GeneFo Releases Updated 2018 Guide to Help Cystic Fibrosis Patients Navigate Treatment Options

GeneFo has put together an updated 2018 guide on treatment options for people with cystic fibrosis (CF) after recent developments again focused attention on the high price tag of some of these medications and the difficulties patients face in accessing them.

The guide includes information on financial and clinical resources to help patients afford cystic fibrosis medications, such as assistance funds, clinical programs, and free insurance counseling/tax reduction programs, as well as access programs that provide free treatments in return for patient feedback.

Patients and members of the medical community have voiced their frustration at the high costs of cystic fibrosis treatments, especially after a U.K. body issued draft guidance recommending against therapy Orkambi (ivacaftor/lumacaftor) in 2016, saying it was not cost-effective.

As an online community of resources for CF patients, GeneFo has seen the increased chatter on its own platform. Aiming to provide real solutions, GeneFo created the guide to keep patients and caregivers updated on the various opportunities available for help with funding CF medication, treatments, and gear.

“We noticed that posting exciting research news about new drug developments and releases often served as a backdrop to patients voicing their concern and frustration about not being able to afford the drug,” Adi Bein, GeneFo’s vice president of strategy, said in a statement provided to Cystic Fibrosis News Today.

“Advocacy and policy are of course the central paths of action when it comes to improving access to healthcare, but at those levels change takes time, and in the meanwhile patients look for pragmatic solutions. That is why we created an up-to-date guide which covers various ways in which patients can get free, reduced-cost or reimbursed access to medication today. And surprisingly, many patients are not aware that those options are open to them,” she said.

So far, 2018 has seen some big news in the cystic fibrosis community, including the U.S. Food and Drug Administration’s approval of Vertex’s Symdeko (tezacaftor/ivacaftor and ivacaftor).

Symdeko is a disease-modifying therapy designed to treat cystic fibrosis in patients with the F508del mutation in the CFTR gene (the defective gene in CF). However, according to GeneFo, like other CF treatments, the price tag of Symdeko reaches hundreds of thousands of dollars, putting this treatment out of reach for many people.

Besides Symdeko, Vertex’s Orkambi and Kalydeco (ivacaftor) are also included in the guide.

Symdeko’s approval reignited the ongoing debate over the price of disease-modifying treatments and the difficulties in accessing these therapies for people who don’t have suitable insurance policies.

Discussion over the high costs of cystic fibrosis medications was sparked in March 2016, when the U.K. National Institute for Health and Care Excellence concluded that the cost of Orkambi was considerably higher than the current standard of care, and issued a draft guidance recommending against Orkambi in the treatment of CF.

“We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it,” Carole Longson, director of the NICE Centre for Health Technology Evaluation, said in a press release at the time.