While the confirmation challenges continue, at least one high-level science and technology appointee is stepping down. Margaret Hamburg, Commissioner of the Food and Drug Administration, will step down at the end of next month (H/T ScienceInsider). Even with slightly less than two years remaining in the Obama Administration, there is a chance Dr. Hamburg’s successor may not be confirmed before a new President is sworn in. (I am encouraged by indications that a nominee has already been identified.)
Hamburg’s tenure is one of the longest in the agency’s history. When she took over, the agency was struggling, with ethical challenges over drug approvals. The agency is currently in the middle of regulatory changes for food safety oversight and medical device approvals, and it has not been perfect. There were complaints over the agency’s inaction over compounding pharmacies (which are not presently regulated by the FDA), and Hamburg was overruled by Health and Human Services Secretary Sebelius over Plan B emergency contraception (though the courts eventually sided with the FDA).
Since the 2013 decision in the Myriad case, which invalidated some patent claims on isolated sequences of two genes relevant in some cases of breast cancer, there have been two major actions taking place. Following the decision the Patent and Trademark Office (USPTO) has been working on guidance for potential patent applicants working with products of nature. The latest guidance memo was released in December (H/T ScienceInsider), and the USPTO is taking comments until March 15. There has been a back and forth with the biomedical research community, which has argued that the initial guidance (from March 2014) went further than the Supreme Court intended. The USPTO has made changes in response.
The other post-Supreme Court thrust has involved lawsuits by Myriad Genetics against companies developing their own genetic tests for the breast cancer genes where Myriad held patent claims. The company was protecting its market share and investment through the lawsuits and the settlement terms it has been able to reach.. ScienceInsider is reporting that many of the remaining cases have settled and The New York Times has noted that Myriad is working to address the remaining cases.
Amidst the laundry list that is the State of the Union was the Precision Medicine Initiative. In short, it’s a program geared toward expanding treatments crafted to address patients that don’t respond to treatments targeted to an ‘average patient.’ The initiative was announced with Bill Elder in the audience. Elder benefited from a ‘precision’ treatment for his cystic fibrosis. The cause of his disease is shared by only 4 percent of cystic fibrosis patients.
Details at the moment are scarce. The Federal budget may give additional details once it’s released next month. The program would likely be led by the National Institutes of Health, though the focus of the Initiative would not necessarily be restricted to medicines, wider use of DNA testing to help craft cures or similar clinical treatments. Refinements in medical imaging can be helpful in this area and health information technology may also be leveraged to develop new cures that can assist those not helped by treatments geared toward the ‘average’ patient.
Setting aside the usual concerns about getting approval for new initiatives in a tight budgetary environment, it remains to be seen if this program can successfully be sold to the general public. If the focus is on treatments focusing on the margins, it could be hard to sell the benefits to a broader public. Of course, if there are members of Congress with direct experience benefiting from targeted treatment, then the acceptance of this program might be easier to get. Unlike the BRAIN Initiative, focused on better understanding of something we all have, the Precision Medicine Initiative runs the risk of being a niche program, not likely to get the large investments it might need. If it follows the organization of the BRAIN Initiative, including several agencies and a few public-private partnerships, the total investment might be spread around sufficiently to mitigate the political challenges of getting lots of money.
The Institute of Medicine, part of the National Academies, has been working on a report outlining recommendations on clinical trial data sharing (H/T The Scientist). The report comes from the perspective (which I share) that there are benefits to making more clinical trial data publicly available. Released on Wednesday, the report recommends a multi-stakeholder process to establish the policies, culture and infrastructure that will be necessary to encourage and facilitate sharing this data.
I can’t emphasize enough the need to change the culture surrounding the (lack of) disclosure of trial data. Ben Goldacre has been working hard for years to encourage the release of clinical trials data, and he and the others supporting AllTrials.net certainly welcome the recommendations in the report (AllTrials wants all past and present clinical trial data to be registered and their full methods and summary reports reported). This report recommends disclosing the full trial data set (with metadata) within 18 months after study completion, and the trial data associated with any publication results should be released within 6 months of publication.
The report identifies current gaps in the clinical trial data environment that need to be filled in order to facilitate more data sharing. Current infrastructure needs to be improved to more efficiently store and manage the breadth of data. Technology is, at present, is not interoperable enough, to facilitate data sharing, nor are existing systems easily searchable or discoverable. There aren’t enough workers trained in data sharing to meet the increased demand, and more equitable cost-sharing mechanisms are needed in order for those burdens to not prevent participation
Increased data sharing can provide a greater return on investment, improve the drug development process, facilitate innovation and reduce redundant investigations. The benefits of making this information widely available outweigh the advantages some parties enjoy from keeping information to themselves. But making such sharing easier will take time. The sponsors of the report (a combination of global pharmaceutical companies, foundations and biomedical agencies from the U.S. and the U.K.) should start working on how to best implement its recommendations.
In what appears to be part of its response to the problems federal labs had this summer controlling pathogens, the Centers for Disease Control (CDC) is looking to hire a person responsible for lab safety agency-wide, with an emphasis on pathogens. Reuters, which broke the story, indicates this was recommended after an agency review following this summer’s incidents. Adding to the urgency, last week a CDC technician may have been exposed to Ebola.
While the hiring search is handled by the CDC head of Infectious Diseases, CDC microbiologist Leslie Dauphin will serve as acting lab safety head. She will likely expand safety training and explore other avenues for improving safety.
The United States Patent and Trademark Office (USPTO) has released another guidance memo for patents derived ‘from nature’ (H/T ScienceInsider). The USPTO released its first memo in March, and between negative public comments and additional court action, releasing new guidance makes sense to me.
It also means that with additional court action likely in the near future, this new memo is unlikely to be the last one. If you need an additional reason, the planned January meeting hosted by USPTO should suffice. Comments are being accepted on the new guidance until March 16, and the public forum scheduled for January 21 will provide additional opportunity for comments.
The new guidance uses a two-step process for determining patent eligibility. Is the claim directed to a law of nature, a natural phenomenon, or an abstract idea? If so, then it is not eligible, unless there are additional elements cited in the claim that amount to significantly more than the law, phenomenon or idea. To help provide additional detail, the USPTO is developing examples.
One of the atmospheric touches early on in Interstellar (no spoilers, I promise) involves Cooper (Matthew McConaughey) and his kids chasing a rogue unmanned aerial vehicle until they are able to take control and land it. They then cannibalize it for parts.
While there is not yet a flock of rogue drones wandering through the air, it’s reasonable to think that eventually some UAVs will crash in places where it won’t be so easy to recover them, in whole or in parts. Absent some spy-level self-destruct magic, one method of dealing with this future debris is to make it biodegradable.
One group working on this for drones is competing at the International Genetically Engineered Machine Competition (yeah, I didn’t know this was a thing either). They have found a company that uses mushrooms to form the bulk of the drone body. Using other natural materials, such as cellulose and proteins from wasp spit, the bodies of these drones are both biodegradable and water resistant.
Now, there are still several parts that are non-biodegradable, but work is proceeding on making biodegradable versions of sensors, rotors and other parts to make a completely biodegradable craft. Besides minimizing the environmental impact of crashing electronics in the wilderness, these craft can also make it harder for technology to fall into the ‘wrong hands’ – unless they get to the craft really fast.