Participate

There are many factors you should consider when deciding whether or not to enroll in a clinical trial. It’s important to understand how the process works and what your family should expect before you begin.

Finding a Trial

Our website provides a list of trials that are currently recruiting for SMA, which is regularly updated with new trial information. The FDA site, www.clinicaltrials.gov, is the best way to get more details about a particular trial, or to see the progress of trials that are in stages other than recruitment.

Other sources include our Cure SMA news section, through which we regularly announce new trials. Your healthcare provider may also have information on trials that are recruiting participants.

Information About Participating in a Trial

The following is a summary of the issues you may consider when choosing to participate in a clinical trial. For more detailed information on clinical trial participation, download our care series booklet, Learning About Clinical Trials.

Benefits and Risks

A well-designed and well-executed trial offers many benefits. By participating, you can:

  • Advance knowledge about SMA, contribute to the development of a potential treatment, and help others affected by SMA in the future.
  • Potentially receive access to an investigational drug.
  • Receive appointments with an SMA study team.
  • Receive trial-related care, monitoring, and assessments.

Possible risks:

  • There may be unpleasant, serious, or even life-threatening side effects to experimental drugs.
  • The experimental drug might not be effective.
  • The protocol may require more of your time and attention than a standard course of treatment, including trips to the study site, more drugs, hospital stays, or complex dosage requirements.

Questions to Ask

When deciding to participate, learn as much as possible about the clinical trial, the care expected, the cost, and the team that will be conducting the trial. We recommend you write down a list of questions to ask before meeting with the study doctors.

Here are some potential questions you might ask:

  • What is being studied?
  • Of researchers are studying an investigational drug, why do they believe it may be effective for SMA?
  • How long will participation last?
  • How often will I have to visit the hospital or clinic? Will any of these visits require an overnight stay?
  • Is there a chance of receiving a placebo or sham?
  • What types of tests and medical procedures will be performed?
  • What are the possible risks and benefits of participation?
  • Who will oversee my healthcare while participating?
  • Will the results of the clinical trial be available to participants?
  • Who will pay the costs associated with participation?
  • Will I be reimbursed for other expenses?
  • Is travel support included?
  • Is there a planned extension trial?

You should also discuss your decision with your primary physician, family members, and, if needed, a counselor, therapist, or spiritual advisor.

During the Trial

  • Follow all instructions given by the study team.
  • Attend all scheduled visits.
  • Complete questionnaires about your health status (or your family member's health status) between visits.
  • Tell the principal investigator of any new health-related problems. Even if you don't consider them to be caused by the clinical trial or the investigational drug, any small change is very important to report.
  • Tell the principal investigator about any new medications or changes in the doses or frequency of medication.
  • Be mindful about discussing the clinical trial with other participants, including whether you think you or your family member may be receiving a placebo or sham.

Expanded Access

If you or your family member is not eligible to participate in a clinical trial, expanded access—also called “compassionate use”—may be an option to explore. In the United States, the FDA allows some drug companies to provide investigational drugs to patients outside of a clinical trial—however, expanded access programs are highly regulated and patients must meet several criteria in order to be eligible.

The FDA must approve the investigational drug for expanded access based on preliminary safety and efficacy data. In addition, the drug company must be willing to make it available for expanded access. Not all drug companies may want to make an investigational drug available or have the ability to do so based on:

  • lack of safety and efficacy data
  • the risks that it could pose to ongoing controlled clinical trials
  • cost (the investigational drug may be too expensive and time-consuming to make)
  • manufacturing (the ability to produce the investigational drug may be limited, especially for smaller drug companies)

In developing an expanded access program, a drug company may consider:

  • whether they need to take resources from the drug development program, which may slow down the approval timeline
  • whether early access to the investigational drug could make phase 2 and 3 clinical trials more difficult to conduct
  • manufacturing capacity (and whether it may limit the availability for clinical trial participants)

Once an investigational drug has been approved for expanded access by the FDA and regulatory authorities, patients still need to meet criteria to be considered to receive it. These may include:

  • If the patient is not eligible to participate in a clinical trial because of age, health problems, distance to the trial location, or other factors.
  • If the patient has a serious or life-threatening disease, like SMA, for which there is no approved treatment.
  • If the doctor believes that the potential benefits of the investigational drug will outweigh any potential risks to the patient.

Costs Associated with Expanded Access

Cure SMA encourages parents to contact their individual health insurance companies to obtain information about potential coverage for costs associated with participation in an Expanded Access Program (EAP). While federal laws–including the Affordable Care Act–do not require that insurance plans cover costs associated with participation in an EAP, some insurance companies may cover the related costs. Cure SMA does not have information on any specific insurance or health plan and therefore cannot guarantee coverage is or may be available.

In the event you are unable to obtain answers from your individual health insurer or need additional assistance, we recommend reaching out to your state insurance ombudsmen who may be able to assist you in navigating state-specific coverage issues.  Many hospitals have case workers and case managers who can assist you in navigating your situation and assist with interfacing with your health insurance company. Also, your federally elected officials have case workers in their district offices who can assist you with respect to coverage under the Medicaid or State Children’s Health Insurance programs.  To find who represents you and contact their local offices for assistance visit: http://www.house.gov/ and enter your zip code in the box in the upper right hand corner.

Also, the following organizations provide support to individuals and families in navigating health insurance matters and they may be able to assist you:

Patient Advocate Foundation (PAF)

PAF is a great resource for case management support and insurance navigation. As well, the PAF Pediatrics Resource Center is potential resource for assistance for parents with children who have SMA. Their website also includes a National Financial Resource Directory that may be helpful for you. PAF can be contacted via phone at (800) 532-5274 or http://www.patientadvocate.org/help.php. PAF also provides a list of additional supportive resources at: http://www.patientadvocate.org/resources.php and through a mobile app at: http://www.patientadvocate.org/about.php?p=956

Health Advocate

Some employers offer Health Advocate as a benefit resource for employees and their families. Ask your employer if this is available to you: http://healthadvocate.com/

Medicare Advocacy Center (for patients who are Medicare eligible)

http://www.medicareadvocacy.org/medicare-info/medicare-coverage-appeals/

FDA Resources

Each country has its own regulatory authority with its own regulations, or laws, for conducting a clinical trial. The regulatory authority reviews and approves the protocol, and ensures that the clinical trial follows national regulations. In the United States, the regulatory authority is the Food and Drug Administration (FDA).

In addition to regulating clinical trials, the FDA has the responsibility to review all new drug applications (NDAs). If a drug developer has evidence from its early tests and preclinical and clinical research that a drug is safe and effective for its intended use, the company can file an NDA to market the drug. The FDA review team thoroughly examines all submitted data on the drug and makes a decision to approve or not to approve it. The FDA also reviews all requests for expanded access.

The following are resources from the FDA or about the FDA's involvement in these areas:

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