Revving the Engine that Propels Us

by Gillian Parrish, Manager, Alliance Development and Communications, FasterCures

Over the past few decades, the U.S. has created and refined an extremely productive framework for developing new biomedical innovations and bringing them to the marketplace.  In fact, the biomedical sector directly and indirectly accounts for some 5 million U.S. jobs, including 1.2 million high-wage private sector jobs in pharma, biotech, medical devices, research and testing. 

However, for the first time since the 1980’s, U.S. preeminence in biomedicine is in jeopardy, vulnerable to falling behind growing powers in Europe and Asia thanks to regulatory, policy, and funding efforts in those nations that offer incentives for innovation and support entrepreneurship in a way the U.S. does not.

The history of our country’s rise to leadership in biomedical innovation, and a suggested roadmap for overcoming the factors that now challenge our dominance in the global market, is the focus of a new Milken Institute Study, The Global Biomedical Industry: Preserving U.S. Leadership.  Released last week at a Washington D.C. briefing co-hosted by the Council for American Medical Innovation, the report offers specific policy recommendations that will ensure the industry continues to grow and flourish here, including:

• Increase R&D tax incentives and make them permanent.
• Cut corporate tax rates to match the OECD average.
• Extend support for emerging biomedical research fields.
• Provide adequate resources for the FDA and NIH to expedite regulatory reviews and clinical trials.
• Leverage existing strengths in medical devices.
• Build human capital for biomedical innovation.
• Promote and expand the role of universities by adopting best practices in technology transfer and commercialization.

It makes a compelling case for action, and reinforces FasterCures’ long-held position that no one sector can do this alone.  It will take collaboration – universities and businesses, corporations working together, private equity entrepreneurs willing to invest in high-risk, high-impact R&D models, innovative intellectual property arrangements, etc. – to maintain U.S. leadership in biomedicine. 

“As recently as 1980, European companies dominated the industry,” said Ross DeVol, Chief Researchers at the Milken Institute. “Unless we act soon and act smartly, we’ll wake up in five to ten years with no products and nothing in the pipeline. This is our industry to lose.”

“The alarm this report sets off is very real,” said Jim Greenwood, President and CEO of the Biotechnology Industry Organization (BIO), at last week's event. “The fear that the U.S. is losing its competitive edge is well-founded.  If we want to have more cancer cures than iPads, we have to invest in them.”

Strengthening the biomedical sector should be central to our country’s economic platform. Government, particularly the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), should work with the private sector to get more and better therapies to patients. 

“The 21st century is going to be the century of life sciences,” said Stephen Ubl, President and CEO of the Advanced Medical Technology Association (AdvaMed).  “The question is: will it be made in America or somewhere else?” 

Many countries are building the infrastructure for biomedical research, and courting the companies who drive it.  Our current leadership is in question, and comes with no long-term guarantee.  But if the advocacy and policy communities can come together to tap into our nation’s most unique attribute – our collaborative, innovative spirit – we can take the decisive steps necessary to not only maintain, but grow our leadership in biomedicine.

 

Five reasons why you should be at Partnering for Cures

Partnering for Cures is an opportunity to find allies who can help you advance your medical research goals, and a platform for solutions needed to accelerate progress.

Five reasons why you should register now and be in New York City on November 6-8, 2011:

1. Find the partner who could make a difference. Whether you're looking for an investor or a collaborator, a customized partnering system allows you to easily find potential allies from all sectors of medical research.
2. See collaboration in action. Collaboration is the name of the game at this meeting. Hear case study presentations from 30 cross-sector programs with innovative paths to advance their R&D goals. A unique opportunity to get a pulse on trends and best practices in real time.
3. Problem-solve with medical research leaders. Every panel discussion will be focused on addressing some of medical research's top concerns in concrete ways. The goal of each panel is not just to inform (or entertain) – but to leave you with real transformative ideas you can run with, best practices you can emulate, and lessons learned you can build from.
   

   Diverse and dynamic panels include: • Regulating Risk: Clearing New Pathways for Progress at FDA
   • Streamlining Drug Repurposing and Approval
   • Philanthropy As Risk Capital
   • Business Is from Mars, Academia Is from Venus
   • Intellectual Property: The Lawyers' Debate
   • Staying Afloat in a Sea of Data
   • Convergence: The Death of Disciplinary Science
   • And many more

4. Get expert advice to help you make informed decisions. Whether it's structuring collaborations or deals, developing marketing strategies, or crafting appropriate intellectual property policies, experts will be available on-site for one-on-one, free consultation sessions to help address your challenges.
   

   Experts on-site to provide you with pro bono analysis and feedback on:• Strategic planning
   • Structuring deals and collaborations
   • Attracting philanthropic capital
   • Communications and marketing
   • Interacting with the FDA

5. Share your ideas openly. Expand your echo chamber and significantly increase your friends and family list. Speeding medical progress requires unconventional approaches from nontraditional allies. Partnering for Cures provides you with a safe haven to explore the possibilities.

For more information, visit www.partneringforcures.org.

RELATED CONTENT: Podcast of BIOTech Now interview with FasterCures executive director Margaret Anderson on the importance of partnering across sectors.

FDA’s Work IS Essential

By Margaret Anderson, Executive Director, FasterCures
“The U.S. Food and Drug Administration provides essential benefits to consumers and patients, and there is no backstop, no other agency, that performs this critical work.” –Alliance for a Stronger FDA* ad, September 2011
No other agency touches American lives daily – consider the products we consume the first 30 minutes of each day– the list of products regulated by this one agency is long. And yet, despite such big responsibilities, its budget has not always kept pace.

As the US Senate Appropriations Committee considers funding levels for the agency. Here’s hoping that the Committee consider the vast needs of this chronically underfunded, but critically important entity:

- Increased funding at the FDA will allow the agency to bolster its scientific infrastructure and expertise and be able to more effectively take the baton of medical innovation from the medical research community and bring it into the marketplace, where scores of patients impatiently await access to potentially life-saving therapies and life-enhancing scientific advances.

A few weeks ago, FDA provided details on exactly how it will go about doing this through its strategic plan for regulatory science. Priorities include stimulating personalized medicine programs, developing medical countermeasures to protect threats to global security, and ensuring agency capability to evaluate new, innovative technologies.

In an editorial in The Wall Street Journal, FDA Commissioner Margaret Hamburg said that the agency is “actively taking steps to improve the clarity, consistency and predictability of our regulatory systems. At the same time, we are working with the broader scientific community to address issues of scientific uncertainty that slow medical-product development and review.”

- Increased funding at the FDA will support industries that are essential to our nation’s growth and prosperity and create high-paying American jobs. Consider that the bioscience industry, which depends on FDA for regulation of its medical device and therapeutic products, directly employed 1.42 million people in the United States in 2008 and generated an additional eight million related jobs**.

An FDA that’s functioning at peak performance can spur economic growth and accelerate the process of bringing to market promising breakthroughs that can help improve the quality of life, and even save lives.

*FasterCures is a member of the Alliance for a Stronger FDA. Margaret Anderson, FasterCures’ executive director is the Alliance’s vice president.
** The U.S. FDA: A Cornerstone of America’s Economic Future

New Opportunities for Patients to Engage

Patients are the most underutilized resource in medical research – and they have the most to gain. Two things this past month struck us as truly promising in the effort to build a research culture that encourages and inspires broader public participation.

1) The National Institutes of Health (NIH) invited patient advocacy and disease research organizations to partake in the strategic planning process for the NIH Common Fund. The NIH Common Fund was enacted into law by Congress through the 2006 NIH Reform Act to support cross-cutting, trans-NIH programs that require participation by at least two NIH institutes or centers and that can be achieved over a 5-10 year period. This program supported NIH initiatives in nanomedicine, molecular libraries and imaging, the Clinical and Translational Science Awards, and the newly formed Center for Regenerative Medicine.

With this request for input, NIH is seeking innovative and transformative ideas from the outside community to drive the formation of new programs to be funded in 2013. This provides an opportunity for patients to help NIH clinical researchers prioritize novel research ideas and collaborations likely to drive new solutions to medical challenges. NIH lists a host of concepts on which they are seeking input, from artificial organs to Big Data initiatives to mobile health technologies. With this announcement, NIH is providing the public with an opportunity to peruse its strategic planning site and share its thoughts on the Nation’s biomedical research plans for the future. It is NIH’s hope that public input will help drive innovative new ideas through the Common Fund.

2) The Department of Health and Human Services (HHS) recently slated an update of the requirements for protecting human subjects in research – referred to as the Common Rule. This update, the first major overhaul in the 20 years since the regulations were enacted, is designed to enhance protection for research subjects while and reducing administrative burdens, delays, and ambiguity for researchers. The Common Rule covers a number of areas specific to Institutional Review Boards (IRBs), which review the ethics of research, and the need to obtain informed consent from research subjects.

HHS is requesting input on a number of key provisions. The department is proposing to simplify the informed consent process and allow consent on biospecimen research to extend broadly to future new studies. Stored biospecimens are an important source of information and material for researchers, and the reuse of existing data and materials can be an efficient resource for a new research study. If appropriately modified, this rule would respect patients’ contribution to science, as well as their rights and welfare, while removing unnecessary timely and costly administrative burdens to researchers.

In addition, in terms of oversight, HHS is proposing to restructure the missions of IRBs by prioritizing their workload based on the risks posed to subjects. The department also is evaluating the use of a single IRB of record to oversee studies conducted at multiple sites. Such modifications could greatly improve efficiency in coordinating research studies.

In this new era of transparency, patients and the broader public have an opportunity to redefine their role in research by sharing their ideas for innovative models of research and sharing perspectives on how to improve the informed consent process. These two opportunities are opening the door to more meaningful public input in the medical research enterprise.

At FasterCures, we are strong proponents of working to improve our medical research system to better work for patients. Similarly, we urge the public, and especially patients, to become engaged, proactive participants in the process.

A few months ago, we released the report Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, which highlights how people affected by HIV/AIDS rallied together and created an advocacy movement that demanded change and got results. We have much to learn from HIV/AIDS activists, how they redefined patient engagement in the medical research process, and changed the drug development paradigm. Thanks in large part to the groundwork they laid, the public is increasingly viewed as an essential ally and partner in advancing medical research.

RELATED EVENT:

REGISTER NOW for a free FasterCures webinar Bits and Bytes: Making Patient Information Available and Meaningful to Researchers
September 16, 2011
1:00 - 2:30 p.m. (Eastern)

Three distinguished leaders will discuss opportunities for accelerating research through improvements in biobanking, the use of electronic medical records, and the release of personal health information for research. Don't miss this important discussion!

Speakers:

• David Blumenthal, M.D., Director, Institute for Health Policy, Massachusetts General Hospital and Partners Health System; former National Coordinator for Health Information Technology


• Carolyn Compton, M.D., Ph.D., Director, Office of Biorepositories and Biospecimen Research at the National Cancer Institute


• Sharon F. Terry, President and CEO, Genetic Alliance


• Moderator: Margaret Anderson, Executive Director, FasterCures

Fixes in Financing Needed to Bridge the Valley of Death

Turning medical science into medical solutions requires innovators to leap all kinds of hurdles, but one of the biggest is the financial “Valley of Death,” where many underfunded, early-stage ventures meet their demise.

To help bridge this funding gap, dozens of investors and innovators convened for a Financial Innovations Lab to explore financial tools and other ground-breaking approaches that are being used to advance initiatives in medicine and other industries. Organized by FasterCures; and the Milken Institute, the Lab was designed to explore whether these financial tools could be applied to translational medical research.

The Lab spotlighted models for partnering in research and development, and identified finance instruments and incentives, some of which are described below.

• Distributed Partnering Model utilizes product definition companies (PDCs) that identify and license promising early-stage assets from research institutes, manage product definition research, and sell de-risked assets to later-stage stakeholders. The PDCs make $3 million to $5 million investments aimed at attracting additional funding.


• BioPontis Alliance is a hybrid investment fund and product development company that aggregates technologies from a consortium of universities, screens these assets, develops them through a translational development network, and then licenses them to strategic pharmaceutical partners. Their activities are supported by a $50 million fund. The model encourages sourcing of early-stage assets from academia by pooling and sharing the value of intellectual property, and helps ensure later-stage funding by establishing relationships with pharmaceutical companies. This proposition is made more attractive to universities via a master IP licensing agreement where the process is expedited and universities receive a pro-rata share of the total value created by the assets.


• Fast Forward, a wholly-owned subsidiary of the National Multiple Sclerosis Society, is a venture philanthropy model that provides leveraged, philanthropic funding to translate academic research and further develop biotech research into new treatments. The technologies are identified and evaluated by scientific and business advisors. Fast Forward makes $250,000 to $1 million investments.


• Israeli Life Sciences Fund uses a standard venture capital-structured fund with the government and private sector as limited partners. The Israeli government, which takes the first loss before the other limited partners, has committed $80 million to boost returns for private investors.


• Flow-through shares, which originated in the Canadian resource industry, encourage higher-risk investments through tax incentives. Oil and mineral exploration companies issue these shares as a way to pass government tax deductions for exploration on to investors, effectively halving the risk of investment.

There was broad agreement that the medical research system has much to learn from financial models that have worked in other industries such as filmmaking, telecommunications, and oil and gas. But participants cautioned against simplistic analogies, noting the unique circumstances that define the medical research process. Science is unpredictable, and failure to fully vet a product has consequences that are measured in terms of life or death.