Life or Death for Young Lady with Cystic Fibrosis—Trikafta Not Approved in UK & Europe and Time’s Running Out

Life or Death for Young Lady with Cystic Fibrosis—Trikafta Not Approved in UK & Europe and Time Running Out

28 year-old Nicole Adams from Belfast is literally being suffocated by her own body due to her case of cystic fibrosis. A wonder drug called Trikafta could potentially save her. The problem: although approved in America, it hasn’t been approved in Europe or the UK; and the UK’s National Health System ‘very busy’ financial watchdog NICE doesn’t seem in a rush to do something until 2021. Perhaps U.S.-based Trikafta drug maker Vertex can spring into action and release the drug for compassionate use? A month supply is a lot—20,000 pounds or about $26,000. A beautiful young lady is in serious trouble.

Cystic Fibrosis

Affecting at least 10,500 in the UK and about 100,000 around the world, there is no cure for the disease. Just in October, the FDA approved Trikafta for cystic fibrosis patients

A Beautiful & Strong Young Lady

The local press The Daily Mail recently covered the plight of this beautiful young lady. With a growing severe case of cystic fibrosis, her body is literally suffocating herself. As she has neared the end stage of the disease, her lung function operates at 13% of full capacity. She desperately struggles with each breath. 

Her Only Hope

The degradation in her health leads all involved to believe Nicole’s only hope is to start taking Trikafta. Yet this pioneering drug is not available in Europe or the UK as of yet, reports Barney Calman for The Daily Mail Sunday. Her and family have pleaded to the NHS in the UK as well as Vertex bosses for help. After all, there is a compassionate use clause in Europe for drugs that are not yet approved.

Apparently, the drug will be available in the Republic of Ireland (Nicole lives in North Ireland)—only a short train ride—but it is an entirely different health care system in a different country.  It wouldn’t be easy for her to just access this system.

What is the Hold Up: Drug Politics

Because the drug isn’t approved in Europe, the 20,000 pound per month drug isn’t available. The projection is that UK doctors will be able to start prescribing in 2021—but Nicole—and perhaps many others—very well may not make it. In the UK, a financial watchdog group, National Institute for Health and Care Excellence (NICE), must analyze the drug, its efficacy and impact, and then assess the value for pricing purposes. It reports to be too busy to deal with this situation only now.

Raising Money and Compassionate Use

Nicole’s boyfriend, Ciaran McVarnock, has been actively raising funds so that she would be able to buy at least one month supply from Vertex for 20,000 pounds—a lot of money for most people. He has nearly raised the total amount. But what Nicole and other desperate patients need is progress, so that it is available in the UK health system. The response and feedback has been overwhelmingly positive—patients from the UK and even America have offered to support, and patients in America have offered to send tablets of the drug. Of course, they cannot accept such a gift.

Recent Vertex and UK Government Deal

Recently, Vertex and the UK settled on pricing for older CF drugs Orkambi and Symkevi—said to be the largest financial deal of its kind in NHS history, amounting to about 100 million pounds over two years. These negotiations went on for years—so long, in fact, that now with Trikafta approved in the U.S., the other drugs are basically near obsolete according to some. Apparently Trikafta is 4x more effective than the older drugs; and the new breakthrough drug is 90% effective. But either way, they have haggle back and forth on cost of the older drugs. The discussions got so complex with what one side was willing to pay versus what the other side was willing to sell the drug for that Trikafta was not included.

Unfortunately, in signing this latest deal, they couldn’t include Trikafta. For it to be approved in Europe, it must get reviewed and approved by the Europeans Medicine Agency (EMA) and then have its value assessed by the National Institute for Health and Care Excellence (NICE). This assessment is based on a complex calculation based on the drug’s cost versus its efficacy. 

NHS Doesn’t Buy into Non-Approved Drugs

Apparently, at least in this situation, the NHS leadership is skeptical to put the commitment and money into a drug not yet approved in Europe.

Compassionate Use

The last hope here is that Nicole can secure sufficient supply under the “compassionate use” clause in Europe and the UK. A spokesperson for Vertex noted, “We cannot comment on specific patients, but take all requests for use of our medicines very seriously. We are working diligently to access these requests as quickly as possible once they are received from a patient’s doctor, which is the sole means of initiating the process.”

It all comes down to her physician and Vertex now. Once her doctor initiates the request (and we are assuming he did), then we sincerely hope Vertex does the right thing in closing the loop and connecting with Nicole to ensure she has the supply she needs.