For those with cystic fibrosis who struggle to breathe, it’s a no-win situation.
Medicaid provides health insurance for about 45 percent of cystic fibrosis patients. Without access to regular medication, their lung function may decrease even more quickly than the 1-3 percent rate at which it already decreases, on average, each year. Not surprisingly, cystic fibrosis comes with debilitating symptoms, such as chronic coughing.
The cystic fibrosis community received news worth celebrating this week.
The Food and Drug Administration approved a new drug that helps address the underlying cause of cystic fibrosis, not simply its symptoms. The drug, a combination of tezacaftor/ivacaftor, is for patients aged 12 and older with certain cystic fibrosis mutations.
How do you put a price tag on a drug that offers life-altering results – but for a small group of patients? The question is becoming increasingly relevant with the rise of innovative, targeted medicine. And it’s at the heart of current debate about how the Institute for Clinical and Economic Review’s value framework applies to orphan drugs.
ICER, as it’s called, is a drug price analysis group. Using its trademark value framework, it assigns drugs a value benchmark price based upon long-term cost effectiveness and short-term budget impact. Recent ICER reports have generated price points for drugs that treat rheumatoid arthritis, chronic pain and other conditions.
This summer ICER released a proposal on how it might alter its framework when evaluating drugs for “ultra-rare” conditions. ICER defines the term as a disease impacting fewer than 10,000 people. Small populations make generating comprehensive clinical trials data difficult, ICER concedes. That requires the organization to tweak how it approaches these therapies.
CFRI's Many Voices ~ One Voice Cystic Fibrosis Advocacy and Awareness Campaign raises the general public's awareness and understanding of the challenges of cystic fibrosis - the most common fatal genetic disease in the United States. This film highlights individuals with CF as they balance daily medical regimens and living life fully.
Former Bengals quarterback Boomer Esiason is co-chairman of the Boomer Esiason Foundation.
Just over 24 years ago, my son Gunnar was diagnosed with cystic fibrosis, a debilitating and unrelenting disease for which there is no cure. At the time of his diagnosis, CF was akin to a death sentence – there were no FDA-approved treatments and we could only hope that we might see a cure in time to save Gunnar’s life.
Learn more at Cincinnati.com
White paper addresses why patients cannot access breakthrough treatments that treat the cause, not just the symptoms, of cystic fibrosis
WASHINGTON – Today the Cystic Fibrosis Engagement Network, a project of the Alliance for Patient Access, released a new white paper, “Making Treatment Accessible for Cystic Fibrosis Patients.” The white paper details what’s standing in the way of patient access to new treatments approved to target some of the genetic mutations that cause Cystic Fibrosis – and how addressing bureaucratic barriers could help the 30,000 Americans living with this rare disease. For the patients with specific mutations, these treatments can significantly improve lung function and quality of life.
The white paper pinpoints two key barriers to treatment:
1. Medicaid barriers. Due to the expense of managing the disease, many patients with cystic fibrosis – about 45 percent – depend upon Medicaid. Criteria and processes for determining prescription drug coverage can vary by state, where Drug Utilization Reviews and Pharmacy and Therapeutics committees often decide which patients get access to certain drugs. Officials do not necessarily communicate with actual patients or confer with experts, leading to decisions that can be “outdated and inconsistent with current practices.”
2. Prior authorization requirements. This technique requires patients and their families to appeal to the health plan for permission to access the medication their physician prescribed. Insurers may require paperwork, tests, or proof that a patient has failed on a less expensive therapy before they approve the requested drug.
The delay can be painful, even damaging for patients with the progressive disease.
These barriers can block vital access to advanced and long-awaited treatments. For decades, patients were able only to treat the symptoms of the disease using mucus-thinning drugs and vests that vibrate the chest cavity to help clear mucus buildup from the lungs and vital organs.
The paper urges policymakers, advocates, patients and health care providers to work toward commonsense solutions that improve access to cystic fibrosis medications.
Cystic Fibrosis Engagement Network https://www.engagecf.org
Alliance for Patient Access http://allianceforpatientaccess.org
Promising clinical trials data has excited patients with cystic fibrosis and their families. But will access barriers dash their hopes for more and better treatment options?
Read more at Institute for Patient Access.
Rare disease patients sometimes discover that, even when research does yield breakthrough treatments, access barriers stand in their way. Just take cystic fibrosis, a disease that causes mucus to cover the lungs, making it difficult to breathe.
As Congress continues to debate the future of the Affordable Care Act, a new advocacy group has a message for key legislators: protect cystic fibrosis patients as you reshape health care policy.
CFEN urges Senators to consider the needs of cystic fibrosis patients while debating health care reform.
CFEN urges members of Congress to consider the needs of cystic fibrosis patients while debating health care reform.
Some proposed policy reforms would harm a group that desperately needs access to affordable treatment.
More than a year after a breakthrough drug for cystic fibrosis became available, fewer than half of patients who meet the Food and Drug Administration’s criteria can actually access the medication.
Patients with rare diseases still struggle to access the medicine they need, despite growing disease awareness and passage of the Affordable Care Act.