Patient-Focused Drug Development Tracker

Posted 01 July 2015 By Alexander Gaffney, RAC

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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.

Those meetings have raised novel questions for regulators, who for the first time are asking patients and patient groups to explain how diseases affect their daily lives, and what roles treatments do—and do not—play in helping them to manage their diseases and symptoms.

Last Updated: 1 July 2015

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Meetings Held and Scheduled

To date, FDA has announced 15 patient-focused drug development meetings:

  1. Chronic Fatigue Syndrome (CFS) and Myalgic Encephalomyelitis
  2. Pulmonary Arterial Hypertension (PAH)
  3. Narcolepsy
  4. Lung Cancer
  5. Human Immunodeficiency Virus (HIV)
  6. Fibromyalgia
  7. Sickle Cell Disease (SCD)
  8. Neurological manifestations of inborn errors of metabolism
  9. Idiopathic Pulmonary Fibrosis (IPF)
  10. Hemophilia A, Hemophilia B, von Willebrand disease, and other heritable bleeding disorders
  11. Female sexual dysfunction (FSD)
  12. Chronic Chagas disease
  13. Breast Cancer
  14. Functional Gastrointestinal (GI) Disorders
  15. Alpha-1 antitrypsin deficiency

For information on those meetings, including meeting transcripts, please see   FDA's patient-focused drug development webpage.

Future Meetings

FDA has also announced one other disease categories it plans to hold meetings on prior to 2016:

  1. Parkinson's disease and Huntington's disease (announced, but details have not yet been released).

In addition, FDA has already begun planning its  next batch of patient-focused drug development meetings. In July 2015, it released a list of eight diseases and conditions it plans to hold meetings for under the PFDD program between FY 2016-2017:

  1. Alopecia areata
  2. Autism
  3. Hereditary angioedema
  4. Non-tuberculous mycobacterial infections
  5. Patients who have received an organ transplant
  6. Psoriasis
  7. Neuropathic pain associated with peripheral neuropathy
  8. Sarcopenia

Other diseases and conditions are likely to be considered under the next reauthorization of PDUFA in FY 2018-2022.

Questions at a Glance: Questions Asked of Each Group by FDA

CFSPAHNarcolepsyLung CancerFibromyalgiaSCDBreast CancerAlpha-1 antitrypsin deficiency
910121515161512
HIVInborn Errors of MetabolismIPFBlood / Platelet DisordersFemale Sexual DysfunctionChronic ChagasFunctional GI Disorder  
166812171016 

 

Questions: In Depth Comparisons

An at-a-glance listing of FDA's most common questions for patients may be found below. (Table may overflow page—scroll if necessary)

FDA's Most Commonly Asked Questions for Patients

QuestionsAlpha-1 Antitrypsin DeficiencyFunctional Gastrointestinal (GI) DisorderBreast CancerChronic Chagas DiseaseFemale Sexual DysfunctionBlood / Platelet DisordersIdiopathic Pulmonary Fibrosis (IPF)Neurologic Manifestations of Inborn Errors of MetabolismPulmonary Arterial Hypertension (PAH)Sickle Cell Disease (SCD)FibromyalgiaNarcolepsyLung CancerHuman Immunodeficiency Virus (HIV)Chronic Fatigue Syndrome (CFS)
Of all the symptoms that you experience because of your condition, which 1 to 3 symptoms have the most significant impact on your life?XXXXXXXXXXXXXXX
What are you currently doing to help treat your condition or its symptoms?XX XXXXXXXXXXXX
How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days? X  X X XXX   X
What are the most significant downsides to your current treatments, and how do they affect your daily life?XXXXXXX X  XX  
Are there specific activities that are important to you, but that you cannot do at all, or as well as you would like, because of your condition?XXXX XXXXXXXXXX
How have your condition and its symptoms changed over time?XX X XXXXXXXX  
What specific symptoms do your treatments address? XXX     XXXXXX
How has your treatment regimen changed over time, and why?

 

 

 

 X XXX  X XX XX
How well does your current treatment regimen treat the most significant symptoms of your disease? XX X  XXXXXXXX
What are the most significant downsides to your current treatments, and how do they affect your daily life?  X      XX XXX
How well have these treatments worked for you as your condition has changed over time? X        XX XX
What aspects of your condition are not improved by your current treatment regimen?X X  X   X  XX 
Assuming there is no complete cure for your condition, what specific things would you look for in an ideal therapy for your condition?XX XXXXXXXXX X 
Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse? X XX     XX   
What worries you most about your condition?XX XXX   XX    
How well do these treatments work for you?XX  XX      X  

 

A complete listing of all questions asked by FDA of each respect disease may be found below. (Table may overflow page—scroll if necessary)

FDA's Questions for Patients Based on Disease

Alpha-1 Antitrypsin DeficiencyFunctional Gastrointestinal (GI) DisordersBreast CancerChronic Chagas DiseaseFemale Sexual DysfunctionBlood / Platelet DisordersIPFNeurologic Manifestations of Inborn Errors of MetabolismPulmonary Arterial Hypertension (PAH)Sickle Cell Disease (SCD)FibromyalgiaNarcolepsyLung CancerHIVCFS
Of all of 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 condition, which one to three symptoms have the most significant impact on your life?Of all the symptoms that you experience because of your breast cancer, which one to three symptoms have the most significant impact on your daily life?Do you experience symptoms because of your condition? If so, of all the symptoms that you experience, which one to three symptoms have the most significant impact on your life?Of all the symptoms that you experience because of your condition, which 1 to 3 symptoms have the most significant impact on your life?Of all of the symptoms that you experience because of your condition, which one to three symptoms (bleeding or non-bleeding) 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 signs or symptoms that you / your child experiences because of the condition, which 1-3 neurologic / neuro-psychological 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 currently doing to 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 breast cancer?What are you currently doing to help treat your condition?What are you currently doing to help treat your condition or its symptoms?What are you currently doing to treat your condition or its symptoms?What are you currently doing to help treat your condition or 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 do your symptoms and their negative impacts on your sexual experiences compare between your "best days" and your "worst days"? How do your symptoms and their negative impacts affect your daily life on the best days?

 

 

 

How do your symptoms and their negative impacts affect your daily life on the worst  days?

 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 disadvantages or complications of your current treatments, and how do they 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 treatments, and how do they affect your daily life?Are there any downsides to the treatments you have used?What are the most significant disadvantages or complications of your current treatments, and how do they affect your daily life?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 treatments, and how do they affect your daily life? 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 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 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 specific symptoms do your treatments address?What specific symptoms do your treatments address?What specific symptoms do your treatments address?     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 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 has your treatment regimen changed over time, and why?

 

 

 

How has your treatment 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 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 your current treatments specifically treat the most significant symptoms of your condition?  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?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, but that you cannot do at all, or as well 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 breast 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? Are there specific activities that are important to you, but that you cannot do at all, or as well 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 / your child but that you / your child cannot do because of these neurologic / neuro-psychological 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 condition and its symptoms changed over time? How has your condition and its symptoms changed over time? How have your condition and its symptoms changed over time?

 

 

 

 How have your condition and its symptoms changed over time?How has your condition and its symptoms changed over time?How have your / your child's neurologic / neuro-psychological 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 aspects of your condition are not improved by your current treatment regimen?  Are there symptoms that your current treatment regimen does not address at all, or does not treat as well as you would like?  What aspects of your condition are not improved by your current treatment regimen?   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? 
If you could create your ideal treatment, what would it do for you (i.e., what specific things would you look for in an ideal treatment)? Assuming there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition? What specific things would you look for in an ideal treatment for your condition?What specific things would you look for in an ideal treatment for your condition?If you could create your ideal treatment, what would it do for you?Because there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?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 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 "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, what things would you consider when deciding whether or not to participate?    If you had the opportunity to consider participating in a clinical trial studying experimental treatments, what things would you consider when deciding whether or not to participate?   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?     
 Do your symptoms come and go or are they ongoing? If so, do you know of anything that worsens your symptoms? Do your symptoms come and go? If so, do you know of anything that makes your symptoms better or worse?Do your symptoms wax and wane 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 therapies improve your ability to do specific activities that are important to you in your daily life?        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 your condition?What worries you most about your condition? What worries you most about your condition?What worries you most about your condition?

 

 

 

What worries you most about your condition?   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 breast cancer?         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 work for you?How well do these treatments stop or slow the progression of your condition?How well do these treatments manage these symptoms? How well have your treatments improved your sexual experience?How well do these treatments work for you?      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 breast cancer?         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?         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 the potential benefits of these treatments versus the common side effects of the treatments?  
  How do you weigh the potential benefits of these treatments versus the less common but serious risks associated with the treatments?         How do you weigh potential benefits of these treatments versus the less common but serious risks associated with the treatments?  
What treatment has had the most positive impact on your life?     What treatment has had the most positive impact on your life?         
 Have you received a diagnosis of a functional GI disorder from a health care provider? If so, please state the condition.  Have you ever received a diagnosis from a healthcare provider of sexual interest/arousal disorder, hypoactive sexual desire disorder, or sexual arousal disorder?          
    How was the diagnosis made?          
    Which symptoms vary the most, and in what ways?          
    Do the changes in your symptoms typically happen over a period of minutes, hours, days, weeks, or months?          
    If you were asked today to accurately rate how good or how bad your symptoms have been over time, would you be able to accurately remember how your symptoms felt one day ago? Over the past 3 days? Over the past week? Over the past 2 weeks? Over the past 3 weeks? Over the past month?          
    Is there anything else that you believe makes your symptoms better? Is there anything that you believe makes your symptoms worse?          
    Overall, have you experienced your condition and its symptoms getting progressively worse, improving, or remaining stable over the past few years?          
  For context, how long ago was your diagnosis of breast cancer? Is your cancer currently in only one area or has it spread to other parts of the breast or lymph nodes or outside of the breast?            
 How well have these treatments worked for you as your condition has changed over time?             
  What supportive care treatments, if any, are you taking to help improve or manage the symptoms you experience because of your breast cancer?            

 

 

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Categories: Biologics and biotechnology, Drugs, Orphan products, News, US, CBER, CDER

Tags: Patients, Patient-Focused Drug Development, FDASIA, View of Patients, PFDD

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