manufacture, or composition of matter, or any new and useful improvement thereof…” The “useful” requirement of §101 has been used to exclude immoral inventions from patentability. While this category used to include things like gambling machines, today things like suicide machines or a letter bomb would be excluded on an immorality basis. Could an active placebo fail the utility test of §101?

§101 utility requires a practical or specific utility, operability, and a beneficial utility. An active placebo would meet the operability requirement by showing that it did in fact give some people side effects. Presumably, the side effects would have to come from a pharmaceutically active ingredient in the placebo, and not be a result of mental expectation from the patient. The placebo would also pass the practical or specific utility test because it is useful in clinical trials for reducing the likelihood of patient drop out. However, is the active placebo beneficial? It is a device used to trick people into thinking they are taking a curative drug, when in fact the pill does nothing but give them a stomach ache!

The beneficial utility test is a very low bar, and a device that deceives the public may still have a beneficial utility. Here, it may come down to how the claims are written. A composition or compound claim for a drug to induce headaches or nausea would almost certainly be allowed. However, a method claim to tricking patients and taking advantage of the placebo or mental effects of the drug might tip the scales in the other direction. In the European Patent Office, the patent may fail, partly because the EPO expressly allows examiners to consider the morality of an invention, but also because the EPO, under the European Patent Convention, would not grant any method claims filed on an active placebo as being a surgical method.

The use of active placebos presents many regulatory and legal issues that the Bioethics Committee should consider, including patent eligibility. Some other issues involve the informed consent doctrine, the ethics of “do no harm”, and the integrity of clinical trials.  No matter what the committee recommends, they will face a tough decision that could affect many clinical study participants in the future.

• The Copyright Office released its latest group of exceptions to the Digital Millenium Copyright Act’s anti-circumvention provision. Wired and cnet news report on the exception for jailbreaking mobile phones.

• Also in DMCA news, Ars Technica discusses the Fifth Circuit decision that bypassing technological protections to access software for a fair use does not violate the DMCA anti-circumvention provision.

• The Supreme Court ruled on patentable subject matter in Bilski v. Kappos. Cnet, The MTTLR Blog, and IP Watchdog give their takes on the decision.

• Patent Docs reports on the memo issued by the USPTO to its examiners in the wake of Bilski. In a similar vein, Patently-O takes a look at the first post-Bilski test cases.

• The case between Joel Tenenbaum and the RIAA has taken another turn as U.S. District Judge Nancy Gertner slashed the jury verdict by 90%, calling it “unconstitutionally excessive.” See the coverage from Wired and Eric Goldman.

• Holman’s Biotech IP Blog looked at the recent limits that the EU Court of Justice has placed on patent protection for gene sequences.

• IP Watchdog has some thoughts on the early stages of a multi-billion dollar lawsuit filed against PayPal by XPRT Ventures, Inc.

Are Genes Patentable?

Naturally occurring things, even if newly discovered, may not be patented.  The ACLU asserts that the isolated genes in Myriad’s patents are no different from the genes that occur in nature.  The ACLU’s lawsuit seeks to invalidate Myriad’s patents.  They also argue that the patent covers basic human knowledge or thought (more specifically, the process of comparing the mutated gene to the normal one) and is therefore a violation of the First Amendment.  If the court accepts this reasoning, genes will no longer be patentable material under § 101.

Why It Matters: The BRCA Genes and Cancer

The BRCA-1 and BRCA-2 genes are linked to hereditary breast and ovarian cancer.  Women with mutations in these genes have a 40-85% chance of developing breast cancer, and suffer an increased risk for ovarian cancer as well.  Diagnostic tests can reveal these mutations, allowing women to better assess their risk.  Many women who have the mutated genes can undergo screening for the cancers earlier and more frequently than usual; some may even elect to have prophylactic surgeries.  Results can also serve to alert female relatives that they are more likely to have the mutation.  However, since Myriad holds patents on BRCA-1 and BRCA-2, it currently possesses a right to exclude others from performing these diagnostic tests.

The ACLU’s complaint points out that Myriad has failed to license its patent widely, which carries with it important implications.  Given that there are no alternatives, Myriad may freely price their diagnostics; many argue that the current rate ($3000 per test) is too expensive for many of the women who need to be tested.  Additionally, without other laboratories conducting similar tests, women are unable to secure second opinions.

The Big Picture

About 20% of human genes are patented.  Patent holders can prevent a researcher from studying or testing a particular gene.  Many of these genes, like BRCA-1 and BRCA-2, are associated with serious illness, including Alzheimer’s, muscular dystrophy, and colon cancer.  The ability to screen for genetic predispositions to these and similar diseases represents a great advance in personalized and predictive care.  There is concern, however, that allowing gene patents might slow development in this area.  The ACLU claims that, with gene patents, there is no opportunity to invent around the patent or to build upon and improve it.  They argue that this produces a disincentive to innovate.

As the court wrote in its opinion denying Myriad’s motion to dismiss, “the resolution of these issues will have far-reaching implications, not only for gene-based health care . . .  but also for the future course of biomedical research.”

For additional reading: Medical News Today, GEN

The ACLU’s complaint is available here.

By Claire Devine and Kyle de Neve.