The only thing that was clear in a roundtable discussion the Patent and Trademark Office (PTO) held Thursday on gene-based diagnostics is that a consensus on the value of patents for the diagnostics, and DNA in general, is as out of reach as ever.
On the one side, industry representatives stressed the need for patents to spur development of new tests, which they said are the foundation of personalized medicine. Opponents of the patents repeated their arguments that DNA, as a part of nature, shouldn't be patentable, saying patents restrict access to second-opinion testing. Both arguments echoed those made at public hearings the PTO held last year on the subject. (See BioWorld Today, Feb. 17, 2012.)
But rehashing the value of the patents wasn't the purpose of the roundtable. Instead, the PTO was looking for recommendations it could make to Congress to keep the patent system in pace with advancing technology.
In passing the America Invents Act, which requires the PTO to conduct a study on genetic diagnostic patents, Congress recognized that the "innovation of tomorrow can't take root in the patent system of the past," Teresa Stanek Rea, a deputy undersecretary at the PTO, said in opening the discussion.
Some suggestions were made at the roundtable:
ban gene-based patents;
mandate compulsory licensing for all patents for genetic testing;
use the government's march-in authority on federally funded research;
create safe harbors for research or diagnostic use;
stick with the status quo.
Henry Wixon, of the National Institute of Standards and Technology, recommended incentives instead of heavy-handed clubs like a march-in. He pointed out that in the 30 years the government has had the authority to march in on patents resulting from federally funded research, it has never done so mainly because of the chilling effect the action would have on investors who provide the resources to turn the research into technology.
March-in should be considered on a case-by-case basis, said Arti Rai, of the Duke University School of Law. If research would be doomed to the so-called valley of death without private investment, march-in shouldn't be used. But if physicians are clamoring for the results of the research, the threat to march in may serve as the nudge needed to take the research to the next level and make it more accessible, she added.
Robert Cook-Deegan, of the Duke Institute for Genomic Sciences and Policy, called for collective action on the part of stakeholders to establish best licensing practices for genetic-based patents. But the PTO isn't the one to lead that action, he said.
In response to repeated comments about patients' rights, Hans Sauer, of the Biotechnology Industry Organization, said access to second-opinion testing should be more than just a recognition of a right. Instead, patients must be informed of their out-of-pocket costs since Medicare and most insurance policies refuse to cover second tests because they're not considered medically necessary, he said.
Sauer also questioned whether emphasizing second tests would create doubt about diagnostic accuracy where there was none before. Patients should be informed of the probability that the results of a second test will likely be the same as the first, he said, and they should be told of the risks of delaying treatment while they wait for a second test.
When the PTO makes its recommendations to Congress, it should follow the "do no harm" principle, Sauer said.
Teva Seeks Adcom for New MS Drugs
With the potential for another drug to enter the multiple sclerosis (MS) space in a few months, Teva Pharmaceutical Industries Ltd. is asking the FDA to hit the brakes in approving new MS drugs without an advisory committee review.
Citing safety concerns, the Jerusalem-based company, which markets MS drug Copaxone (glatiramer acetate), has filed a citizen petition asking the agency to require advisory committee evaluation of MS candidates as part of its approval process.
While the petition covers all MS candidates, it specifically mentions Biogen Idec Inc.'s BG-12 (dimethyl fumarate), which has a March 27 PDUFA date but no scheduled meeting with the Peripheral and Central Nervous System (PCNS) Advisory Committee.
"Biogen recently posted some troubling information about the safety and effectiveness of . . . BG-12 on a publicly accessible website" that noted kidney changes in animal studies, Teva said in the petition, adding that the website indicated the relevance of that information to humans isn't known.
To make its case for requiring an adcom evaluation, Teva mentioned safety concerns that cropped up postmarket for other MS drugs, including Biogen's Tysabri (nataluzimab) and Novartis AG's Gilenya (fingolimod).
In its petition, Teva reminded the FDA that the Federal Food, Drug and Cosmetic Act requires "advisory committee review of all new molecular entities prior to approval unless FDA provides sufficient justification for not doing so."
Given that numerous safe and effective treatments are available for MS, including relapsing-remitting MS (RRMS), Teva said there's no legitimate reason to bypass advisory committee evaluation of potential new MS therapies.
While the FDA did convene the PCNS advisory committee for Gilenya, it has approved other MS drugs without an adcom. Last year, for instance, it approved Genzyme Corp.'s once-daily oral Aubagio (teriflunomide) without input from the PCNS committee. (See BioWorld Today, June 14, 2010, and Aug. 28, 2012.)
If the FDA acts positively on Teva's request, the company may, in the future, have to take its laquinimod before the committee. A once-daily, oral immunomodulatory compound, laquinimod is being developed for RRMS as Teva's blockbuster Copaxone faces patent expiration in the next few years. With 2011 global sales of nearly $3.6 billion, Copaxone is Teva's top seller, accounting for almost a fifth of the company's sales revenue.
FDA Advises Lower Dose of Sleep Drugs
Citing new data, the FDA recommended halving the bedtime doses for women of insomnia drugs Ambien, Ambien CR, Edluar, Zolpimist and their generics, all of which contain zolpidem.
In a MedWatch Thursday, the agency said blood levels in some patients may be high enough the morning after use to impair activities such as driving. The MedWatch doesn't change the recommended doses of Transcept Pharmaceuticals Inc.'s Intermezzo, a lower dose zolpidem product approved for middle-of-the-night awakenings.
Intermezzo was approved in November 2011 with a label recommending a lower dosage for women than for men. (See BioWorld Today, Nov. 28, 2011.)