TRAIN E-News

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TRAIN groups give patients voice through Patient-Focused Drug Development initiative

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Under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) in 2012, the U.S. Food and Drug Administration (FDA) initiated the Patient-Focused Drug Development Initiative (PFDDi). Over the course of the five-year commitment, FDA has collected input from 2,500 patients during PFDDi meetings, which have informed the regulatory decision-making process. To expand the value of participant input, the FDA initiated a process for patient groups to host externally-led PFDDi meetings that the FDA is open to participating in. These meetings are structured largely the same as the FDA-sponsored ones, giving FDA staff the opportunity to gather patient input from a broader group of disease areas.

So far, around 10 patient organizations have hosted these externally-led PFDDi meetings, including TRAIN groups such as the Leukemia & Lymphoma Society, Tuberous Sclerosis Alliance, and Parent Project Muscular Dystrophy. These groups have put together "Voice of the Patient Reports” that provide a summary of the patient perspectives gathered at the meetings. They also involved FDA staff to expose them to a diversity of patient voices within these communities.

To host these externally-led meetings and collect patient perspectives, foundations need to collaborate with other patient groups and stakeholders as well as FDA. This work is an excellent example of the unique impact patient groups can have as honest brokers within the medical research system. 

Recently, the FDA hosted a public workshop on Patient-Focused Drug Development: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data. During the meeting, a diverse set of stakeholders spoke about improvements to the development, scope, and submission of draft guidance. FasterCures looks forward to following the outcomes from this (and upcoming) PFDD meetings, and we continue to celebrate the modernization of the FDA’s drug regulatory system in an effort to expedite medical research

As we look to the future, refresh your knowledge of the history and advances of patient-centricity since PDUFA V was passed through FasterCures’ report, From Aspiration to Application: 5 Years of Patient-Centricity.

News from FasterCures

Milken Institute Global Conference tackles medical research issues

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Earlier this month, the Milken Institute hosted the 2018 Global Conference in Los Angeles. Throughout the conference there were several health-focused sessions, including panelists such as Lou DeGennaro of the Leukemia & Lymphoma Society on Paying for Cures, and Louise Perkins of the Melanoma Research Alliance and Jill O'Donnell-Tormey of the Cancer Research Institute on Preserving the Promise of Cancer Immunotherapy.

Join us for an exciting conversation about the FDA’s Patient-Focused Drug Development initiative

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On June 7, FasterCures will host a webinar to discuss the FDA’s Patient-Focused Drug Development (PFDD) initiative. During this webinar, panelists will share their experiences working with patient communities to plan for these often-intense PFDD listening sessions, as well as what they’ve learned from them and how they are applying this information. The discussion will be moderated by Cynthia Grossman (Director, Science of Patient Input, FasterCures), and panelists include Meghana Chalasani (Analyst, Center for Drug Evaluation and Research, U.S. Food and Drug Administration), Annie Kennedy (Senior Vice President for Legislation & Public Policy, Parent Project Muscular Dystrophy), and Colleen Rye (Director, Research and Regulatory Policy, FasterCures). Register today!

Elevating the patient perspective on patient-reported outcomes

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Following the publication of our report, “Patient-Reported Outcomes: Design with the End in Mind,” FasterCureshas continued the important work of evaluating how the biomedical research and development system measures and utilizes the outcomes that matter most to patients. Recently, Cyndi Grossman spoke with Daniel Levine of the Bio Report about the challenges and opportunities of patient-reported outcomes (PROs). Listen to the podcast or explore the topic further by watching our short video series on PROs.

What patient trust in health-care organizations means for patient engagement

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Before patient data can be gathered for research, patients need to trust the organizations they communicate with in the health-care system. Through FasterCures' Health Data Basics project, we surveyed over 700 patients and caregivers about their perspectives on health data and how much they trust others with it. To discover what we’ve learned so far, read our blog.

Create effective partnerships with TRAIN toolkits

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Collaboration is critical to accelerating research. But it can be difficult to build lasting and effective partnerships that have an impact. Whether your foundation is seeking to create a partnership with a company or a university or hoping to build capacity by convening collaborations, TRAIN’s toolkits have information to help and improve those efforts. Get started by exploring these tools.

TRAIN Organization Updates

Lipedema Foundation funds novel research in the field

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An article from Giving Compass recently highlighted a new TRAIN participating organization, the Lipedema Foundation. In the article, the foundation’s founder, Felicitie Daftuar, said, “To make an impact, you need to do your homework…It takes effort to make sure that your giving strategy is aligned with the outcomes you want to see for patients. We wanted to make sure our investments were not only high-risk, but also had the opportunity for high reward.” The foundation has made significant progress in the last two years through their funding of research that comprehensively studies the microenvironment in which lipedema forms and persists.

CURE’s Epilepsy Genetics Initiative makes important discovery of gene mutations

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Genomic data from CURE’s Epilepsy Genetics Initiative has revealed that de novo mutations in the PPP3CA gene lead to a severe form of the neurodevelopmental disease characterized by seizures. This is an important discovery for those who have epilepsy, as the knowledge may inform the causes of the disorder and the possible treatments for patients.

Research could lead to preventive treatment for Alzheimer’s

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According to an article from the Daily Beast, two Harvard scientists are hopeful that their current research may lead to a preventive treatment for Alzheimer’s, which is commonly thought of as impossible in the field. With funding from Cure Alzheimer’s Fund and Open Philanthropy, the researchers are mapping the Alzheimer’s brain by looking at autopsies in Alzheimer’s patients and elderly subjects without the disease. This could lead to a test for young people to see if their brains are susceptible to the disease.

Study led by TGen may hold the key to ovarian cancer drug

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A study led by the Translational Genomics Research Institute (TGen) discovered that a powerful anti-cancer drug used to fight leukemia might have promise in treating aggressive forms of ovarian cancer. According to TGen’s announcement of the findings, “more than 14,000 patients in the U.S. will die this year from ovarian cancer, making it the 5th leading cause of cancer death among American women.”

News You Can Use

Vivli opens beta version of its innovative data-sharing platform for clinical trials

According to a news release, Vivli has created a novel global platform for sharing clinical trial information. The beta version is now open, with the official launch event to be held at the National Academy of Sciences in Washington on July 19. With the launch of the Vivli platform, the global research community will gain a powerful new way to discover, share, and analyze clinical trial data. You can register for the free launch event online and find more information about the agenda, speakers, and logistics on Vivli’s website.

Government incentives could prod investment in addiction research

Research on vaccines and treatments for addiction is underway at Scripps Research Institute, Weill Cornell Medicine, Walter Reed Army Institute of Research, Baylor College of Medicine, and Selecta Biosciences, but the lack of a viable market keeps many drugmakers from funding addiction research, says FasterCures Senior Fellow Bernard Munos. Addiction poses enormous personal and economic burdens, and government-funded incentives may be needed to spur innovation and investment, Munos says.

Researchers make patients active partners in studies

The Broad Institute and the Dana-Farber Cancer Institute are making patients in the Metastatic Prostate Cancer Project active partners in the research by asking for their input, holding events, and providing progress updates, according to an article in the Wall Street Journal. It is part of a larger effort to change how research is done because scientists using genomics need large databases of information and long-term patient involvement.

Calif. universities harnessing the power of collaboration

University of California cancer centers at San Francisco, Los Angeles, San Diego, Irvine, and Davis are collaborating on the five-year UC Cancer Consortium, sharing best practices and data, and building statewide clinical trials. Collaboration boosts efficiency in research, but it is “horribly underutilized” and more difficult than it should be, says Clay Johnston, former associate vice chancellor of research at UCSF, in the San Francisco Business Times.

Study finds 8,000 unstudied proteins for future drug research

An analysis of 20,000 proteins that are potential drug targets showed about 8,000 had not been mapped or studied by researchers or pharmaceutical companies, researchers reported in Nature Reviews Drug Discovery. The study categorized the 20,000 proteins into four groups and ranked them on future potential for drug development.