Life Science Compliance Update

March 28, 2017

Whatever the Question, Transparency Isn’t the Answer

Transparency

Many in political (and non-political) circles have blamed a lack of pricing transparency for increasing healthcare costs. However, as recognized in a study published in The Journal of the American Medical Association, improving transparency is not the answer. More than half of the states in the United States have passed laws that either establish websites with health care prices or require plans, doctors, and hospitals to disclose them to patients. Some employers and other organizations even provide health care prices to employees and the public.

However, according to the study, such transparency doesn’t always help patients spend less. The AMA article investigated the effect of transparency of the Truven Treatment Cost Calculator, a website that is available to more than twenty-one million workers and their family members. The website provides users with the costs (both the total price and the portion the user would be responsible for) from over three hundred services, including various sorts of imaging, outpatient operations and physician visits.

The researchers compared outpatient health care spending of 150,000 employees who had access to the website with that of about 300,000 comparable employees who did not. However, despite its features, the cost calculator wasn’t popular. Even though sixty percent of employees with access to it had a deductible of at least $500, only ten percent used it in the first year of availability and twenty percent after two years of availability. The study found that price transparency did not reduce outpatient spending, even among patients with higher deductibles or those who faced higher health care costs because of illness.

This Study is Not an Outlier

Many other studies show the same thing. Health plans report that use of their price transparency tools is limited, with many enrollees entirely unaware that they even exist. The majority of plans now provide pricing information to enrollees, but roughly two percent actually look at it. Aetna, for example, offers a price transparency tool to 94 percent of its commercial market enrollees, but only 3.5 percent of the enrollees use it.   

A study of New Hampshire residents found that only one percent used the state’s health care price comparison website over a three-year period. Another study found that the use of the price transparency platform Castlight Health was associated with lower payment for lab tests, advanced imaging and office visits; however, the study did not examine outpatient spending overall.

Why Is This?

Dennis Scanlon, a Penn State health economist, is not surprised by this result. “Health care choices are different than most product and services. Most decisions are driven by physician referrals, and insured patients usually face little variation in costs across options”

Another possible reason that people don’t cost-shop for health care is that they find the process to be too complex. As we have said time and time again, providing more information to consumers doesn’t always improve their decision making, in fact, many times, it can overwhelm a consumer and lead to poorer choices. It is easier to go somewhere based on a recommendation, even if it costs more.

September 19, 2016

New Rule and Guidance Issued on Clinical Trials

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Clinical trials are one of the most visible components of the biomedical research field, in part due to the way they directly engage human participants. Clinical trials have done a lot of good, providing advances in diagnosis, treatment, and prevention, but there are still large challenges. As such, changes are always needed to reflect science and society’s movement to increase efficiency, accountability, and transparency in clinical research.

Health and Human Services Final Rule

In an effort to make information about clinical trials more widely available to the public, the United States Department of Health and Human Services (HHS) issued a final rule that specifies requirements for registering certain clinical trials and submitting results information to ClinicalTrials.gov. The final rule expands the legal requirements for submitting registration and results information for clinical trials that involve drug, biological, and device products regulated by the Food and Drug Administration (FDA).

The requirements found within the rule generally apply to the “responsible party,” typically the sponsor of the clinical trial or designated principal investigator. The rule lays out a process for determining who the rule applies to, and which studies are considered “applicable clinical trials.”

Requirements under the final rule apply to most interventional studies of drug, biological and device products that are regulated by the FDA. The requirements do not apply to phase 1 trials of drug and biological products, or small feasibility studies of device products. The final rule also specifies how and when information collected in a clinical trial must be submitted to ClinicalTrials.gov. It does not, however, dictate how clinical trials should be designed or conducted, or what data must be collected.

Responsible parties must register their trial within twenty-one days of enrolling the first participant. The rule includes specific data elements that are to be submitted upon registration. Additionally, if the product studied is available via expanded access (when a product is used outside of a clinical trial, before it is FDA approved), the responsible party shall submit information about how patients can get access to that product.

Results information from applicable clinical trials shall generally be submitted within one year after the trial’s primary completion date. Submission of results information may be delayed, in certain circumstances, for up to two additional years for trials of products regulated by the FDA that are unapproved, unlicensed, or uncleared.

The type of information submitted will consist of “tables of information summarizing: 1) participant flow information, 2) demographics and baseline characteristics of the enrolled participants, 3) primary and secondary outcomes, including results of any scientifically appropriate statistical tests, and 4) adverse events.” As part of adverse events information there will be a table for all-cause mortality. The information will be submitted in aggregate, with no personally identifiable information. The rule requires responsible parties to update the information annually.

The final rule will be effective January 18, 2017. As of that date, ClinicalTrials.gov will allow responsible parties to comply with the rule; there is a ninety-day grace period for responsible parties to come into compliance with the requirements of the rule.

National Institutes of Health Policy Guidance

The National Institutes of Health (NIH) is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is the largest public funder of clinical trials in the United States. With such investment comes great responsibility. As such, NIH must work to ensure supported trials: investigate a mission-relevant question of high priority, do not duplicate previously conducted trials that do not need replication, and have the highest likelihood to advance knowledge and improve health.

NIH has launched a new effort to improve the quality and efficiency of clinical trials. These initiatives will reengineer the process by which clinical investigators develop ideas for new trials, how NIH reviews and selects clinical trials for support and oversees the progress of the research, and how results and aggregate data are shared broadly and rapidly.  

As part of the aforementioned initiatives, NIH issued a policy on the same day as the HHS final rule, in an attempt to promote broad and responsible dissemination of information from NIH-funded clinical trials through ClinicalTrials.gov. The policy establishes the expectation that investigators that conduct clinical trials funded at all by NIH will ensure that the trials are properly registered and that results information of the trials are submitted to ClinicalTrials.gov.

The policy, similar to the rule, requires applicable clinical trials to be registered in ClinicalTrials.gov no later than twenty-one calendar days after the enrollment of the first participant.

Applicants and offerors seeking NIH funding will be required to submit a plan for the dissemination of NIH-funded clinical trial information that will address how expectations contained within the policy will be met.

The new NIH policy applies to all NIH-funded trials, including phase 1 clinical trials of FDA-regulated products and small feasibility device trials, as well as products that are not regulated by the FDA, such as behavioral interventions.

The policy will take effect January 18, 2017. Failure to comply with the terms and conditions of the policy may result in enforcement actions, including termination.

FDA and NIH Comments

“When people participate in clinical trials, they are volunteering to create generalizable knowledge to help others in the future and we want their participation honored by ensuring that the existence of trials and their results are available to all patients and their healthcare providers, as well as researchers,” said FDA Commissioner Robert M. Califf, M.D. “The FDA will help ensure compliance with these new requirements so that patients and providers can have confidence in and access to significantly more clinical trial information, and researchers can improve clinical trial focus and design.”

“Access to more information about clinical trials is good for patients, the public and science,” said NIH Director Francis S. Collins, M.D., Ph.D. “The final rule and NIH policy we have issued today will help maximize the value of clinical trials, whether publicly or privately supported, and help us honor our commitments to trial participants, who do so much to help society advance knowledge and improve health.”

A summary table of both the final rule and new guidelines can be found here.

March 11, 2016

Biogen Joins the Center for Therapeutic Target Validation

The Center for Therapeutic Target Validation (CTTV) has a new member to boast about, helping them improve the success rate for discovering new medicines: Biogen. The CTTV, originally formed by GlaxoSmithKline, the Wellcome Trust Sanger Institute, and the European Bioinformatics Institute, works to foster deep, ongoing interactions between academic and industry members to develop open, transformative approaches to selecting and validating novel targets in drug development.

The CTTV covers all aspects of human health and disease and has an agreement between collaborators that experimental data and other information gathered within the CTTV will be shared to benefit the broader scientific community, but only after it has gone through basic quality control checks to ensure consistency and compliance with data sharing guidelines of both involved institutes.

Target validation, an exercise that CTTV focuses on, clearly defines the role that a biological process plays in a disease and is an important phase in drug discovery. Currently, it is estimated that ninety percent of compounds that enter clinical trials do not demonstrate the necessary efficacy and safety requirements, and therefore never actually reach patients as life-saving medicines. Oftentimes, this is because the biological target chosen is not completely understood.

Jeffrey Barrett, the Director of the CTTV, announced the collaboration,

the CTTV is proud to announce the addition of Biogen to our collaboration to improve the process of identifying and validating targets. The pre-competitive nature of CTTV is critical: the collaboration of our members allows us to make the most of commercial R&D practice while making the data and information available to everyone. It is truly exciting to apply so many different areas of expertise, from cell biology to large scale genome analysys, to the challenge of creating better medicines.

Sally John, the Vice President of Computational Biology & Genomics at Biogen, is looking forward to being a part of this partnership, stating, "we are committed to advancing evidence-based target discovery and opening up the field for researchers to create innovative methods and tools to accelerate the development of new medicines. Being part of the CTTV helps us realize this vision and provides a practical, harmonized way to share data with the scientific community."

The Vice President of Digital Health Technology & Data Sciences at Biogen, Philip Ma, also believes this partnership will be beneficial moving forward, stating, "The importance of accessing and managing searchable, structured data is critical to sharing knowledge on target validation. We look forward to combining our expertise in data sciences with the leading capabilities of GSK, the Sanger Institute and EMBL-EBI."

Biogen's membership with the CTTV follows the launch of the new CTTV Target Validation Platform, which helps researchers identify therapeutic targets for new medicines. The platform has had over 8000 visits since its December 2015 launch.

Is This Data Socialism?

The CTTV collaboration differs from the ICMJE proposed requirements and "data socialism" we previously wrote about, in part because the CTTV collaboration is a voluntary collaboration, whereas the ICMJE proposed requirements would mandate sharing hard-earned and heavily-researched data generated by interventional clinical trials. While this collaboration has some big names behind it, only time will tell as to whether or not it serves as a benefit to industry and the American public.

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