Patient-Focused Drug Development

FDA Dockets open until November 1, 2012   Request Celiac disease be added to list!!!

Read the CSA submission 

FDA seeking  public comment on nomination of diseases meeting the following criteria for disease selection.
Celiac Disease fits ALL of the criteria include the following:

  • Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;  
  • Disease areas that reflect a range of severity; 
  • Disease areas for which aspects of the disease are not formally captured in clinical trials;      Few clinical trials in celiac disease
  • Disease areas that have a severe impact on identifiable sub-populations (such as children or the elderly) 
  • Disease areas that represent a broad range in terms of size of the affected population; 
  • Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives. 

On October 25, FDA will host a "Patient-Focused Drug Development" public meeting from 9 a.m. to 12:30 p.m. ET at its White Oak Campus in Silver Spring, MD.  FDA is also currently accepting public comment online on the topics that will be discussed at this hearing.

The public meeting is open to all..  The deadline for submitting online comments is Thursday, November 1, 2012.  Click here  to read about this in the Federal Register, sign up for the hearing and/or submit online comments.  

Public input is being sought in line with performance commitments FDA made as part of the recent reauthorization of the Prescription Drug User Fee Act.  Specifically, FDA is seeking to provide for a more systematic approach to obtaining patient perspective on the severity of various types of diseases and currently available treatments for those diseases.

 FDA  preliminary list of nominated disease areas for the patient-focused drug development initiative -- NOMINATE CELIAC DISEASE  

  • Pulmonary arterial hypertension
  • Heart failure.
  • Primary glomerular diseases.
  • Narcolepsy.
  • Huntington's Disease.
  • Depression.
  • Autism.
  • Peripheral neuropathy.
  • Fibromyalgia.
  • Obesity.
  • Nocturia.
  • Chronic fatigue syndrome.
  • Irritable bowel syndrome.
  • Inflammatory bowel disease.
  • Alopecia areata.
  • Diabetic ulcers.
  • Female sexual dysfunction.
  • Interstitial cystitis/painful bladder syndrome.
  • Fracture healing.
  • Diabetic foot infections.
  • Hepatitis C.
  • HIV.
  • Patients who have experienced an organ transplant.
  • Sickle cell disease.
  • Chronic graft versus host disease.
  • Amyloidosis.
  • Aplastic anemia.
  • Melanoma.
  • Lung cancer.
  • Cancer and young patients.
  • Cancer treatment in pregnancy.
  • Cancer and sexual dysfunction.
  • Cancer and depression.
  • Clotting disorders (e.g., hemophilia A (factor VIII deficiency) and von Willebrand disease).
  • Thrombotic disorders (e.g., antithrombin deficiency and protein C deficiency).
  • Primary humoral immune deficiencies (e.g., common variable immune deficiency).
  • Neurologic disorders treated with immune globulins (e.g., chronic inflammatory demyelinating polyneuropathy).
  • Hereditary angioedema.
  • Alpha-1 antitrypsin deficiency.