Making Treatment Less Rare for Rare Disease Patients

Waiting is a fact of life for patients with rare diseases such as cystic fibrosis.

Many of these men and women, whose disease causes wheezing and frequent lung infections, live waiting for new and effective medications.  They have to delay activities due to symptoms that make breathing a struggle.  They wait for treatment, fighting through health plan barriers such as prior authorization or awaiting the decision of opaque bureaucracies such as Medicaid pharmacy and therapeutics committees.

This Rare Disease Awareness Day, the Cystic Fibrosis Engagement Network is explaining at least one way to reduce the wait.


In a new video, “Why FDA Fast Tracking Matters to Cystic Fibrosis Patients,” the organization examines how fast tracking streamlines the drug approval process for treatments that fill an unmet need in addressing a serious illness.  Fast tracking entails close, frequent communication between regulators and the manufacturers of drugs that meet certain criteria:

  • The new drug is expected to be much more effective than what is currently available, or it would become the first to treat a certain disease
  • The condition will become more serious if left untreated
  • The new drug will likely improve patients’ day-to-day functioning
  • The new drug could help patients live longer.

Emerging treatments don’t work for all cystic fibrosis patients, the video acknowledges, but for those who can be treated, fast tracking is an important way to shorten patients’ wait for innovative medicine and the quality-of-life improvements it provides.

The Cystic Fibrosis Engagement Network is an educational and advocacy organization focused on policy issues impacting patients’ access to optimal care.

The fast track process was instrumental in approving a new drug that treats the underlying cause of cystic fibrosis, instead of just its symptoms.  The process benefits patients with other rare diseases as well.

And those patients are speaking out in force this week in recognition of Rare Disease Awareness Day.  From concerts to lecture series to sporting events, patients are acknowledging their struggles and celebrating their solidarity with 359 events across 77 countries.

Feel free to share the new fast tracking video from @EngageCF and to join the conversation online using #ShowYourRare.

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