Building a culture of participation in research

By LaTese Briggs Program Analyst at FasterCures

“I have been waiting 174 days to say this: 'Good Morning America.' "

IDA MAE ASTUTE/ABC

Good Morning America anchor, Robin Roberts, made her triumphant and long awaited return to the talkshow last month after a five month recovery from a bone marrow transplant to treat myelodysplastic syndrome (MDS). The show and her fans have been celebrating and supporting Roberts’ through her courageous fight and recovery. An emotional Roberts’ thanked her fans, colleagues, and doctors for helping her through her battle with the disease, but she also took the time to thank all of the courageous people that participate in clinical trials.

“The standard treatments of today are developing because patients before you participated in clinical trials, and in a certain sense it’s our obligation as physicians and the patients of today to develop the standards of care for the patients of tomorrow.”

By participating in clinical research studies, these giving volunteers play a key role in improving the standard of care for themselves and future patients. Clinical trials are critical to bringing new drugs and interventions to patients in need. In a recent editorial by Francis Collins, Director of the National Institute of Health (NIH), he explains that the wealth of discoveries related to the genetic and environmental causes of disease, have led scientists to numerous new targets for drug development; however, the rate at which new drugs and other therapeutics are reaching patients has not increased proportionately. A key impediment to the translation of these discoveries to new treatments is the poor participation rate of Americans in clinical trials. This problem has been well-demonstrated in cancer where according to the NIH, only 3% of adults with cancer are participating in clinical trials.

According to studies conducted by CenterWatch, 94% of Americans recognize the importance of participating in clinical research in order to assist in the advancement of medical science; however, 75% of Americans state they have little to no knowledge about how clinical trials work and the participation process. This staggering statistic highlights the need to raise clinical trial awareness among the general public. Even small efforts like a simple thank you to clinical trial participants from a beloved morning show anchor, can have a major impact on achieving this goal. Thus involvement from the media and high profile individuals to illuminate the importance of clinical trials can be used as a powerful tool to educate the public on the link between improvements in healthcare and clinical research.

Educating patients and non-patients alike has become infinitely easier with the advent of online registries and databases. Sites like clinicaltrials.gov help doctors, patients, friends, and family members find clinical trials soon to be conducted in their area, their specific disease or a find a call for a person with a particular medical history.

The involvement of patient advocacy groups has also proven to be an effective way to increase clinical trial awareness and patient enrollment. A good example of this is the Love/Avon Army of Women collaboration between the Dr. Susan Love Research Foundation, the Avon Foundation, and the American Association of Cancer Research. The goal of this initiative is to recruit one million women willing to donate time, biospecimens and data to breast cancer research. To date, the organization has recruited nearly 400,000 women.

In addition, the use of outreach workers to educate patients about clinical trials can also potentially have a huge impact. These workers would not only make patients aware of clinical trials, but also work to address concerns and misconceptions about clinical trials that some may have. Common concerns include fear of experimentation, logistical concerns related to costs, insurance coverage, travel, missing work and/or time with family. Some common misconceptions are that clinical trials are last-resort efforts and one should only participate when all other approved treatments have failed, placebo treatment means that no treatment will be provided, and the assumption that intervention or treatment in the clinical trial is more invasive than standard treatment.

Clinical trials are truly the crux of drug development and medical innovation. Most trials evaluate new treatment options for safety and efficacy before making them commercially available; however, clinical trials are also used to understand the root cause of disease, improve disease detection, and compare commercially available drugs to determine which are more effective in certain patients. 

By raising awareness and educating the public about the power of clinical trials, we will be able to move research forward faster such that the many patients plagued by one disease or another will be able receive new and innovative medical solutions that hopefully exceed the current standard of care.

To find out more information about clinical trials and how you can get involved, check out these links:

-- http://www.armyofwomen.org/

-- http://www.patientslikeme.com/

-- https://foxtrialfinder.michaeljfox.org/

-- http://www.alz.org/research/clinical_trials/find_clinical_trials_trialmatch.asp

-- http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/app/clinical-trials-matching-service

-- http://clinicaltrials.gov/

FasterCures' Patients Helping Doctors (PHD) Program
The Patients Helping Doctors (PHD) program anchors everything we do at FasterCures. In fighting disease, patience is not a virtue—patients are. Through PHD, we are building a culture of participation in research where patients and healthy volunteers understand the fundamental value they bring to clinical research. We focus our efforts on unlocking patient information—medical records and biological material such as tissue, blood, and DNA—and making these available to clinical researchers in a meaningful way. Learn more here.

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.

Time=Lives Story of the Week: Phil Gattone

"There really isn't much logic in sending your child to brain surgery, but we did. Because we knew we were losing him."

President and CEO of the Epilepsy Foundation, Phil Gattone, and his wife Jill began the fight for their son Phillip’s life in 1991 after he suffered his first seizure at the age of four that lasted almost an hour and landed him in the emergency room.

Watch Phil’s story here

“It was a researcher who we probably will never know who helped well before our son started having seizures,” said Phil. “Because of the tremendous efforts of his clinicians and those researchers, there was a sense of hope.” 

Now in his mid-20s, Phillip is a software engineer at Northrop Grumman Aerospace Systems and is able to effectively manage his epilepsy.

According to the Centers for Disease Control and Prevention, epilepsy affects 2.2 million Americans and 65 million people worldwide. Approximately 1 in 26 people in the United States will develop epilepsy at some point in their lifetime, with an aging baby boomer generation reaching retirement age the number of citizens with the disorder is predicted to grow.

Advances in treatment for epilepsy have provided a functionally normal life for people like Phillip, but despite how common it is, epilepsy remains one of the least understood chronic medical conditions. While medications and other treatments help many people of all ages who live with the condition, more than a million people continue to have seizures that can severely limit their school achievements, employment prospects and participation in all of life's experiences

Research is the key to the future for people with hard to control seizures, and the trajectory of epilepsy – and other disease for which there are no cures – depends on attracting the best scientific minds and funding innovative clinical investigation. The science is promising, but without continued support and resources, we risk patients like Phillip falling by the wayside.

To learn more about the Epilepsy Foundation and get involved in their work, go to http://www.epilepsyfoundation.org. 

And check out the Time=Lives campaign to find out what you can do to help speed the medical research process by participating in a clinical trial, sharing your story, or simply spreading the message that medical research must be a national priority.

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

FasterCures Finds Outcomes, Metrics, and Strategic Leadership Define the Effectiveness of Venture Philanthropy Groups

Two new reports outline the practices and approaches of nonprofit funders of medical research

FasterCures today released two reports that paint a vivid picture of how medical research foundations, also known as venture philanthropy groups, have transformed the medical research enterprise. These entrepreneurial groups are accelerating medical research and development by tackling science where it needs the most effort and resources, and applying innovative business approaches in the pursuit for a cure.

Both resources tell a compelling narrative of how venture philanthropy groups are creating a culture in medical research that is mission-driven, results-oriented, and focused on the true bottom line: preventing, diagnosing, and curing disease.

• The first, Honest Brokers for Cures: How Venture Philanthropy Groups are Changing Biomedical Research, features insights from leaders of 20 venture philanthropy groups. This publication features valuable insights and perspectives from leaders of medical research foundations that are transforming the cure enterprise by virtue of how they find and fund research. In this report, FasterCures dissects the business model that has emerged from the shared sense of urgency and frustration stemming from these patient-driven organizations, and their laser-sharp focus on outcomes.

• The second, Measuring and Improving Impact: A Toolkit for Nonprofit Funders of Medical Research, is a how-to guide for foundations seeking to apply some of the best practices and lessons learned from venture philanthropy groups who’ve demonstrated their effectiveness.  It provides a common framework for assessing and improving organizational effectiveness, and a panoply of ideas, questions, and models to help guide new and emerging nonprofits with strategic and tactical choices.

Venture philanthropy groups play an outsized role in improving the medical research and development system to better meet the needs of patients. Since its inception, FasterCures has continued to shine a light on the successes of these foundations in an effort to amplify their lessons learned and sense of urgency to the rest of the medical research community.

“In the end, the most central characteristic of these groups is their close connection to the disease they are pursuing. For most, it is personal—either they or a family member or close friend is or has been affected by the disease,” said Margaret Anderson, executive director of FasterCures. “It spurs them to find the dollars to meet the challenges and change the trajectory of research. We can all benefit from the lessons they've learned.”


                                                  

To download these publications, visit http://www.fastercures.org/Publications/vp.php

 

Time=Lives Story of the Week: Derrick and Meredith Day

“I think I’m the same, except I just can’t see.” 

– Derrick Day, 6 years old

Derrick (6) and Meredith’s (4) story came to Time=Lives via the Foundation Fighting Blindness, an organization dedicated to driving research to prevent, treat and cure people affected by retinal degenerative diseases.

View Derrick & Meredith’s story here.

Born legally blind, the siblings suffer from a rare inherited eye disease called Leber’s Congenital Amaurosis, a disorder that can also create central nervous system abnormalities.

Seemingly simple tasks for a sight-abled person become difficult hurdles or even impossibilities when disease has robbed someone of their sight. Derrick’s dad dreams of a day when his son will be able to drive a car. “I never thought about Derrick’s not being able to drive,” said his mom. “When we got to the stop sign, he presented the question to me: Mom, how are we going to make that sign braille so that I can drive?”

Whether it’s as simple as experiencing a rainbow, or as complex as driving a car, the only way for kids like Derrick and Meredith to be able to achieve these seemingly insurmountable tasks is to be able to see. “And the only way that we can have them see,” says their mom, “is with research, and by funding scientists to find a cure.”

The good news is that retinal disease science is advancing. For example, just yesterday the Foundation Fighting Blindness reported that the Argus II retinal prosthesis, a device that can restore some vision to people who are blind from advanced retinitis pigmentosa (RP), received U.S. market approval from the Food and Drug Administration (FDA). More than 20 years of research went into the development of Argus II, with early support from the Foundation.

Also, earlier this month The Washington University School of Medicine created an innovative method for treating vision-robbing diseases using genetic reprogramming. And while their research is at an early stage in mice, it has revealed valuable clues about how to potentially save vision in people.

But promising research like Washington University is conducting wouldn’t be possible without federal funding.  And with sequestration – mandatory, across-the-board budget cuts – looming, the researchers who study diseases like Leber’s Congenital Amaurosis and others could face debilitating cuts to their research programs that would, at best, slow innovation and progress, and at worst, cause entire labs to shut down.

Want to know more about sequestration? Visit FasterCures’ Sequestration Station for facts, forecasts, and ways to add your voice to the fight for funds. Medical research matters, and must be supported. Derrick, Meredith, and millions of others suffering from debilitating diseases are counting on it.

See more stories about the power and promise of medical research, and tell us why medical research matters to you.

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

Funding for Translational Research at NIH

This month, The National Institutes of Health will be accepting its first round of applications for a new funding opportunity focused on supporting collaborative translational research projects.  First announced last fall, this U01 research program – Opportunities for Collaborative Research at the NIH Clinical Center – is designed to provide extramural (non-NIH) investigators a mechanism to establish collaborations with NIH intramural investigators and to take advantage of the unique research opportunities available at the NIH Clinical Center.

It is being offered by 12 NIH institutions and is intended to facilitate the translation of basic biological discoveries into therapeutic candidates for clinical testing.

With the creation of this funding opportunity and the establishment of NIH’s newest Center, NCATS, NIH continues to push forward its vision to transform the translational process for the benefit of patients. Recognizing the critical advancements that have been made in the understanding of basic disease biology, the agency continues to look for ways to infuse new and innovative thinking into not only the scientific discovery process, but also the process of getting important new medicines from discovery to patient.

We’re excited to see the increased focus on collaboration – for entry to the program, extramural research projects must have a collaborating investigator in the NIH Intramural Program as well as a “Collaboration Plan” – and eagerly await the first crop of awardees.

Fully utilizing the NIH Clinical Center was one of the recommendations that FasterCures had put forward in 2008 through its blue-ribbon task force focused on the NIH Intramural Research Program led by Nobel Laureate and FasterCures board member Dr. David Baltimore. In fact, of the five recommendations the task force presented, three have been acted on, with outcomes that will save lives by shortening the time it takes to turn breakthroughs into medical solutions.

To learn more about the application process for this grant program, watch the pre-application webinar or visit the official announcement page.

Resources:
Sequestration Station – Check out FasterCures’ Sequestration Station for more information on how sequestration will affect the National Institutes of Health.

Need Cures? Who Ya Gonna Call?

By Margaret Anderson, Executive Director, FasterCures
As seen on HuffingtonPost 

Do you know anyone whose life hasn't been touched by disease? As I get older I become more aware that the frenetic life I lead and call normal could be interrupted in the blink of an eye.

We all know the drill. People in our lives are just going about their business when something goes amiss -- a twitch in their leg, some dizziness, a lump, a pain, an abnormal result, an accident, an ambulance, a hospitalization, a doctor's visit, a test, a diagnosis. A new reality. A replacement of what once was to a new normal.

I can conjure up times in my life that disease has interrupted life just like that. Here are just a few that were easy to recall; I am sure you have just as many.

• "I didn't see this coming -- I thought it was my heart medication." -- My father after his leukemia diagnosis.


• "This week, I found a lump in my breast." -- My friend when she put her hand on my arm after I asked what's new.


• "My dad is at the end of life after battling Alzheimer's." -- My colleague after her dad began his final days.


• "My doctor had told me I would be dead in three years." -- My friend recounting how in the earlier days of the AIDS epidemic there was no prospect of survival.


• "I also realize that while I have what may be the nastiest cancer out there, I have it easy compared to others." -- My friend describing his fight against pancreatic cancer.

That moment when you or someone you love become a patient, the patient. And then what?

Well, hopefully the wonders of science and medical research will have led to good therapeutic options for you. At our recent Celebration of Science event, we witnessed scientific discovery literally unfold on the stage of the National Institutes of Health as scientists and patients told breathtaking stories of research triumph. Here's a glimpse of how scientific discoveries have improved health and saved lives. It's remarkable. But, consider that of the 4,000 diseases for which we have the exact molecular basis, only 250 have treatments available.

So, while we need to celebrate the success stories in medical research that allow us to carry on our lives -- be those successes through prevention, diagnostics, devices, or medical intervention -- we have more work to do. We must ensure that we continue to have a robust flow of scientific discoveries that we can then translate into better health.

The well-being of too many of our friends and family depends on this.

The pathway to treatments and cures is littered with failure, lack of funding, scientific and regulatory challenges, reimbursement issues, health care delivery issues, and if that is not enough there are immediate challenges like the impending fiscal cliff and sequestration. Decisions that our reelected President Barack Obama and the U.S. Congress will make could substantially impact the future of treatments and cures.

I could cite data to show how medical research matters.

• It saves lives.
• It creates jobs.
• It maintains us leadership in the global economy.
• People's lives depend on it.

We face a major paradox -- that the potential of science is greater than ever but the outlook for funding has never been bleaker. If an agreement on how to prevent sequestration doesn't happen, here's what we'd see:

• U.S. government science funding programs would see their budgets cut by 8.2 percent in 2013.
• $2.52 billion decline in funding at the NIH.
• 2,300 fewer NIH grants -- representing almost a quarter of new and competing grants.
• Total employment supported by NIH awards would fall by 33,704.
• The Food and Drug Administration (FDA) will lose $318 million from its budget, which wasn't adequate to begin with.
• Virtually all user fees paid to the agency to ensure swift application reviews would be considered part of the sequester.
• About 80 percent of the agency's budget is devoted to personnel, so cuts would mean reduced staffing levels or imposing furlough days

So, given that the need is great, the promise is there, and the arguments in support of this funding are strong, now what?

1. Get educated. Go to our website called Sequestration Station to get smart on the issues and see how you can get involved.
2. Tell us your story. Be a part of our new social media campaign called Time Equals Lives. We are collecting personal stories -- from patients and scientists, researchers and caregivers, industry executives and academics -- to make a compelling case about the critical importance of medical research. We'll share these stories with leaders and decision makers to remind them of what is at stake.

Because when you or a loved one needed a cure, we want to make sure you knew you'd have someone to call on.

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For more by Margaret Anderson, click here.
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