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/