Tag: and Emerging Biotechnology

In one of my first blog posts, I mentioned that the Office of Science Policy (OSP) works with various stakeholders within and outside of NIH to develop policies that promote progress in the life sciences. These stakeholders can be local, state, federal, and international partners, research institutions, private companies, as well as non-governmental organizations. Building and fostering collaborative relationships with these groups gives NIH the opportunity to join forces with other experts and better use the biomedical research enterprise to improve public health, lengthen life, and reduce illness and disability.

Here, I want to spotlight a specific collaborative relationship that OSP has facilitated in recent years with the Patient-Centered Outcomes Research Institute (PCORI). Congress established this independent, nonprofit, non-governmental organization through the Patient Protection and Affordable Care Act of 2010. PCORI supports comparative effectiveness research, which is  aimed at improving the quality and relevance of available clinical evidence. These findings will help patients, care givers, advocates, policy makers, and public health officials make better health care decisions.  With their charge defined, Congress encouraged PCORI to work with NIH to manage the funding and conduct of research when needed and appropriate.

OSP works with the various NIH Institutes and Centers (ICs) to help encourage information-sharing and professional consultation with PCORI when a need arises. When these interactions lead to an opportunity for a collaborative research activity, OSP serves a central role at NIH to ensure that collaborations run smoothly and efficiently, from the earliest phases of concept development and throughout the life of the supported research. Specifically, OSP facilitates the agreements between the ICs and PCORI that establish the operational framework for the proposed collaborations. As such, OSP led the NIH efforts to develop this framework, which ensures that collaborative projects follow the policies of both organizations and provides guidance on initiative development, legal affairs, financial management, communications, and extramural activities.

Two formal NIH-PCORI research collaborations are now underway. For these activities, PCORI provided the research funding and the NIH ICs administered the resulting initiatives. The operational framework developed by OSP continues to facilitate communication between both organizations during these initiatives. The first of these joint projects, in 2014, was the National Institute on Aging Falls Injuries Prevention Partnership. This $30 million, five-year project supports a clinical trial to test individually tailored care plans in order to help reduce serious fall injuries, a common source of disability and death among the elderly. Another major collaboration involves the National Heart, Lung, and Blood Institute and National Institute of Neurological Disorders and Stroke jointly administering the Hypertension Disparities Reduction Program Partnership. This $23 million program is supporting two activities—awards announced in September 2015—performing randomized trials comparing evidence-based approaches for improving hypertension control in high-risk populations.

Collaborations like these represent exciting opportunities to address complex real-world public health issues. This important research has the potential to generate information that will help patients and care givers make better informed health care decisions. To this end, OSP will continue working with our colleagues at PCORI and leveraging similar approaches to collaborate with other like-minded organizations supporting biomedical research.

In September 2015, I wrote about the U.S. government’s deliberative process to re-evaluate the risks and benefits associated with certain “gain-of-function” studies involving pathogens.  Such studies are undertaken to better understand how pathogens infect people and spread through the population. These studies help assess the risks associated with emerging pathogens, particularly those like new influenza strains that may have pandemic potential.

However, these studies can also generate pathogens with enhanced characteristics, such as pathogenicity (ability to cause disease) or transmissibility (ability to spread) in mammals.  Concerns have been raised about whether a modified laboratory pathogen could be accidentally or intentionally released from a lab, threatening local or even global populations.  Some have questioned whether such studies are worthwhile.

The National Science Advisory Board for Biosecurity (NSABB) has been examining the issue and on January 7 and 8, on the NIH campus, the Board will convene to discuss the risks and benefits, as well as ethical and policy issues.  To inform NSABB deliberations, NIH commissioned a thorough risk and benefit assessment and an ethical analysis—the results of both will be presented for the first time at the upcoming meeting.  NSABB will also begin discussing its preliminary findings and a potential path forward for funding and conducting gain-of-function studies in pathogens with pandemic potential.

The meeting is free and open to the public.  It will also be webcast and questions during the meeting can be submitted to the NSABB inbox at NSABB@od.nih.gov.  Written comments on the topic can also be submitted to NSABB at the same address. See here to register for the meeting and to learn more.

The gain-of-function debate comes at a time when we are having similar discussions about how to rapidly and responsibly advance new biomedical technologies to promote public health and well-being. It raises difficult policy questions about how to maximize the potential benefits of some areas of scientific research while mitigating potential risks.  I hope you can join us for what should be a very interesting meeting.

‘Twas the night before Christmas, and at NIH,

There were still policy issues, keeping us wonks awake.

The sIRB comments have been read with care,

Soon a final policy will be ready to share.

The Common Rule comments are still flowing in,

Thoughts on consent forms for biospecimens.

NSABB staff are busy, organizing fora to discuss,

The Gain of Function studies that have raised such a fuss.

And I at my computer, and staff at theirs, too,

Are pushing along the CT.gov final rule.

Precision medicine and chimeras are both front of mind,

Biosafety compliance is not far behind.

The new Strategic Plan raised a small clatter,

As everyone read it to find what really matters.

Data sharing policy, don’t forget about that,

Even as we update our disease burden stats.

The Biennial report is soon to emerge,

Full of good info – read it, we urge!

The RAC staff is ready, FR comments in hand,

To implement the streamlined process we planned.

Then what to our wondering eyes should appear,

But an omnibus bill for this fiscal year!

And as OSP Director, true policy geek,

I keep policy wheels rolling, week after week,

“Now, consent! Now, DURC! Now, gametes and eggs!

On NPRM! On agencies and regs!

To extramural and intramural, and all in between,

To new science policy in 2016!”

The Office of Science Policy (OSP) has added technology transfer and innovation policy to its scope of advice and support to the NIH Director on issues affecting the biomedical research community, the NIH, and the public.  As described previously, OSP participates in the development of new policies and programs in a variety of arenas that span the research continuum from basic laboratory science, through translational and clinical research, and from domestic to global public health.   With the addition of tech transfer policy to our portfolio, OSP staff now collect data and assess activities involving the commercialization of technologies based on NIH funded research.  Ongoing research programs invariably require the application of policies related to technology transfer. In addition, the advent of new technologies and collaboration paradigms raises questions and may merit application of new policies.

For example, in receiving a material from a third party, what level of restrictions, if any, is NIH willing to accept on its use, the publication of research results, and ownership of newly developed materials?  If a new inventive technology arises, will it be patented, what institution(s) owns the patent, and who has rights to use it for research or commercial use?  These are typically fact-specific questions made by NIH Institutes and Centers (ICs) and grantees.   However, policy guidance shapes where the lines are drawn and when.  Access to new technologies and creative collaborative arrangements may require creative applications of existing policies or the development of new ones.

While taking on a leading role within the Office of the Director, OSP works closely with the Office of Intramural Research (OIR), the Office of Extramural Research (OER), and ICs on policy issues within their respective domains.  In the past, technology transfer policy issues have included arrangements for the receipt and distribution of approved stem cell lines, patenting of genomic technologies, and the restrictions under which samples of pandemic flu strains would be distributed internationally to researchers and organization preparing vaccine seed strains.  The reorganization of technology transfer at NIH and the introduction of staff focused on technology transfer in OSP have led to the newly created Technology Transfer and Innovation Policy Division.   Among our areas of current interest: the evolving nature of complex prognostic tests requiring greater clinical development prior to commercialization and an analysis of drugs and biologics approved for precision medicine applications.

This staffing of expertise within OSP will benefit the OD and the NIH community in strengthening the management and oversight of policy functions and the programs that provide vital support to the NIH community on matters involving technology transfer and innovation policy.