Patient Engagement in Drug Development: Myth or Reality?
Is the patient voice adequately represented in the drug development process? The situation has improved but the patient community is not yet recognized as a co-equal in drug development or regulatory review according to Marc Boutin, executive vice president and chief operating officer at the National Health Council, a panelist at a BIO International Convention session on Wednesday. “We scare you,” declares Boutin who says there is an adversarial connection between patients, regulators and industry but the relationship has evolved over the last decade. Patient advocates have developed strategies to amplify their voice and demand access, providing the data and evidence to help transform healthcare delivery.
A section in the 2012 FDA Safety and Innovation Act fosters participation of patient representatives in appropriate FDA meetings with medical product sponsors and investigators, and a new transparent framework to communicate FDA benefit-risk approval decisions to the public. From the patient perspective, “benefit-risk decisions are value judgments, they are not science,” says Boutin. He acknowledges that FDASIA meetings are a critical step but sophisticated patient engagement is still lacking. In-depth interviews, focus groups, crowd sourcing and other strategies should be part of the equation, he adds. Getting the patient perspective in benefit-risk could change the discussion dramatically and improve healthcare delivery, Boutin says. Ensure that clinical trials are designed appropriately for people with chronic conditions, for example, to understand the benefit-risk for a subset of the population. Continue reading →
Global Consortium Protects Drug Supply Chain
Maximizing the return on investment in quality innovation was the topic of a BIO International Convention Super Session on Tuesday which explored challenges and opportunities for pharmaceutical companies in improving efficiencies and ensuring patient safety. Panelist Martin VanTrieste, Senior Vice President, Quality, Amgen described a global initiative formed by industry and regulators to share knowledge and best practices that will help secure the pharmaceutical supply chain and quality of materials. Rx-360, a consortium of pharmaceutical companies, suppliers and regulatory agencies, is tasked with developing voluntary standards for the quality and authenticity of supplies and suppliers, and developing audit standards, training and certification programs. The goal is to prevent counterfeited, adulterated and contaminated materials from entering the supply chain, and jeopardizing patient safety. One tragic example was the introduction of contaminated heparin, a blood thinner, linked to 80 deaths in U.S. patients and allergic reactions in 2008. VanTrieste said the consortium “has been highly effective in bringing industry leaders in the quality and supply chain security space together to come up with common solutions” in addressing the very difficult problems associated with globalization. “By working together we get to the answer faster and it’s more efficient.” Continue reading →
Innovative small businesses get boost from NIH, NSF
Federally-funded research projects that have advanced medical innovation will be on full display at the BIO International Convention Innovation Zone June 23 – 26 in San Diego. Among the new technologies, a device to prevent secondary cataract formation developed with a National Institutes of Health SBIR grant awarded to Sharklet Technologies, Inc. Secondary cataract, a serious complication of cataract surgery, occurs in 25% to 50% of patients. This complication requires a follow-up laser treatment which presents an additional risk to patients and adds more than $300 million in medical costs per year in the U.S. The novel device, a micro-patterned membrane designed to be integrated into a next-generation intraocular lens that has added functionality to prevent secondary cataract formation, could have a significant impact on improving patient care and reducing health care costs.
Improving patient care was also the idea behind a device developed by Actuated Medical. Many patients rely on feeding tubes for medication, nutrition or decompression, however those tubes can sometimes become clogged. A solution was needed to reduce risk and discomfort for patients and lower the expense of tube removal and replacement. SBIR grants from the National Science Foundation (NSF) helped take Actuated Medical’s feeding tube cleaning device from concept to FDA approval. Actuated Medical received a Phase I grant to investigate the technology and prove the feasibility of the device, and then a Phase II grant to develop the device from concept to verification-and-validation testing. Actuated Medical is also exploring various concepts that can be applied to reducing pain and understanding human hormones through the support of SBIR.
Elsewhere, researchers at P2D Bioscience received an NIH SBIR grant to test their lead compound which is an excellent anti-Alzheimer’s disease drug candidate.The research aims to develop an effective drug that can be taken orally to target the underlying neuroinflammation in Alzheimer’s to modify disease progression and improve cognitive function.
The NIH and NSF require robust and sustained funding to support small businesses that are improving the health of Americans. Even if it brings no immediate benefits, a majority of Americans agree that basic scientific research is necessary and should be supported by the federal government, according to public opinion polling commissioned by Research!America.
Sharklet Technologies, Actuated Medical and P2D Bioscience are among the small businesses exhibiting at the BIO International Conference Innovation Zone #BIO2014. For more information about how the Small Business Innovation Research (SBIR) grant program is helping biotech companies across the country, visit: http://www.sbir.gov/
Bold Public-Private Partnership aims to AMP-lify Medical Innovation
Image credit: National Institutes of Health
Technological advances have paved the way for researchers to access a wealth of data about the biological cause of disease. Yet translating these discoveries into treatments remains a challenge. Promising drugs often fail in late phase clinical trials, costing time and money, and leaving patients’ lives hanging in the balance. One reason is that the right biological targets were not chosen from the start.
To improve the current model for developing new diagnostics and treatments, the National Institutes of Health (NIH) and several biopharmaceutical companies and non-profit organizations formed the Accelerating Medicines Partnership (AMP), www.nih.gov/amp. “The good news is that recent dramatic advances in basic research are opening new windows of opportunity for therapeutics…But this challenge is beyond the scope of any one of us and it’s time to work together in new ways to increase our collective odds of success,” NIH Director Francis Collins, MD said in a press release. “We believe this partnership is an important first step and represents the most sweeping effort to date to tackle this vital issue.” Dr. Collins will be among the distinguished panelists at the “AMP-lifying Innovation” discussion on Wednesday, June 25 at the BIO International Convention in San Diego http://convention.bio.org/ #BIO2014 Continue reading →
Sharing a Photo/Selfie to Support Medical Progress
Selfies are a common form of expression on social media. They can be funny, serious, awkward or emotional. They can also be an impactful way of participating in a cause, which is a big reason we are launching a selfies/photo submission project for the Ask Your Candidates! national voter education initiative. As part of this effort, participants are encouraged to take a photo with a sign (download here), indicating why they support medical progress, or they can create their own sign, framing the issue in a way that speaks to their own experiences or organization’s mission.
The goal is to generate buzz for the initiative and increase the volume of the conversation surrounding medical progress, hoping to inspire more Congressional candidates to email statements to Ask Your Candidates on what they will do to accelerate medical progress, if they are elected. These statements will then be archived on the Ask Your Candidates! website.
To participate, just follow these three easy steps:
- Personalize an AYC sign about why you support medical progress or create your own sign using a blank piece of paper, so that you can talk about medical progress in your own way. For example, “I support medical progress in honor of…” or “Medical progress is important because…”
- Have someone photograph you with your sign, or take a “selfie.”
- Promote your photo on social media, using the hashtag #AYCresearch.
Below are two examples of text you can use to complement your social media post, but please feel free to tailor them for your needs
- I’ve shared why I support medical progress. Ask your candidates for Congress if they care? bit.ly/1sVNA7Z #AYCresearch
Or
- Here’s why I support medical progress. Show me why you support medical progress, too: bit.ly/1mS0pwR #AYCresearch
We can’t wait to see your photo or “selfie.” Remember, use the hashtag #AYCresearch so that we know that you participated, or email us your photo and we’ll be happy to share it for you.
If you need inspiration, check out some of our staff photos on Facebook. A few of them are below.
National Men’s Health Week – June 9-15, 2014: It’s time to do more about prostate cancer
Leading up to Father’s Day and as part of National Men’s Health Week, the American Cancer Society is raising awareness about risk factors for cancer in men. Among the cancer threats men face, prostate cancer is particularly lethal. In fact, it is the 2nd most deadly cancer for American men behind lung cancer. This year alone in the United States, an estimated 233,000 men will be diagnosed with prostate cancer and 29,480 men will die from the disease. Prostate cancer is also one of the most costly forms of cancer, with $11.9 billion spent on treatment each year in the US.
Despite these grim statistics, significant progress has been made in the area of prostate cancer research. Geneticists have identified mutations in the “HOXB13” gene as a cause of early-onset prostate cancer and certain protein markers have been found to be correlated with how much the cancer will spread. Advances in detection methods are making early diagnosis easier and more accurate. It is hoped that a new laboratory test currently in clinical trials will lead to fewer false positives and false negatives. Continue reading →
Are we doing enough to accelerate medical progress?
Excerpt of an op-ed by Research!America Chair The Hon. John E. Porter and Research!America Board member The Hon. Kweisi Mfume published in The Hill.
The Hon. John E. Porter
The Hon. Kweisi Mfume
The value of innovation has captured the attention of policymakers as they debate the merits of federally funded medical and health research. There is clearly bi-partisan support for research but battle lines have been deeply drawn over funding for research agencies in this tight fiscal climate.
The National Institutes of Health – the world’s leading funder of game-changing basic medical research – and other agencies contributing to the research pipeline are still affected by sequestration, the ongoing automatic spending cuts that have gutted promising research and shuttered labs. Congressional support for legislation to fund children’s health research demonstrates interest in accelerating medical progress, but the amount is miniscule compared to what’s needed to fuel our engine of discovery. The NIH has lost about 25 percent of its purchasing power in the last decade, jeopardizing the development of lifesaving therapies unleashed by genomics and other scientific breakthroughs. As a result, young scientists unable to secure grants for innovative research are leaving their careers, and institutions are struggling to continue important NIH-funded studies that could help combat national and global health threats.
To address the recent spread of polio in the Middle East and Africa, and the growing epidemic of noncommunicable diseases, we need more training and research to effectively put scientific discovery into practice. Sufficient resources will enable the NIH to train more scientists to better address global health issues which also affect us closer to home – heart disease, cancer, mental illness, diabetes and HIV/AIDS. Outbreaks of polio have impacted a nearly three-decade effort to eradicate the disease globally. The World Health Organization has declared the disease an international public health emergency as it re-emerges in Pakistan, Cameroon, Syria and other countries previously free of polio which can kill or cripple the hardest-hit victims.
Why isn’t Congress paying closer attention to the health threats before us? To accelerate innovation, protect health and save lives, policymakers must close the massive gap between the level of funding necessary to advance medical progress and the token funding levels allocated to research over the last several years.
Member Spotlight: The American College of Neuropsychopharmacology
By Peter W. Kalivas, PhD, President of the American College of Neuropsychopharmacology. Dr. Kalivas is Professor and Chair, Department of Neurosciences at the Medical University of South Carolina.
The American College of Neuropsychopharmacology (ACNP), founded in 1961, is the nation’s premier professional society in brain, behavior, and neuropharmacology research. The field of neuropsychopharmacology involves evaluating the effects of natural and synthetic compounds upon the brain, mind, and human behavior, and the ACNP serves as a forum for advancing the latest discoveries about the brain towards cures for neuropsychiatric diseases.
The core purpose of the ACNP is to catalyze and advance scientific discovery about disorders of the brain and behavior in order to help prevent, treat and cure brain diseases. The ACNP members are nominated from the national leadership in the fields of Biological Psychiatry and Neuroscience, and the College and its Annual Meeting are kept small by design (just over 1,000 members) in order to facilitate scientific exchange and career mentoring at the Meeting. Importantly, the ACNP is a venue at which the best scientists from academia, government, and industry gather to share, discuss, and debate their research. The College also plays a key role in mentoring early career clinicians and scientists in the field of neuropsychopharmacology via education, travel grants and providing individual mentors. Continue reading →
5 by June 5 – Turning up the Volume on Medical Progress
When it comes to midterm primaries, June is a blockbuster month: 17 states are holding primaries through June 24. Today, primaries are set for Alabama, California, Iowa, Montana, New Jersey, New Mexico and South Dakota, and next Tuesday, June 10, primaries will be held in Maine, Nevada, North Dakota, South Carolina and Virginia.
From now through the general elections, it is a great time to get involved in the Ask Your Candidates! voter education initiative. One way to participate is by joining us in our 5 by June 5 (5X5) awareness effort, in which we are guiding voters to ask their candidates about medical progress and recruit five people to do the same by June 5.
As you may know, Congress plays a key role in determining funding for noncommercial medical research and formulating policies that incentivize – or de-incentivize – private sector medical innovation. Who we elect affects whether new treatments and cures are discovered or lie dormant. That’s why voter education is so important. Continue reading →
Investing in Cutting-Edge Technology to Advance Cancer Research
From KIYATEC website
Matthew Gevaert and David Orr developed an innovative approach to cancer research, testing new drug compounds using live cells from patients with a device that resembles a Lego. Gevaert and Orr’s “3DKUBE,” a cell cultured plasticware, creates a 3Dmodel of patient cells that allows researchers to study the growth of the cells in a cultured environment that mimics the conditions of the human body. The process is designed to produce more relevant data on drug safety and efficacy, and determine which drugs are most effective for treating cancer patients. To expand use of this technology, the National Cancer Institute (NCI) awarded a $295,000 Phase 1 Small Business Innovation Research (SBIR) contract to Matthew and David’s company KIYATEC to develop a 3D model specifically for breast cancer patients. The company plans to eventually use the model to more accurately predict a patient’s response to certain drugs for lung and brain cancer.
