Science takes center stage at world's top finance conference

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The long-term effects of sequestration on medical research

“I worry about the treatments that we’re not going to discover as a result of these cuts. What does this mean downstream; what does it mean in terms of the unmet needs of patients who have diseases with no treatments or cures? The NIH investment is a very powerful driver to this entire enterprise.” 

- Margaret Anderson

Last week, FasterCures Executive Director Margaret Anderson appeared on the BioCentury broadcast with host Steve Usdin to examine the long-term ramifications on medical research and health outcomes if sequestration is not appealed. Watch the whole segment here. Anderson was joined on the Sunday, March 10 segment by Lynn Marquis, Director of the Coalition for the Life Sciences, who talked about the effects that these cuts will have on the scientific workforce.

“We’re going to lose an entire generation of scientists moving forward,” Marquis cautioned. “We’re already not seeing kids pursuing scientific careers because there is no future for them.”

Though optimistic about the possibility to reverse this alarming trajectory, Anderson and Marquis warned that medical science is an area in which the United States cannot afford to fall behind.  They emphasized the outsized impact that investments in research have on both human health and the health of our economy, and noted that it is hard to compare these types of cuts with others.

“This is a powerful investment in the future - for patients, for the economy, for jobs, for industry. I don’t think it’s the same as cutting other programs,” Anderson said. Marquis agreed and offered a bit of hopefulness, “I think we see from both Republican and Democrats…there is huge support for NIH’s biomedical research, primarily because of the human health and economic benefits that we reap from our investment.”

So if Congress understands that medical research is vital to the future of our country, what can be done to ensure we don’t lose our preeminence in the life sciences?

Anderson thinks there’s hope for the NIH to avoid budget-desolation. “I’m an optimist. I think that people need to speak their mind to their members of Congress about how they feel about this investment.” She also spoke about the power of team science and cross-sector collaboration in moving promising research from lab to patient faster.

Just this year, NIH will be forced to reduce its spending by $1.6 billion, significantly slashing the United States’ ability to fund basic scientific research.  Yes, that’s billion with a capital B. Devastating to the medical research community – and more importantly, to the hundreds of thousands of patients suffering from diseases for which there are no cures and few treatment options – does not begin to describe the impact of sequestration.

What Can Be Done?

• Contact your Members of Congress and let them know that further cuts to the NIH and FDA will set us back on the path to new therapies and cures for disease, jeopardize our economic competiveness, and result in job loss in communities throughout our country. Share with them why a strong investment in medical research is important to you, your family, and your community.
• Submit your story about why medical research and protecting funding for new research matters to Time=Lives.
• Attend the Rally for Medical Research on April 8th here in Washington, DC. Even if you can't attend, check out their site for more way to get involved!

Needing teamwork for translation

Science is like football, said National Center for Advancing Translational Sciences (NCATS) Director Chris Austin during the Partnering for Cures panel, “Molecule to Man (and Woman): Speeding Translation to Human Trials.” Translational science needs true teamwork, everyone in the ecosystem coming together – researchers, pharmaceutical companies, venture capital firms, patients groups, and government – in order for new treatments to be brought to trial faster. But, “the incentive has always been for individual achievement,” Austin said. Instead of playing football, the scientific community is playing golf. And many hope the creation of NCATS will help change that.

“NCATS is a different beast,” Austin said. “It was set up as a collaborative instrument. It’s founding principle is that we have to do these very, very important things that no one else can do.” 

Anna Barker of the Arizona State University School of Life Sciences hopes that mission includes creating systems to speed up translation. “We haven’t thought about creating an end-to-end system that starts with the patient and ends with the patient,” she said. And a key to this system is requiring high quality at every level, for which she feels the scientific community needs standards. “We have not thought about how to deliver molecular findings to patients in a high-quality, controlled way,” she said. 

Todd Sherer of the Michael J. Fox Foundation advised caution. “The challenge is finding the balance between overstandardization vs. innovation,” he said.

Some steps that could speed translation involve solving problems that have no return on investment, so it is difficult for researchers or big companies to justify using the resources. “How we use our resources has to be ever more strategic,” said Dale Edgar of Lilly Research Laboratories, who said his company is pursuing success by identifying “pillars of excellence,” such as maintaining quality standards of practice for the data it stores.

Research decisions can’t be made in a vacuum, said Stelios Papadopoulos of Exelixis, especially not in an investment vacuum. Decisions still must be made to get the maximum return on investment. “I have not seen a compelling investment proposition to go in and engage in [translational research],” he said.

NCATS, which is free from needing to find a cure for a particular disease or make a particular return on investment, can help, said Austin. He feels this gives the agency an obligation to work on the general issues that will help advance translation.

The panelists agreed that true collaboration is key. Every stakeholder has to go “all in” for the collaboration to work, cautioned panel moderator Margaret Anderson of FasterCures. Edgar pointed to the Innovative Medicines Initiative in Europe as a good example of all stakeholders working together to find and overcome critical gaps in driving innovation to the patient. He said the exciting thing about NCATS is “thinking about the patient as the end point and not the next grant.” Innovation, he said, “requires a line of sight to the patient from the earliest point.”

“Something NCATS brings to the table is leadership, galvanizing the industry and all of the players,” said Sherer. He advised Austin to plan for some early accomplishments in the next 18-24 months to prove to naysayers that the agency can affect change.

Edgar encouraged NCATS to continue to foster partnerships, because “great things will happen.”

“The way you make these things work increasingly is to be inclusive,” said Barker. “Get the best input you can up front from as many in the community as you can. Get as much buy-in as you can.”

And Austin is doing just that at NCATS. He reported that he is in talks now with stakeholders in “concentric circles” in the process, including institute directors, academic institutions, patient groups, pharma, biotech, and VCs. As the agency develops, his goal is to keep the patient at the center of everything. “Having patient involvement helps you stay on the critical path,” he said.

Anderson concluded with emphasizing the important role that all stakeholders need to play in medical research. “It is unrealistic to think that NCATS can do it alone. We’ve got to do this together.”

The FDA: Balancing innovation and safety

If you talk about the need to speed up the approval process for new drugs and devices, the U.S. Food and Drug Administration is often the first criticized for the time lag to new treatments. But, as the Partnering for Cures session, “What You Probably Don’t Know about Innovation at FDA” made clear, the agency is trying to become more innovative and approve new treatments more quickly and efficiently, but there are obstacles.

The biggest danger, said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, is “where public criticism and controversy outweigh science.” Deliberations should be made based on the latest science, she said, but controversy affects how the agency works and creates what she called a “circling of the wagons,” a “let’s not be too innovative” mindset.

She noted that the agency approved 10 cancer drugs in the last fiscal year and is on track to do so again this year, but it is work the FDA should not be doing alone. “It is the job of the larger biomedical community in concert with FDA” to get new drugs on the market, she said.

Some drug makers are wary of using innovative approaches with the FDA. “Once we get to the clinical study, if we want to be innovative, we have to do the traditional approach alongside, sort of carrying two tracks to get the product to market,” said Joseph DeGeorge of Merck Research Laboratories.

Many challenges to new drug approval start well before a drug finds its way to the FDA. Jonathan Leff of Warburg Pincus noted that venture capital investment in life sciences has been decreasing dramatically, and in the first nine months of this year, the number of first-time companies receiving venture capital investment was the lowest it has been since 1995. Those venture capital firms that are investing, he added, are shifting their investments to other countries or in less innovative products. “We have tremendous opportunity to translate science that is happening into cures, but the system isn’t working as well as it needs to,” he said. “We are not getting the throughput. We need to address the issues of translating scientific discoveries to new therapies.”

Some stakeholders are finding ways to assist the FDA in the process. Richard Insel of JDRF said his organization decided it had a role in educating the agency in novel treatments and their development. And when JDRF wanted to work on an artificial pancreas, there was no regulatory guidance. So with the FDA’s knowledge, the group convened opinion leaders and created draft guidance that was submitted to the FDA. This helped the FDA draft its own regulatory guidance for the artificial pancreas, which just came out in final form.

Frank Douglas with Austen BioInnovation Institute said his organization decided to bring the FDA in earlier in the process and found that things went smoother. “We spent a lot of time trying to figure out how to approach the FDA to make them feel they were in a safe harbor for collaboration,” he said.

The panelists agreed that bringing patient foundations, as well as the patients and families, into the regulatory process to discuss risks and benefits of individual drugs is vital. “We need to hear from affected communities about what tradeoffs they think are acceptable,” said Woodcock. “That should be our guiding light.”

So what would help the FDA improve the process, moderator Margaret Anderson of FasterCures asked the group.

The innovative spirit that is evident at the highest levels of the FDA need to be transitioned to the practitioners of the review process, said Insel.

Douglas noted that as the FDA collaborates more, it should identify other bodies that can be brought in to help with reviews.

Woodcock noted that the workload at the FDA continues to be a problem. It is hard to feel innovative when you are facing a mountain of paperwork, she said.

Anderson noted that the Alliance for a Stronger FDA, which she urged attendees to support, is focused on increasing the agency’s budget.

One panelist answered the question with praise. “The FDA environment, in terms of helping to advance and stimulate innovation, is better today than it has been in a while, and that is a credit to the leadership,” said Leff.

Novel Funding for Bioscience Companies

By Karlee Stewert, Communications Coordinator at FasterCures 



FasterCures Executive Director Margaret Anderson joined the Mid-Atlantic Bio Conference this fall for a discussion on novel funding sources for bioscience companies, a major concern plaguing the biotech and medical research industries. The panel discussed best practices to attract funding, how to develop important relationships with possible funders, and the opportunities crowdsourcing can bring to a small organization. “The biggest concern plaguing bioscience industry is funding,” moderator Peter Ginsberg, North Carolina Biotechnology Center’s VP for Business and Technology Development, pointed out, “the way a company approaches funding is the key to bringing in resources.”

“Building relationships is an obvious but important move,” Anderson recommended. “Start a conversation with a potential partner about something innovative you can do together.” Fellow panelist, John Hollway, president of Pragmatos Consulting, emphasized Anderson's point and warned that "the time to build a relationship with the federal government isn't when you’re applying, it's before." 

Crowdfunding, rallying support from others who can pool their resources rather than seeking out a major donor, has quickly become one of the most popular methods for small businesses to fund their endeavors. This approach to funding has permeated the biotech industry with organizations like Cloud-Based Quality Assurance for Cancer Treatment and myHealthPal, which use the vast opportunities the Internet provides to build their vision, raising funds and gaining access to their target audience.

Research organizations seeking funding from the National Institutes of Health (NIH) for their projects may unfortunately  be faced with the reality of sequestration of critical capital to create life-saving products and treatments. If sequestration goes into effect in January 2013, NIH will face a drastic funding cut. Anderson stressed the importance of scientists and biotech speaking out and making their stories heard by those in Congress: “I encourage you to understand what this means and make your voice heard. Talk to your members about what they can do and why sequestration matters. Speak to Congress about what it means to be a scientist, and tell them how much of an investment the federal government has made in you.”

To start to compensate for the uncertainty of federal funding, venture philanthropy groups will be looked on even more to step up and provide funding that the medical research community now relies heavily on.

Anderson recommended utilizing your members’ extended networks – both on and offline, ‘friends-of-friends’ connections —  building strong relationships with the funders can mean the difference between struggle and survival for your company.

And because this IS a FasterCures blog, we’d be remiss not to remind you of a great opportunity to find nontraditional allies who could be key to helping you advance your R&D goals – Partnering for Cures. This meeting, to be held November 28-30 in New York City is a unique opportunity for biotechnology companies to network and find true partners. This meeting brings together more than 800 leaders from all sectors of the medical research enterprise, across therapeutic areas. It is focused on solutions and making collaboration happen - and it works because the participants are there to get things done. A special rate is set for start-up and emerging biotech companies.

For more information on unique approaches to funding, check out these resources:

• FasterCures’ Fixes in Financing
• Crowdfunding: Maximizing the Promise and Minimizing the Peril
• crowdsourcing.org