The year 2016 in the industry of clinical studies is sure to be remembered by the following events that will impact the area of experimental medical research and ultimately people’s health for the years to come.
$2.7 Million Research Funding from PDF
PDF – the Parkinson Disease Foundation – is interested in research proposals for emerging ideas that can help treat and end the disease. With this aim in view, the organization (which is a Parkinson’s Foundation division) is ready to make a total of $2.7 million investments as a part of its strategy to attract scientists into the pursuit of the task.
Even though there was little scientific progress in Parkinson’s over the past 10 years, the organization currently has more drug targets than 10 years ago, with some of them holding the potential of slowing or even stopping the disease some day. To make this happen, PDF makes efforts to mobilize researchers into pursuit of every promising idea.
Just to give perspective on the issue: the number of people living with Parkinson’s is estimated to double by 2030.
Age Factor in Thyroid Cancer Treatment
The Journal of Clinical Oncology, in October 31, 2016, published results of a clinical study that finds lack of statistical evidence to support the current age-based treatment practice of thyroid cancer patients. According to current practice, patients under age 45 are evaluated and treated differently from those of age 45 and older. Such guidelines were suggested by AJCC (the American Joint Committee on Cancer), the revised version of which (taking effect in January) confirms the same approach.
According to these AJCC guidelines, patients of 45 and older are evaluated on a 4-tier system, with stage 1 diagnosis meaning the earliest, and stage 4 – the latest phase of the disease, indicating that it has spread to other parts of the body. Patients under 45 are diagnosed by 2-tier system, according to which even a 44-year-old patient with metastases spread to lymph nodes throughout the neck is classified as stage 1, whereas the same condition with a patient a year older would be diagnosed as stage 4. Along with this dramatic difference in diagnoses comes also an equally dramatic difference in prognosis and treatment.
This is the current approach that the medical research conducted at Duke Cancer Institute (DCI) finds to be unjustified: among nearly 32,000 papillary thyroid cancer cases they studied, there were no age-based specifics that could affect patient’s prognoses and justify the age-based approach.
The data generated during this study suggests that when determining the cancer stage and prognosis, patients of all ages should be evaluated by the same standards, including lymph node involvement and tumor size. And this is because patient’s survival is determined by the same things no matter how old they are.
FDA’s Updated Guidance on Ethnicity and Race Data in Clinical Studies
Recently the FDA released updates on its 2005 guidance. It featured the agency’s expectations regarding collection of race and ethnicity data by sponsors and CROs during experimental medical research of drugs, medical devices and biologics. These updates were made in order to introduce a standardized approach to race and ethnicity data collection, meeting the requirements of:
- Section 4302 of the 2010 ACA (Affordable Care Act),
- Section 907 of the 2012 FSASIA (Safety and Innovation Act).
The report published by FDA according to these acts, failed to meet the abovementioned requirements regarding diversity and demographic subgroup analysis in applications submitted to the agency. This report revealed race-related inconsistencies in clinical trial participation, analysis and reporting, with whites being represented in highest percentage among study participants for drug, biologics, and medical device applications. According to the agency’s report, unlike cases of drugs and biologics, submissions of medical devices were less likely to include analysis of subgroup ethnicity and race.
Patient Engagement Study from DIA
The Study of Patient-Centric Initiatives in Drug Development – conducted by the Drug information Association (DIA) – measured the cost and ease of conducting study, and presented the impact these two aspect have on study results.
Thus, the study found that patient-centric, lower-cost initiatives are helpful and beneficial for drug development in a number of aspects, such as:
- reduced screen failure rates,
- improved subject retention,
- faster patient recruitment,
- greater amount of patient relevant endpoints,
- reduced protocol amendment.
The efficiency of patient-centric initiative is strongly supported by such activities as:
- advocacy group involvement and support
- patient counseling and education
- social media and online engagement
- patient advisory panels and focus groups.
For instance, patient advocacy group initiatives carried out in these four ways allow for such notable insights from the study as improved clinical trial performance and volunteer feedback.
The study revealed that the majority of pharma and biotech companies participating in the survey have introduced patient-centric initiatives into their drug development processes. Actually, 65% of the mentioned companies are financing and investing in patient-centered initiatives. Unfortunately, implementation was constrained by such conditions as lack of staff, risk tolerance, time, and budget. Additionally, quantification of the results was also challenging, given the limited comparative analytic data to support definite methods of patient-centricity.
Prior to this DIA study, almost all stakeholders (including CROs) believed that engagement of patients in the drug development process is important and valuable. However, the study showed that there wasn’t a single opinion about exactly why or how it is important and valuable.