Investment analyst predicts explosive biotech growth

In a recent interview with The Life Sciences Report, a Casey Research analyst likened genetics to other twentieth-century technologies which remained in development for many years but eventually yielded consumer products that are now all but everywhere.

Just as the plasma TV (invented in the 1930s), the LED light (1960s), the industrial robot (also a child of the '60s), the touch-screen interface for computers (early 1980s) and other inventions we think of as thoroughly modern took decades to go from the lab into our everyday lives, it will take considerable time for genetic medicine to fully develop.

When it was first invented in the 1990s, he noted, genetic sequencing was prohibitively expensive and time-consuming, requiring billions of dollars and years to decode a single genome. Today, the same task can be completed in a day and for a few thousand dollars. This, he said, now makes possible the large-scale development and marketing of genetic medicine.

Appeals court upholds California law requiring arrestees to give DNA sample

Last month, the Ninth Circuit Court of Appeals upheld a California law requiring individuals arrested on  suspicion of having committed felonies to provide a DNA sample to the government. Proponents of the law point out that it helps identify criminals by allowing the government to compare the DNA samples it collects from arrestees to other DNA profiles found in criminal databases. Because DNA is unique to every individual, matching DNA profiles can lead to the perpetrators of unsolved crimes.

Critics, however, worry that the law represents an undue intrusion on our privacy. In effect, the law requires individuals to provide a DNA sample even though they may not be convicted of any crime ultimately. The DNA thus obtained is stored in databases accessible to local, state, national, and international law enforcement agencies.

In 2009, the ACLU challenged the law arguing that it was unconstitutional under the Fourth Amendment’s prohibition on unreasonable searches and seizures.

In a 2-1 decision, the Ninth Circuit balanced the government’s interests against those of the individual affected by the law and held that the law did not violate the Fourth Amendment. It explained:

Given the arrestee’s diminished privacy interests; the de minimis nature of the physical intrusion entailed in the taking of a buccal swab; the carefully circumscribed scope of the DNA information being extracted; the stringent limits on the manner in which that information may be used; and the well-established law enforcement interest in obtaining arrestee’s identifying information, and further, to deter future criminal acts and to exculpate innocent arrestees—the balance of interests tilts strongly in favor of upholding the constitutionality of [the law].

The case is significant because it provides an indication on whether similar laws enacted in other states will survive constitutional scrutiny. Last year, New Jersey became the 25th state to have passed a DNA arrestee law. 

Michael Risher, an attorney working for the ACLU said his clients would seek review of the decision by the full Ninth Circuit. 

BIO testifies in support of reauthorization of the Prescription Drug User Fee Act

Testifying before the House Subcommittee on Health on behalf of the Biotechnology Industry Organization, Richard Pops, CEO of Alkermes, called on Congress to reauthorize the Prescription Drug User Fee Act (PDUFA).The PDUFA allows the FDA to collect fees from entities seeking the approval of a new drug or biologic product. It also authorizes fees based on manufacturing facilities and covered products currently on the market. The FDA uses the revenue to fund and expedite the application review process. First enacted in 1992, the PDUFA was reauthorized in 1997, 2002, and 2007. Congress is considering its fifth reauthorization this year.

Pops indicated that since 1992, the user fees prescribed under the PDUFA have reduced the review time for new drugs by more than year. Still, he pointed out that the average time it takes for a treatment to become available to patients from the time of its discovery is 10 to 15 years.

He stated:

In short, BIO supports quick enactment of the PDUFA V recommendations as we believe they can enhance the drug development and review process through increased transparency and scientific dialogue, advance regulatory science, and strengthen post-market surveillance. Most importantly, from the standpoint of young, innovative companies, our hope is that PDUFA V will provide patients and doctors with earlier access to breakthrough therapies.

Pops said that the PDUFA V will make possible the following:

- greater scientific dialogue and transparency between FDA and Sponsors under the proposed review program for novel drugs and biologics;
- enhanced communication during drug development between FDA and Sponsors;
- modernization of regulatory science;
- robust drug safety and post-market surveillance capacity.

In addition, Pops discussed the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA) which he said "have generated a wealth of pediatric drug information for physicians and parents, contributing to improved health outcomes for pediatric patients." He called on Congress to "(1) reauthorize the existing framework and incentive for ongoing pediatric research and (2) make the programs permanent by eliminating their sunset provisions."

As reported in Genetic Engineering & Biotechnology News, FDA Commissioner Margaret A. Hamburg has also called on Congress to approve the fifth authorization of the PDUFA.

FDA drug safety decision may have been influenced by big pharma

The Project on Government Oversight (POGO) reports that big pharmaceutical companies may be having an undue influence over the Federal Drug Administration (FDA), raising doubts over the agency's drug safety determinations.

On December 8, 2011, an FDA panel of medical experts held a vote on the relative safety of two best-selling contraceptive pills manufactured by Bayer. Asked whether a panelist's ties to Bayer had "affected her judgment in any way regarding her vote," a spokesman replied "no comment."

Two other panelists had prior ties to Bayer but claimed that their vote was unbiased.

Under consideration were two contraceptives named Yaz and Yasmin containing the synthetic hormone drospirenone. The panel concluded that the risks of taking the contraceptives did not outweigh their benefits in a 15 to 11 vote.

The FDA panel did not take into account, however, an internal Bayer document revealed in litigation against the company showing that one of the contraceptives had a rate of serious "adverse events" that was "10 fold higher than other contraceptives."

Overall, POGO found that four out of twenty-six voting panelists had prior relationships with Bayer or a generic company that markets a generic equivalent of the Bayer contraceptives. All four voted in favor of Bayer contraceptives.

The four panelists disclosed their links but the FDA did not object to their participation on the panel. Based on the panel's deliberations, POGO concluded that there was "no indication that fellow panelists were ever aware of these links as the group spent hours deciding how to deal with the well-documented risks associated with Yaz, Yasmin, and related drugs."

Legal, social, and ethical issues raised by whole genome sequencing

Using whole genome sequencing (WGS), scientists are able to describe an individual's entire genome on a molecular level.  In 1-4 years, it is expected that WGS will cost as little as $1,000, an impressive advancement given that it cost $100 million to complete the sequencing of the first human genome in 2000.

In last fall's briefing of the ABA Biotechnology Law Committee, Gary Merchant and Rachel Lindor of the Sandra Day O'Connor College of Law, discuss the legal, social, and ethical issues that WGS implicates.

The authors believe that WGS will soon bring about a genomic revolution that will have a significant impact on how we treat human disease.

The issues they identify are the following:

- whether the patents currently covering 4000 human genes will impede WGS (for an argument that this is unlikely, see this post);
- the difficulty of obtaining informed consent from research participants because of the complexity of genetic information and the potential scope of findings;
- whether findings incidental to the original purpose of a WGS should be revealed to the patient;
- how to maintain the confidentiality of the genetic information revealed and protect the patient's privacy;
- whether WGS should be conducted in non-CLIA certified labs;
- whether the FDA should pass regulations permitting only physicians to request WGS;
- liability issues in product liability and toxic tort cases and in cases against physicians;
- social and ethical issues related to predicting an individual's predispositions and predilections.