Americans Say Congress Should Take Swift Action to Assure Patients Benefit from Treatments and Cures for Diseases
New Poll Data Summary booklet reveals concerns among Americans about the pace of medical progress
Majorities across the political spectrum say it is important that the new 114th Congress takes action on assuring the discovery, development and delivery of treatments and cures for diseases in the first 100 days of the legislative session (75% Democrats, 64% Republicans and 60% Independents), according to America Speaks, Volume 15, a compilation of public opinion polls commissioned by Research!America. As Congress considers numerous proposals in support of research, including the 21st Century Cures draft legislation aimed at speeding the delivery of lifesaving treatments to patients, it is notable to see public support in favor of accelerating medical progress.
“The new Congress has the opportunity to reinvigorate our research ecosystem and enact policies that will enable the private sector to expand innovation,” said Research!America Chair John Edward Porter. “Congress must work in a bipartisan fashion to realize the potential of promising studies to prevent and treat disease.”
An increasing percentage of Americans say the U.S. Food and Drug Administration (FDA) should move more quickly in order to get new treatments to patients, even if it means there may be risks. In 2015, 38% favor faster regulatory review, compared to 30% in 2013. Meanwhile, 25% say the FDA should act more slowly in order to reduce risk, even if it means patients may wait longer for treatments. Another 19% are undecided on this question and 18% do not agree with either position. Continue reading →
Statement by Research!America President and CEO Mary Woolley on 21st Century Cures Initiative Discussion Draft
The release of the 21st Century Cures Initiative discussion draft is a major bipartisan accomplishment that represents a truly remarkable diversity of innovative ideas to speed the delivery of lifesaving treatments to patients – a testament to the extraordinary commitment of Congressman Fred Upton (R-MI), Congresswoman Diana DeGette (D-CO) and their respective staff members. The Initiative could be a game changer for the medical innovation ecosystem with provisions touching on virtually all phases of the research and development pipeline – from basic and applied research, to FDA review, to coverage and access. Among the many beneficial provisions Research!America fought for is a measure to reduce the administrative burden on researchers. We look forward to working with the 21st Century Cures team to greatly boost our nation’s commitment to groundbreaking research and drug development.
The tiny increases included in the “Cromnibus” bill for the National Institutes of Health (NIH) and our nation’s other health research agencies are just that. The underwhelming support for the NIH, the Centers for Disease Control and Prevention, the National Science Foundation and the Food and Drug Administration following years of stagnant funding and budget cuts begs the question – how low can we go, given health threats the likes of which stand to bankrupt the nation? And the decision to flat-fund the Agency for Healthcare Research and Quality does not provide what it takes to reduce the much-complained of inefficiencies in our health care system. The pain and economic drain of one disease alone – Alzheimer’s – is not going to be effectively confronted without stronger investments in research. Every American who wants to see our nation overcome health threats, create jobs and shore up our economy for sustained prosperity must make it clear to the next Congress that it can and must do more, making research and innovation a strategic national priority.
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 →
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 →
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/