HSPP- July-August-September, 2018- Newsletter

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AAHRPP Site Visit 2018

 The Association for Accreditation of Human Research Protection Programs (AAHRPP) is an independent, non-profit accrediting body that works to protect the rights and welfare of research participants and promotes high-quality research through an accreditation process.

UConn Health’s Human Subjects Protection Program (HSPP) achieved AAHRPP accreditation in 2006.  As an accredited organization, our HSPP must periodically apply for re-accreditation.  This is a two-step process.  Step 1, which has been completed, consisted of AAHRPP reviewing all of our policies and procedures to ensure they meet regulatory and accreditation standards.  Step 2 is an onsite visit by AAHRP representatives to ensure that our policies and procedures are followed.  While specific dates have not yet been determined, the next accreditation site visit will occur in the spring of 2019.

During the site visit, representatives from AAHRPP will conduct interviews and review studies records to ensure that policies and procedures have been implemented effectively.  AAHRPP representatives will interview the Institutional Official (Wesley Byerly), IRB members, HSPP staff, investigators and research study team members, and individuals that represent various research compliance functions, such as radiation safety, research pharmacy, scientific review, and conflicts of interest.  Individuals selected for such interviews will be asked questions about regulatory and ethical issues related to research with human participants and how they interact with the IRB. Investigators and research staff will also be asked about the conduct of their research studies.

In general, topics that may be included during an AAHRPP interview process include:

  1. Familiarity and compliance with HSPP/IRB polices and procedures
  2. Roles and responsibilities of investigators and research staff
  3. Obtaining and documenting informed consent
  4. Conflict of interest disclosures
  5. Minimizations of risks to research participants and protecting human subject’s rights and welfare
  6. Types of training individuals have received in human subject’s protections.

If you are selected for an interview, you will be notified a few weeks prior to the site visit.  An educational handout will be provided to help you to prepare.

In addition, you can request an educational session with the Educational Specialist to learn more about the site visit and possible interview questions.

 

Are you liable for ClinicalTrials.gov Civil Money Penalties?

 On September 18, 2018, the Food and Drug Administration (FDA) announced a draft guidance titled Civil Money Penalties relating to the Clinical Trials.gov Data Bank. The guidance addresses how the FDA Centers (Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health, for drug, biological, and device products, respectively) identify whether parties have failed to submit required clinical trial registrations, results, or certifications, or knowingly submitted false or misleading information.

The guidance explains that when a responsible party has potentially failed to comply with its disclosure obligations, the FDA will send a Preliminary Notice of Noncompliance Letter, and allow the responsible party 30 days to make corrections. If the FDA determines that the responsible party has still failed to comply, the FDA may issue a Notice of Noncompliance, assess civil monetary penalties, or seek an injunction and/or criminal prosecution. The statutory maximum for civil penalties is “not more than $10,000 for all violations adjudicated in a single proceeding,” plus “not more than $10,000 for each day that the violation continues” past the 30-day notification period.

The deadline to submit your comments on this draft, electronically or by paper, is November 20, 2018.

  • To check if your trial meets the criteria for ClinicalTrials.gov registration and results posting set forth by the FDA, see the checklist found here. Applicable clinical trials include those evaluating at least one drug, biological, or device product regulated by the FDA.

 

  • If your NIH-funded study involves human subjects, check here to see if it meets the NIH’s broad definition of a clinical trial and will require registration and results posting. NIH defines a clinical trial as a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.
  • For questions about ClinicalTrials.gov, please contact Research Compliance Monitor, Ellen Ciesielski at 860-679-6004

 

Does NIH require you to register your basic science research in ClinicalTrials.gov?

 NIH has released a Request for Information (RFI), Registration and Results Reporting Standards for Prospective Basic Science Studies Involving Human Participants. This RFI seeks input from the scientific research community and the general public regarding how best to implement the NIH Policy on the Dissemination of NIH-Funded Clinical Trial Information (NOT-OD-16-149) for prospective basic science studies involving human participants. NIH is specifically interested in learning more about studies that meet the NIH definition of a clinical trial (NOT-OD-15-015) but are not defined as applicable clinical trials (42 CFR Part 11), and also meet the definition of fundamental research. You can submit your comments here by November 12, 2018.

 

Access to Investigational Drugs through  Clinical Trials, the Expanded Access Program, or  the Right-to-Try Law

An investigational drug is one that has been tested in a laboratory and in animals and is being studied in human for efficacy and safety. Investigational drugs are not yet approved by the Food and Drug Administration (FDA) for use in the general population.

There are various mechanisms through which patients may obtain access to an investigational drug. These mechanisms include 1) clinical trials 2) the FDA’s expanded access program or 3) the recently passed Federal law known as the Right to Try Law. Each of these mechanisms will be discussed below and then compared in table format.

Clinical Trials: The standard mechanism for obtaining access to an investigational drug is through participation in a clinical research trial. A clinical research trial is conducted in accordance with a formal research protocol and its purpose is to evaluate the effectiveness and safety of an investigational drug. To participate in a clinical trial the patient must have the disease or disorder that is being studied and must also meet any specific inclusion or exclusion criteria defined in the research protocol. Patients can search for clinical trials that are being conducted at https://clinicaltrials.gov/.

Expanded Access Program: If participation in a clinical trial is not possible, access to an investigational drug may be sought through the FDA’s Expanded Access Program (EAP).

Access through the EAP may be appropriate when all of the following criteria are met:

  • The individual must have a serious/immediately life-threatening disease or terminal condition.
  • There must not be a comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
  • The individual is unable to participate in a clinical trial.
  • Potential patient benefit justifies the potential risks of treatment.
  • Providing the drug to the patient will not interfere with the conduct of the clinical trial.

To apply for expanded access, the patient’s physician should first submit a request to the manufacturer of the investigational drug to obtain approval for the use of the product outside of the clinical trial setting. The manufacturer is under no obligation to approve such requests. If the manufacturer will provide the drug, the physician must then submit a request for approval to an Institutional Review Board (IRB) and to the FDA. An IRB is a committee that reviews the plan for the use of the drug to ensure that the rights and welfare of the recipient are protected. If FDA and IRB approval is provided, in the majority of cases the doctor must obtain informed consent from the individual who will receive the drug.  The individual’s consent is documented by signing an informed consent form that, among other things, describes the potential risks associated with the drug. More about the FDA’s Expanded Access Program is available at https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm

Right to Try Law: The Right-to-Try Law (RTT) was enacted on May 30, 2018. It is intended to provide terminally ill patients a more streamlined mechanism for access to investigational drugs. The Right to Try Law provides a right to request access to the drug. The manufacturer of the drug is under no obligation to grant such requests.

Like the EAP, the RTT law allows for the use of an investigational drug by a patient who has been diagnosed with a life-threatening disease or condition, who has exhausted approved treatment options, and who is unable to participate in a clinical trial involving the eligible investigational drug. An eligible investigational drug means a drug for which a Phase 1 clinical trial has been completed but the drug has still not been fully approved by the FDA. A Phase 1 clinical trial is done primarily to determine the dose of the drug that may be given safely to humans. Determining whether the drug is actually effective comes in later phases of the trial.

Unlike the EAP, the RTT law eliminates the need for approval from the FDA and Institutional Review Board and it provides the sponsor and physician protection from liability associated with harm caused by use of the investigational drug. While the RTT law does specify that the treating physician must obtain the informed consent of the patient, unlike the EAP, the RTT law does not specify the required elements of that informed consent.

Because the RTT law is fairly new, it is not yet clear whether this mechanism will be accepted by manufacturers of investigational drugs.

The following table describes the major similarities and differences of the three current processes by which seriously ill patients may obtain access to investigational drugs.

 

Clinical Trials Expanded Access Program Right –to Try-Law
Who can participate? Patients with a condition for which a drug is being developed who meet the inclusion criteria for the trial.

 

 Patients with a serious/immediately life-threatening disease or terminal condition, unable to participate in a clinical trial. Patients with a serious/immediately life-threatening disease or terminal condition, unable to participate in a clinical trial.

 
Investigational Drug Not FDA Approved Not FDA Approved Not FDA Approved, Phase 1 trial completed

 
FDA Approval Required Required Not Required
IRB Approval Required Required Not Required
Informed Consent Required and must include specific elements required by regulation

 

 Required and must include specific elements required by regulation

 

 Required, but no specific elements are identified

 

 
Manufacturer Obligated to Provide the Investigational Drug Yes, to those individuals  who provide informed consent to participate in the trial No No
Liability waived for any risk associated with Drug No No Yes

 

How can I learn about research studies that are looking for volunteers?

To find ongoing studies that are currently recruiting participants at UConn, you can explore the following online listings:

To search for trials recruiting nationally, please check out the websites below: