Section X, First Class: FDA Selects 20 Diseases for Special Regulatory Treatment

Posted 12 April 2013 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) has announced its inaugural class of disease areas that it will enroll into a new "Patient-Focused Drug Development" program that aims to construct a new development paradigm that more closely involves patients in the regulatory review process.


Patient groups have long sought greater interaction with, if not outright influence on, regulators. In general, those same groups have expressed that patients of a particular disease are often more tolerant of certain risks, particularly when faced with few good treatment options in a rare or neglected disease area.

In light of those concerns, legislators included a provision (Section X) in the 2012 FDA Safety and Innovation Act (FDASIA) that instructs FDA to assess its risk-benefit decision making as it relates to disease areas where treatment options are less than optimal, and to facilitate a program that assesses 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 a September 2012 Federal Register posting announcing the program.

That same notice explained that initially FDA would seek out opinions from patients in 20 different disease areas over the next five years, with each disease area meriting its own public meeting. Topics are set to include the severity of the disease, the spectrum of severity, the impact of the disease on patients, what patients perceive to be beneficial outcomes and the benefit of existing treatment regimens.

Deluge of Comments

FDA said it received a massive number of comments on the proposal: 4,500 comments in relation to more than 90 different disease areas.

"Many comments discussed the impact of the disease on daily life and the symptoms that were most concerning to patients," regulators explained. "Others addressed lack of treatment options or the nature of specific treatments."

Still, several disease areas rose to the top, with approximately half of all comments addressing lung cancer, narcolepsy and interstitial lung disease. Other areas that received a "significant" number of comments included migraine headaches, pulmonary fibrosis, amyloidosis, chronic fatigue syndrome, Lou Gehrig's disease (ALS), chronic obstructive pulmonary disease, lysosomal storage disorders, peripheral neuropathy, dystonia and fibromyalgia.

These comments, FDA said, were eye-opening, allowing it to better understand "the aspects of diseases that are not formally measures in clinical trials as well as cases where available therapies do not directly impact the aspects of disease that matter most to patients."

Section X, First Class

So which diseases make up the inaugural class of Section X?

  1. Alpha-1 antitrypsin deficiency
  2. Breast cancer
  3. Chronic Chagas disease
  4. Female sexual dysfunction
  5. Fibromyalgia
  6. Hemophilia A/B
  7. Von Willebrand disease and other heritable bleeding disorders
  8. HIV
  9. Idiopathic pulmonary fibrosis
  10. Irritable bowel syndrome
  11. Gastroparesis
  12. Gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors
  13. Lung cancer
  14. Myalgic encephalomyelitis/chronic fatigue syndrome
  15. Narcolepsy
  16. Neurological manifestations of inborn errors of metabolism
  17. Parkinson's disease
  18. Huntington's disease
  19. Pulmonary arterial hypertension
  20. Sickle cell disease.

So far, only one meeting is scheduled, a 25-26 April 2013 meeting on Chronic Fatigue Syndrome that was announced last month. Other disease meetings will be made available on an FDA webpage as they are scheduled, FDA explained.

Diseases not included in the initial wave of 20 diseases are set to be included in a second wave taking place in 2016 and 2017.

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