Look Who's Talking at Partnering for Cures 2013

Remember to visit us on our new blog: www.fastercures.tumblr.com Partnering for Cures is a meeting focused on innovation, science, and collaboration. It is a platform for big ideas, transformative programs, and tangible results brought forth by some of the most disruptive innovators of our time. A shared sense of urgency binds together meeting participants, because we all know that when it comes to the search for cures, "soon" is just not good enough. JOIN US!  Look who's talking at #P4C2013 Confirmed speakers »  Panel topics »   Partnering for Cures emphasized the urgency that time is lives... We have to be willing to approach [challenges] in a different way to get solutions."  – Academic scientist   It's a place to challenge conventional thinking."   – Start-up company executive   [Partnering for Cures] is the biggest gathering of patient advocacy groups, biotech companies, pharma companies, and regulators."   – Xconomy Have you registered yet?   http://www.partneringforcures.org »

Evaluating “Value”: How Do We Foster Innovation in Today’s Cost-Conscious Healthcare Environment?

Two out of three participants in a recent FasterCures workshop believe that patients’ needs and priorities are not driving decisions made by payers, innovators, and providers. To have a stronger grasp on the role the patient can and must play in reimbursement decisions, FasterCures and the Cystic Fibrosis Foundation convened a one-day multi-stakeholder workshop earlier this month titled, “Value and Innovation: What Will the New Day Look Like for Patients?”

Fifty-three leaders participated from across the four Ps – venture philanthropy patientorganizations from TRAIN (the Research Acceleration and Innovation Network), biotechnology and pharmaceutical company executives, payers (public and private), and provider organizations.

Anchoring the day-long conversation, the first of five sessions focused on the stakes for patients in a cost-constrained environment, and how patients and their advocates can help shape the dialogue and decisions about who will pay for development of new treatments and rising healthcare costs. An overview of the Affordable Care Act followed, providing important context to the conversation about curbing health care costs. Next, the group focused on resources and infrastructure for research being created by innovative patient foundations – and how these groups are doing more and more to transform both the path toward cure and care for their disease. The fourth session looked into the types and sources of evidence payers use and are looking for to make informed reimbursement decisions. Finally, participants dug into the meaning of the term “value” and how new payment and delivery models are driving the definition of value and affecting innovation.

Five themes surfaced during the dynamic discussion:

1. Patient-relevant outcomes will ultimately drive the definition of value: Patient engagement in all stages of drug and device research and development, care delivery and payment decisions is vital. Determining and measuring outcomes that are important to patients is an emerging area of research and practice that will require experimentation and refinement.
2. Innovators and patients need to understand the evidentiary needs of payers: Defining what evidence is needed and collecting it at the earliest possible point can help drive toward a shared understanding of value across all stakeholder groups. High quality evidence is needed to make regulatory and payment decisions and will ultimately be generated through both traditional and novel study designs and analytical methods.
3. Better data is needed to make better decisions: A lot of data and information is being collected and stored from multiple sources across multiple platforms. Agreeing on standards and facilitating stronger interoperability would enable sample sizes to grow and better evidence to be harvested for the benefit of all stakeholders.
4. All stakeholders agree that it’s imperative to remove waste from the healthcare system: Up to 50 percent of healthcare delivered is of uncertain value and this uncertainty impacts all stakeholders. Cost pressures can force payers into the uncomfortable role of being “watchdogs” for quality and the evidence available to make decisions is uneven and may be applied inconsistently by different payers in the system.
5. The concept of value is derived from the total cost of a patient’s care, not the cost of an individual product: There is a strong need to unite sources of information about costs of care delivered in different settings by different providers, including family caregivers, and to identify means to calculate savings achieved through prevention and avoidance of harms. Costs of individual products are important but not sufficient to inform decisions about effective care to achieve better health outcomes for the individual and the broader population.

The call to cut waste out of the health care system and streamline the process of finding, delivering, and paying for the best possible therapy was a resounding theme.  Patient groups may also find themselves in the unfamiliar position of having to advocate for reducing inefficient treatments and care for their own patients out of the system.

Participants concurred that innovation and value aren’t mutually exclusive concepts and in fact need to be mutually reinforcing. Balancing the real-world competitive market factors for innovators and payers while striving to meet patient needs will require transparency, flexibility, communication, and – above all – trust within and between stakeholder groups.

These are issues of growing concern to many organizations with diverse constituencies and you’ll be hearing more about them. FasterCures will issue a detailed report on the workshop and its outcomes and an upcoming blog post will focus on recommendations generated at the workshop.

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.

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.