Clinical News Roundup: FDA Updates Guidance On Race/Ethnicity
By Ed Miseta, Chief Editor, Clinical Leader
The FDA recently updated its 2005 guidance detailing expectations of sponsors for collecting race and ethnicity data in clinical trials for drugs, biologics, and medical devices. According to the agency, the guidance has been updated to reflect a standardized approach to collecting race and ethnicity data in accordance with requirements under the 2010 Affordable Care Act (ACA) and the 2012 FDA Safety and Innovation Act (FDASIA).
Under Section 4302, the ACA established new requirements intended to improve the understanding of health disparities, including requirements for standardization, collection, analysis, and reporting of demographic data. Then, under Section 907 of FDASIA, FDA was required to publish a report looking at the extent to which industry was meeting requirements for diversity in clinical trial participation and analysis by demographic subgroups in applications submitted to the agency.
The report found inconsistencies in the clinical trial participation, reporting, and analysis, particularly when it came to race. "Whites represented a high percentage of clinical trial study participants for biologic, drug, and medical device applications,” wrote FDA. “In many cases, other racial subgroups were underrepresented.” The agency also noted that medical device submissions were less likely to include a subgroup analysis for race and ethnicity than those for drugs and biologics.
DIA Releases Results Of Patient Engagement Study
DIA has announced the results of its Study of Patient-Centric Initiatives in Drug Development, reporting the majority of pharmaceutical and biotech companies surveyed have adopted patient-centric initiatives in drug development. The study, conducted in partnership with Tufts University, found that 65 percent of pharmaceutical and biotech companies surveyed have budgeted and are investing in patient-centric initiatives in drug development. However, barriers such as risk tolerance, lack of staff, time, and budget have constrained implementation. Furthermore, quantifying results is challenging as there is limited comparative analytic data that support specific methods of patient-centricity.
Still, the study found lower-cost, patient-centric initiatives are benefiting drug development. Reported benefits include reduced screen failure rates, faster patient recruitment, improved subject retention, reduced protocol amendments, and a greater number of patient relevant endpoints. The study measured the cost and ease of study conduct and reported impact. The four standout patient-centric initiatives against these measurements were:
- Advocacy group support and involvement
- Patient advisory panels and focus groups
- Social media and online engagement
- Patient counseling and education
Notable insights from the study include improved study performance and study volunteer feedback as a result of engaging patient advocacy groups in these four ways.
“Almost all stakeholders agree that engaging patients in the drug development process is valuable, but the DIA study reveals that they do not necessarily agree on why or how,” said Sudip Parikh, PhD, senior VP and managing director, DIA Americas.
The DIA Study of Patient-Centric Initiatives in Drug Development is available at www.diaglobal.org. It includes an executive summary, webinar, and detailed findings that provide insights on a wide range of patient-centric drug development initiatives.
Parkinson's Disease Foundation Opens $2.7 Million In Research Funding
The Parkinson's Disease Foundation (PDF), a division of the Parkinson's Foundation, is seeking research proposals for emerging ideas to help solve, treat, and end the disease. PDF investments of $2.7 million are part of its comprehensive strategy to mobilize the pursuit of all possible paths to a cure. Scientists can apply by visiting grants.pdf.org.
"Over the past 10 years, scientific progress in Parkinson's may have been difficult to see, as only a handful of new drugs were approved,” says James Beck, Ph.D., VP, scientific affairs for PDF. “But behind the scenes, thanks to PDF's commitment to basic science, we have more potential drug targets than we had 10 years ago. Some of them may one day slow or stop the disease in its tracks. To get there, we need to pick up the pace – to mobilize researchers to pursue every promising idea to improve lives and end the disease forever."
It is estimated the number of people living with Parkinson's will double by 2030. While research is promising, no therapy has been proven to slow or stop the disease. To meet the needs of its growing community, PDF grant opportunities reflect a continued commitment to basic science to keep the therapy pipeline full and identify possible cures and increased investments for cutting-edge clinical research to solve unmet needs in Parkinson's disease.
"For people like me who live with Parkinson's, every day matters," said Ron Wincek, a PDF research advocate. "In working alongside PDF and its expert scientists, I learned that it matters to them too. I am hopeful that together, we can end this disease."
Should Age Drive Thyroid Cancer Treatments?
A study from the Duke Cancer Institute (DCI) finds a lack of statistical evidence to support the current practice of treating thyroid cancer patients under age 45 differently from those 45 and older. The study, published October 31, 2016 by the Journal of Clinical Oncology, found that in nearly 32,000 cases of papillary thyroid cancer, there was no specific age at which patients’ prognoses changed so significantly as to require age-based standards.
The data suggest doctors should evaluate patients of all ages using the same standards, including tumor size and lymph node involvement, to determine the stage and prognosis of the disease. The findings challenge current thyroid cancer staging guidelines from the American Joint Committee on Cancer (AJCC), as well as revised AJCC guidelines that were published this month and take effect in January.
“The bottom line is that a staging system based on age may not be the optimal way to assess a patient’s prognosis and guide their treatment,” said author Julie Ann Sosa, M.D., an endocrine surgeon and surgical oncologist at Duke. Sosa herself served on the AJCC panel to draft new guidelines, which convened before the new data were available.
“Our preliminary data show there is no dichotomy in outcome at any specific age,” Sosa said. “Regardless of whether patients are younger or older, the same things influence their survival. The implication is simple: we need to rethink and potentially revise the current staging system.”
Current AJCC guidelines split patients into two groups: those under 45 and those 45 and older. The older patients are evaluated on a four-tier system, in which a stage 1 diagnosis means the cancer is in its earliest phase and stage 4 indicates the cancer has spread to the lymph nodes or other parts of the body. For patients under 45, the AJCC offers just two stages to encompass everything from a small, local tumor to cancer that has spread aggressively beyond the neck. Based on these standards, even metastatic cancer that has spread to lymph nodes throughout the neck in a 44-year-old patient is classified as stage 1. In a patient just a year older, the same disease would be described as stage 4 with a more grave prognosis and potentially more aggressive treatment.
Alzheimer’s Drug Clears Human Milestone
In a study published in Science Translational Medicine, a team from Merck Research Laboratories reports results of early human and animal trials of a drug called verubecestat, which targets the production of protein plaques associated with Alzheimer’s disease. “It's a summary of the discovery and early-stage profiling of what we hope is going to be a new therapeutic for Alzheimer's,” says team leader Matthew Kennedy. “It represents well over a decade of investment in this project by many scientists.” Definitive conclusions will have to await the results of larger, ongoing Phase 3 clinical trials to assess their efficacy, effectiveness, and safety.
Verubecestat is a so-called BACE1 inhibitor. BACE1 (for Beta-site Amyloid precursor protein Cleaving Enzyme 1, aka beta-secretase 1) is an enzyme involved in producing amyloid beta (Ab), a protein that clumps together, eventually forming the plaques surrounding neurons that are the disease's key hallmark. By blocking BACE1, the hope is this approach could prevent the buildup of these plaques in the first place. Until now, development of these drugs has been hindered by finding molecules with the right characteristics as well as concerns over theoretical and actual side effects.
The Merck team developed a molecule that appears to overcome these challenges. The molecule showed positive results in animals with no signs of toxicity, even after treatment of up to six months in rats and nine months in monkeys. The researchers then moved on to small, early-stage human trials to assess safety and tolerability and determine the choice of suitable doses for later trials.