Regulatory Focus™ > News Articles > New Patient-Focused Drug Development Meeting to Concentrate on Neurologic Disorders

New Patient-Focused Drug Development Meeting to Concentrate on Neurologic Disorders

Posted 24 April 2014 | By Alexander Gaffney, RAC

Since the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012, the US Food and Drug Administration (FDA) has been publicly meeting with patients who suffer from debilitating conditions without adequate treatment options in the hopes of better understanding their wants and needs in the drug development process.

Now the agency is gearing up for its eighth meeting for patients with neurologic manifestations of inborn errors of metabolism, illustrating an abbreviated list of questions also asked of other disease areas.

Background

The patient-centered drug development program was the brainchild of disease advocate groups, which convinced legislators to include it in FDASIA. Under Section X of the act, FDA is instructed to assess its risk-benefit decision making as it relates to disease areas where treatment options are less than optimal. Patients may be willing to accept more risk if the alternative to treatment is worse, proponents of the section hypothesized, and FDA was ordered to facilitate a program that assesses that tolerance for risk on a disease-wide level, instead of doing so on a piecemeal basis for individual products.

"The human drug and biologic review process could benefit from a more systematic and expansive approach to obtaining input from patients who experience a particular disease or condition," FDA noted in its Federal Register posting announcing the program.

Practically, the program means FDA will start to assess the benefit-risk paradigms that each patient group is willing to accept in return for a treatment. A patient group suffering from an under-treated but otherwise medically benign condition might be less willing to accept high levels of risk in return for an effective treatment. Conversely, those suffering from a disease with few or even no recognized treatment options might be more willing than most to accept a drug that might either be less effective or more dangerous than FDA's benefit-risk paradigm ordinarily would permit.

The point, however, is for FDA to determine this framework in tandem with patients. The agency recently launched a new Patient Network website aimed in part at educating patients about FDA's regulatory process so that they can better engage with regulators at these meetings and at other venues.

New Meeting

Previously, FDA has announced meetings around seven different disease areas: chronic fatigue syndrome (CFS), HIV, lung cancer, narcolepsy, fibromyalgia, sickle cell disease (SCD) and pulmonary arterial hypertension (PAH).

Now FDA is preparing to ask questions of patients with not just a single rare disease, but common causal factors. FDA's eighth meeting will focus on neurologic manifestations of inborn errors of metabolism, a range of genetic disorders which cause a buildup of harmful metabolites due to a faulty enzyme.

Diseases related to the condition include phenylketonuria, lysosomal storage disorders, Wilson disease, and others.

As with other diseases under FDA's patient-focused drug development program, the agency has listed a number of questions it intends to ask of patients at its meeting.

While all but one of the questions are shared with those asked of other diseases, that new question is notable in that it relates to the process of informed consent, which FDA defines at length. The question, which asks about what "important considerations" a patient might take into account when enrolling their child into a study, is broad enough that it will likely be found on future patient-focused drug development questionnaires.

FDA Question on Patient Perspectives: Informed Consent
The process of informed consent is an important way for researchers to communicate the purpose of a clinical trial and the potential benefits and risks of the trial so that people can make an informed decision about whether to participate. Informed consent also ensures that parents are fully informed and are given opportunities to ask questions about the clinical trial. In addition to informed consent from parents, assent from children may also be needed. Assent is the term used to describe when a child agrees to be in a clinical trial. Among other considerations, children should be old enough to understand basic facts about the clinical trial in order to provide assent to participate.
In the informed consent process, what are important considerations to take into account in cases when the potential participant is a child? For example, how should the informed consent clearly communicate to the patient the potential benefits and risks of a study?

The set of questions is also notable for its brevity-just six questions, or 50% fewer than any other disease type thus far. This may represent FDA becoming better at focusing its questions around "key" areas capable of yielding more impactful answers that can be incorporated into new regulatory risk acceptance models.

Questions Asked of Each Group by FDA
CFSPAHNarcolepsyLung CancerFibromyalgiaSCDHIVInborn Errors of Metabolism
91012151516166

A complete listing of all questions asked by FDA of each respective disease may be found below

FDA's Questions for Patients Based on Disease
Neurologic Manifestations of Inborn Errors of MetabolismPulmonary Arterial Hypertension (PAH)Sickle Cell Disease (SCD)FibromyalgiaNarcolepsyLung CancerHIVCFS
Of all the signs or symptoms that you/your child experiences because of the condition, which 1-3 neurologic/neuropsychological signs and/or symptoms have the most significant impact on your/your child's life?Of all the symptoms that you experience because of your condition, which one to three symptoms have the most significant impact on your life?Of all of the ways that sickle cell disease affects your health, which one to three effects have the greatest impact on your life?Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact on your life?Of all the symptoms that you experience because of your condition, which one to three symptoms have the most significant impact on your life?Of all the symptoms that you experience because of your lung cancer, which 1 to 3 symptoms have the most significant impact on your daily life?Of all the symptoms that you experience because of your condition or because of your therapy or treatment, provide one to three symptoms that have the most significant impact on your life?What are the most significant symptoms that you experience resulting from your condition? What are the most negative impacts on your daily life that result from your condition and its symptoms?
What are you/your child currently doing to help treat the condition or its signs/symptoms?What are you currently doing to help treat your condition or its symptoms?Are you currently using any prescription medicines or medical treatments to prevent or treat any negative effects of your sickle cell disease?What are you currently doing to help treat your condition or its symptoms?What are you currently doing to help treat your condition or its symptoms?Are you currently undergoing any cancer treatments to help reduce or control the spread of your lung cancer? How well do these treatments manage these symptoms?What are you currently doing to help manage your HIV and any symptoms you experience because of your condition or other therapies?What treatments are you currently using to help treat your condition or its symptoms?
How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?How does sickle cell disease affect your life on an "average" day? How does sickle cell disease affect your life on the "worst" days, such as days when you have a pain crisis or have to be hospitalized for some reason?How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?What changes have you had to make in your life because of your condition? How does the condition affect your daily life on the best days and worst days?
What are the most significant downsides to your current treatments, and how do they affect your daily life?What are the most significant downsides to your current therapies, and how do they affect your daily life?How do these downsides affect your daily life?
What specific parts of your sickle cell disease do these treatments address?What specific symptoms do your treatments address?What specific symptoms do your therapies address?What supportive care treatments, if any, are you taking to help improve or manage the symptoms you experience because of your lung cancer?What specific symptoms do your therapies or treatments address?What specific symptoms do your treatments address?
How has your treatment regimen changed over time, and why?How has your treatment regimen changed over time, and why?How has your treatment regimen changed over time, and why?How long have you been on treatment and how has your treatment regimen changed over time?How has your treatment regimen changed over time and why?
How well does this current treatment regimen treat the neurological symptoms of your/your child's disease?How well does your current treatment regimen treat the most significant symptoms of your disease?How well do these treatments work for you? For example, how well do they reduce your number of pain crises, hospitalizations, or strokes? How well do they help you manage your pain, breathing difficulties, or other health effects?How well does your current treatment regimen treat the most significant symptoms of your disease?How well does your current treatment regimen treat the most significant symptoms of your disease?How well do these treatments manage these symptoms?How well does your current treatment regimen treat any significant symptoms of your condition?How well does your current treatment regimen treat the most significant symptoms of your disease?
Are there specific activities that are important to you/your child but that you/your child cannot do because of these neurologic/neuropsychological signs or symptoms?Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition?Are there activities that you cannot do at all or as well as you would like on these "worst" days?Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition?Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition?Are there specific activities that are important to you but that you cannot do at all, or as fully as you would like, because of lung cancer?Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition?Have these treatments improved your daily life (for example, improving your ability to do specific activities)?
How have your/your child's neurologic/neuropsychological signs or symptoms changed over time?How have your condition and its symptoms changed over time?How well do these treatments work for you?How well have these treatments worked for you as your condition has changed over time?How well have these therapies worked for you as your condition has changed over time?How well do these treatments manage these symptoms?How well have these treatments worked for you as your condition has changed over time?How well have these treatments worked for you as your condition has changed over time?
What are the biggest problems with these treatments? How do these problems affect your daily life?What are the most significant downsides to your current treatments, and how do they affect your daily life?What do you consider to be the most significant downsides of these treatments?What are the most significant downsides to your current therapies or treatments, and how do they affect your daily life?What are the most significant downsides of these treatments (for example, specific side effects)?
What parts of your sickle cell disease do your current treatments not treat at all or not as well as you would like?Are there symptoms that your current treatment regimen does not address at all, or does not treat as well as you would like?Are there symptoms that your current regimen does not address at all, or does not treat as well as you would like?
Assuming there is no complete cure for your/your child's condition, what specific attributes would you look for in an ideal treatment for the condition?Assuming there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?Assuming that there is no cure for sickle cell disease, what specific things would you look for in an ideal treatment?What specific things would you look for in an ideal treatment for your condition?Assuming there is no complete cure for your condition, what specific things would you look for in an ideal therapy for your condition?Assuming there is currently no complete cure for your condition, what specific things would you look for in an ideal therapy or treatment to manage your condition?
In the informed consent process, what are important considerations to take into account in cases when the potential participant is a child?
Have the medications for pulmonary arterial hypertension made a difference to you? If so, in what ways?
Are there activities that you cannot do at all or as well as you would like on these "average" days?
Are there activities that you cannot do at all or as well as you would like on these "worst" days?
What specific concerns do you have about sickle cell disease in infants and young children, in adolescents and young adults, or in older adults?
Besides prescription medications, what else do you do to prevent or treat any negative effects of your sickle cell disease? What are the biggest problems with these treatments?
If you had the opportunity to consider participating in a clinical trial studying experimental treatments for sickle cell disease, what things would you consider when deciding whether or not to participate?
How have your condition and its symptoms changed over time?How have your symptoms changed over time?
Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse?Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse?
How well do these treatments improve your ability to do specific activities that are important to you in your daily life?How well do these therapies improve your ability to do specific activities that are important to you in your daily life?
What worries you most about how sickle cell disease could affect your health in the future?What worries you most about your condition?
When thinking about your overall goals for treatment, how do you weigh the importance of prolonging your life versus improving the symptoms you experience because of your lung cancer?
How well do these treatments manage these symptoms?
What factors do you take into account when making decisions about using treatments to help reduce or control the spread of your lung cancer?
What information on the potential benefits of these treatments factors most into your decision?
How do you weigh the potential benefits of these treatments versus the common side effects of the treatments?
How do you weigh potential benefits of these treatments versus the less common but serious risks associated with the treatments?

The meeting will take place on 10 June 2014 at FDA's White Oak Campus in Silver Spring, MD.

 

Federal Register notice.


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