Regulatory Focus™ > News Articles > Fourth Patient-Focused Drug Development Meeting Announced With Focus on Common Questions

Fourth Patient-Focused Drug Development Meeting Announced With Focus on Common Questions

Posted 18 July 2013 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) has today announced the details of its fourth patient-focused drug development meeting, this time focusing on narcolepsy, a condition characterized by an patient's inability to regulate sleep habits.

Background

Under the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, FDA was charged with setting up a "Patient-Focused Drug Development Initiative," which is set to seek out opinions from patients in 20 different disease areas over the next five years.

"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 explained in a Federal Register posting announcing the program in September 2012.

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 that 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.

Meetings

FDA has already held three meetings concerned with patient-focused drug development: lung cancer, chronic fatigue syndrome (CFS), and human immunodeficiency virus (HIV).

Similar to prior meetings, FDA has assembled a list of questions it is looking for answers to. While noting that "there is no cure for narcolepsy," medications and lifestyle modifications can help patients live more fulfilling lives by managing their respective symptoms. The question for FDA, then, is what patients believe to be meaningful outcomes short of a cure.

And, as with prior meetings, the questions asked of patients follow similar-albeit with a unique perspective-trends.

The meeting will be held on 24 September 2013 at FDA's White Oak campus in Silver Spring, MD.

 

FDA's Questions for Patients Based on Disease

Narcolepsy

Lung Cancer

HIV

CFS

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 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?

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 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 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 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 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 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 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 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)?


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 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?

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?




How well do these therapies improve your ability to do specific activities that are important to you in your daily life?





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?




Tags: patients

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