Working together towards cures for rare diseases

By Karlee Stewart, Communications Coordinator at FasterCures

7,000 different types of rare diseases and disorders

          1 in 10 Americans suffers from a rare disease

                    95% of rare diseases do not have a single FDA approved drug

                                        The numbers don’t add up.

“Take on the voice that I’m sharing with you today, reach out to your fellow patient advocates who frequently don’t see beyond their own situation and help them understand the bigger systematic challenges we all face.” – Hugh Hempel, The Addi and Cassi Fund 

On February 28, 2013 we joined friends and colleagues in the rare disease community to commemorate Rare Disease Day, the culmination of a week's worth of activities to bring widespread recognition of rare diseases as a global health challenge. 

This year, we were lucky enough to be a part of a major event here in DC kick-starting the week and celebrating the screening of Here.Us.Now a documentary highlighting the Hempel Family and their struggle to save their twin daughters, Addi and Cassi, who are living with Neiman Pick Type C. To learn more about the Hempel’s journey, check out their Time=Lives story.   

The documentary focuses on the pitfalls of an ineffective system for patients with rare diseases who have hit a wall in the search for treatment options or a cure. It features prominent members of the medical research community from academia, pharma, biotech, and patient groups calling for change in this uncommunicative and slow system.

Panel moderated by Margaret Anderson

Following the screening, we heard from National Center for Accelerating Translational Science (NCATS) Director Chris Austin during a panel moderated by Margaret Anderson. “The bad news is that there are at least 5,000 more disease like [Addi and Cassi’s] that when doctors like me see them in the clinic, all you can say is please come back to me when you need supportive care.”

Answers are what the Hempels, and others like them, are looking for. However, because rare disease research today is so siloed and disease-specific, communication between groups is limited, leading to occasional duplication of research efforts and time wasted in a world where there’s no time to waste.

“Reach out to your fellow advocates and rally them together for rare disease research,” Hugh Hempel urged the panel’s audience, “We find ourselves fighting over crumbs from funding from the NIH…let’s work together.” 

Hempel said the biggest issue that smaller, disease-specific groups have is not working together for one ultimate purpose, cures.

Besides an insightful talk and plans for collaboration between advocacy groups, here are some outcomes from Rare Disease Week: 

• Reg4All – a “community of people storing and sharing information about ourselves, our health, and our diseases– to improve health and speed therapies across all disease.” An award granted by the Sanofi US Collaborate Innovate challenge that allowed the Genetic Alliance, CFIDS Association of America, National Psoriasis Foundation and the Inflammatory Breast Cancer Research Foundation to build the registry. 

• Boehringer Ingelheim collaboration with PatientsLikeMe on the rare lung condition idiopathic pulmonary fibrosis (IPF.) The partnership will provide “a customized experience for its users that will allow them to monitor their health and progress over time and connect with other to learn more about the condition.” 

• United States Senate agreed to resolution designating February 28, 2013 as Rare Disease Day

FasterCures was pleased to participate in RDLA's 3rd Annual Rare Disease Cocktail Reception and Movie Screening

Make sure to join FasterCures on Wednesday, March 20 for our FREE Webinar, Building New Patient-Centered Research Networks: The T1D Exchange and Registries for All Diseases and learn about: 

• The T1D Exchange, which consists of an integrated clinic network of more than 65 clinics across the U.S., a biorepository, and Glu, an online community with mobile capabilities for people touched by type 1 diabetes. Its mission is to improve outcomes in the community by facilitating better care and accelerating new therapies on the path to a cure.
• Registries for All Diseases (Reg4All), which recently won the Sanofi Collaborate/Activate challenge – a cross-disease, crowdsourced registry that promises to break down the silos between diseases and gather information from patients in a standardized way.

The World According to BIO CEOs

By Kristin Schneeman. Program Director, FasterCures

We look forward every year to attending the BIO CEO & Investor Conference in New York in February, because it gives us a window into what the issues of interest and concern to the biotech community are, as we’re planning our own activities for the year.

Here are a few nuggets picked up at this year’s conference:

• Reimbursement continues to be a key and thorny issue – and not everyone in the industry is dealing with it.  Companies and deal-makers noted that even in early-stage programs, commercial considerations are “at the table,” and that today you need to have not only a biological and clinical hypothesis in early research but a commercial hypothesis as well.  However, almost immediately after a fascinating panel discussion about “Reimbursement in an ACA World,” which catalogued some of the fundamental ways in which the world of not just healthcare but research is changing, a panel of very accomplished investors was asked how reimbursement factored into their world view right now – and they all fell back on the conventional wisdom that “truly innovative products will always get paid for.” One investor in a later panel mentioned that “the Street is ‘catching on’ to reimbursement” – just catching on?


• Everyone seems to love the FDA this year.  Investors described the regulatory environment as “favorable,” friendlier,” and companies praised the new accelerated approval and breakthrough designation initiatives – while noting that they remain something of a “black box.”  


• Rare diseases are hot, with companies and investors.  They see that these products can command high prices.  Personalized medicine is causing common diseases to be redefined as subtypes that in many cases could be considered “rare,” and clearly companies were embracing this line of thinking.  Hepatitis C, on everyone’s lips last year, was almost nowhere to be found – instead we heard about multiple myeloma, lysosomal storage disorders, and “genetically defined cancers.”  This is great news for patients with rare diseases, but how long will the fad last?  What happens to patients then?  And, as one speaker noted, “just having orphan designation isn’t a get-out-of-jail-free card on pricing anymore.”


• China is attracting not just big pharma companies but smaller biotechs as well – and not only to take advantage of cheaper gene sequencing or clinical research capacity, but to manufacture and sell products as well as to raise capital.  While doing business in China is not an easy row to hoe, you could hear the excitement of the panelists talking about this new frontier for U.S. companies.  Many U.S.-educated Chinese scientists and businesspeople are returning home to make their careers.  Will development of the academic infrastructure to fuel homegrown science and companies be far behind? 


• Sadly, there was almost no discussion about the impact of impending budget cuts on NIH, which fuels much of the science these companies thrive on, or FDA, which must have the capacity to regulate and approve their products. Their own user fees, which go directly to FDA review of their products, are threatened with sequester – where is the outcry?  Besides a promising few talking about the value of these agencies, the policy agenda seemed largely focused on repealing IPAB, the Independent Payment Advisory Board, from the Affordable Care Act.  

As always, an informative and thought-provoking two days, interspersed with fascinating glimpses of the products coming down the pipelines of dozens of companies.

Relevant postings:
--- Medical research delivers cures, saves lives and – oh, by the way – is pretty great for our economy too
--- Cutting-Edge Science, Collaboration, and Sustained Funding Needed to Get New Medicines from Lab to Patient
--- The Next Big Thing In Biotech: BIO CEO Conference
--- Are Drugs for Ultra-Rare Diseases the Future of Biotech? Francois Nader of NPS Weighs In

The election’s impact on medical research

The Partnering for Cures panel “Election 2012: What Does it Mean for Medical Research?,” offered an inside look into the implications of the November election outcomes for biomedical research and innovation. In an animated discussion, panelists covered some of the hottest issues, ranging from funding for the Food and Drug Administration (FDA) and National Institutes of Health (NIH) to sequestration to the importance of maintaining U.S. competitiveness in healthcare innovation.

Neera Tanden of the Center for American Progress emphasized the need for Americans to maintain their competitive, innovative, and economic edge in the healthcare sector. In recognizing that “the president is extremely mindful of NIH’s role and its role in America’s competitiveness,” Tanden identified not only the need to solve the fiscal crisis, which has created a “high level of uncertainty that is unhelpful,” but also the strong connection between the budget and long-term American competitiveness.

Cheryl Jaeger of House Majority Leader Eric Cantor’s office drew an optimistic picture of possible bipartisan actions that could take place with the new Congress that takes office in January. Jaeger argued that while opinions may differ on entitlement spending, “there really is bipartisan agreement on the importance of funding NIH and FDA. If there is one area where both sides of the aisle come together, it is on medical research.” The challenge lies not in realizing the crucial necessity of these organizations for medical research, but in understanding how “we can grow the economy and how to make sure that individuals recognize that these programs are the highest priority within the funding infrastructure.”

Wendell Primus of House Minority Leader Nancy Pelosi’s office focused largely on the government’s need to raise more revenue in general and to take care of the large wave of baby boomers that are set to retire in the next decade. “We are not going to take Medicaid and Medicare spending down enough to increase NIH budgets – it ain’t going to happen. If we don’t raise revenue, the NIH budget will continue to go down in real terms,” he argued. For Primus, Obama’s re-election has meant the chance to raise government revenue and bring the NIH budget back to a place where it is not losing value due to stagnant funding levels and a loss of purchasing power over time.

Scott Gottlieb of the American Enterprise Institute pointed out that company formation has been limited, VCs are consolidating, and funds for startups are beginning to dry out. Gottlieb argued that the NIH is only part of the ecosystem when it comes to innovation, and there is a need to “look at what’s happening on the policy front and look at the other components: the capital formation, what’s happening with reimbursement, immigration policy, [as] these are exceedingly important to biomedical innovation.”

Moderator Greg Simon of Poliwogg posed a final question to the panelists about sequestration and if a substantial deal will be made before the “fiscal cliff” deadline. The verdict was largely optimistic, with Jaeger and Tanden believing a solution was “hopeful,” Primus stating that a solution will be reached “because we have to,” and Gottlieb suggesting that most of the major issues will likely be punted to the next Congress.

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

When it comes to medical research, saving time means saving lives

That's the theme of a new social media campaign by FasterCures.

We launched Time Equals Lives to make a compelling statement about why we must invest in medical research. Time Equals Lives is a platform for personal stories, each one on its own is a strong case for why research matters, and when woven together collectively these stories create an imperative to make medical research a national priority. 

  

The campaign is anchored around FasterCures’ operating principle that to save lives, we must save time in medical research - the way we search for discoveries, turn these discoveries into therapies, and bring these therapies to patients. 

We are collecting stories from:

• patients and their families whose lives have been altered by disease,
• scientists and researchers facing incredible obstacles to advancing their work, but whose relentless efforts are bringing us closer to a cure, and 
• impatient advocates who know too well that improving a system means disrupting it and playing an active role in reshaping it.

The Time Equals Lives campaign provides our nation with a bird’s eye view of the critical importance of medical research. It is our goal to ensure we share these stories effectively and amplify their messages to leaders and decision makers whose work can chart the course of our future well-being. Here are some of the things we look forward to doing throughout this campaign:

1. delivering these stories in a creative and compelling ways to Members of Congress;
2. packaging these stories and sharing them effectively with Administration officials;
3. preparing these stories for all stakeholders in the medical research advocacy community to use as appropriate to advance their respective goals. 

 

We built the site in the public domain with the express purpose of making this resource available for the medical research advocacy community to use as appropriate.  

 

We urge you and those you know to add your stories to the mix. Here's how to get involved:

• VISIT the campaign Web site
• LIKE the Facebook page
• TWEET with us at #TimeEqLives
• DOWNLOAD and SHARE the Message
• TELL us your story

Introducing Fixes in Financing: Novel R&D Funding Models

by Elizabeth West, Program Manager, FasterCures

If you could buy a revenue-securitized bond to fund cancer research, would you? What about financing a clinical trial for a rare disease through crowdfunding? Thanks to a growing field of disease-focused venture philanthropies and nonprofits, and the emergence of creative new models for financing medical research, these scenarios could soon be reality.

Here at FasterCures we have spent the last few years examining, tracking, and highlighting the innovative funding mechanisms that are accelerating medical progress. These include pre-competitive partnerships to de-risk research investment, methods of diversifying research portfolios, bridge funding, and government-backed ventures. To highlight these emerging models – including those that reflect the priorities of medical research finance 2.0 – we recently launched a new monthly series called Fixes in Financing: Novel R&D Funding Models. The series will depict financing structures that are active, launching, or conceptual, and analyze them using a uniform set of metrics that includes investment focus, stage of research, and return on investment (financial, societal, and hybrid returns).

Check out the inaugural case, Breakout Labs, a revolving fund that gives small grants to newly formed companies to fund ground-breaking ideas. And let us know if you have an original funding concept we can feature in the series by sending an email to Elizabeth West ([email protected]). We’d love to hear your ideas from both within and beyond the biomedical research sector! 

Finance, especially creative finance, can be value-adding for research, not just another burden or obstacle between promising science and proven therapies.

Glass Half Full

FasterCures has been helping to incubate a new disease research entity – the Melanoma Research Alliance. The Web site went up recently (www.melanomaresearchalliance.org) and the first funding RFP is out (details can be found on the Web site). We took lessons learned from the experience of the Prostate Cancer Foundation and other disease research groups, and shortened the number of pages for the funding proposal, condensed the review turnaround time, and we will send grant award notifications out in spring.

By coincidence, I had a routine appointment with my dermatologist right after our State of Melanoma Research Call to Action meeting in November. When I mentioned to my dermatologist that I’d been part of the process to create this new melanoma entity, he replied that we already know all about melanoma but of course new funds for research were always a good thing.

More money can help, but that being said, this non-scientist would posit that we don’t have all of the answers. The American Cancer Society estimates almost 60,000 people in the United States were diagnosed in 2007. Additionally, 15 percent of those with metastatic melanoma will live for five years. It got better, or worse. When I asked if I should be getting a full body check since I’d been a lifeguard through my teenage years and spent most waking moments poolside, the reply was “sure, that is a good idea.” Hmm, I thought it would be a good thing if it hadn’t been my idea, but at any rate, I made an appointment. However, even if it is a good idea, detection methods still need to be improved and are currently imprecise.

Certainly knowing one or two more cures than we know now would be a major improvement and would save thousands of lives. It is certainly of concern when the medical profession’s knowledge of what is known and not known is lagging. Much to do. While this story might end on a negative note, I’ll go with the glass half full approach, since I am invigorated by the prospect of what the newly formed Melanoma Research Alliance will be getting done in 2008 for those currently diagnosed with melanoma and those at risk. Here's to getting the research done in a streamlined way, getting results translated into therapies as fast as possible, and getting up-to-date information to clinicians who are on the front lines.

Margaret Anderson, COO, FasterCures