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Anaerobic bacteria cultured from CF airways correlate to milder disease-a multisite study

Anaerobic and aerobic bacteria were quantitated in respiratory samples across three cystic fibrosis (CF) centres using extended culture methods. Subjects, ages 1–69 years, who were clinically stable provided sputum (n=200) or bronchoalveolar lavage (n=55). Eighteen anaerobic and 39 aerobic genera were cultured from 59% and 95% of samples, respectively; 16/57 genera had a ≥5% prevalence across centres. Analyses of microbial communities using co-occurrence networks in sputum samples showed groupings of oral, including anaerobic, bacteria whereas typical CF pathogens formed distinct entities. Pseudomonas was associated with worse nutrition and F508del genotype, whereas anaerobe prevalence was positively associated with pancreatic sufficiency, better nutrition and better lung function. A higher ratio of total anaerobe/total aerobe colony forming units was associated with pancreatic sufficiency and better nutrition. Subjects grouped by factor analysis who had relative dominance of anaerobes over aerobes had milder disease compared to a Pseudomonas-dominated group with similar proportions of subjects being homozygous for F508del. In summary, anaerobic bacteria occurred at an early age. In sputum producing subjects anaerobic bacteria were associated with milder disease suggesting that targeted eradication of anaerobes may not be warranted in sputum producing CF subjects.

Full article here.

The Hospital Comfort Kit Is Now Available!

The Hospital Comfort Kit Is Now Available!

When Rebecca Poole was admitted to the hospital in December 2014, she had no idea that she would not be discharged for 219 days. Her husband Ray focused daily on what he could do to make her more comfortable. Friends and family would ask what they could do to help and at the time he didn’t have an Continue reading The Hospital Comfort Kit Is Now Available!

£11 million-a-year US pharmaceutical boss is accused of trying to ‘blackmail’ Theresa May into buying cystic fibrosis drug

By Stephen Adams and Glen Owen

The boss of a US pharmaceutical giant was last night accused of trying to ‘blackmail’ Theresa May into buying a groundbreaking drug at an eye-watering annual cost of £100,000 per patient.

Thousands of people with the deadly lung condition cystic fibrosis (CF) have been pleading with NHS bosses to strike a deal with Vertex over Orkambi, a new treatment that could extend their lives by years.

The talks collapsed acrimoniously ten days ago, after which Vertex boss Dr. Jeff Leiden wrote to the Prime Minister threatening to scale back the firm’s investment in the UK unless NHS England agreed to buy Orkambi on its terms.

In an extraordinary outburst, Dr. Leiden accused Britain of placing ‘a lower value on the life of a CF patient than other countries’ which have purchased Orkambi, and warned that British jobs relied on his benevolence.
The row comes amid growing anger among health chiefs about how the ‘profiteering’ of drugs companies is heaping pressure on the NHS budget.

The starting price for Orkambi negotiations is thought to be £104,000 per patient per year.

Tory MP Bob Stewart said: ‘Drug companies have every right to turn a profit to fund their vital research and development work, but it is completely immoral to overcharge for a life-saving drug. If the CEO is trying to blackmail the Prime Minister that is utterly disgraceful.’

In his letter to Mrs. May, Dr. Leiden, who received £11 million in salary and stock options last year, wrote: ‘Vertex is heavily committed to the UK with its international HQ in London, an R&D site near Oxford and runs many clinical trials in the NHS. Since 2006, Vertex has invested over £1.2 billion in the UK and now employs 250 people, of whom over 110 are scientists.’

But he added: ‘We are questioning this ecosystem as [the UK] is unable to value life-changing medicines for the patients that need them.’ Without major changes to how new drugs were assessed, he pointedly warned that ‘any future biotech investment in the UK is at significant risk’.

In 2014, Dr. Leiden, who owns a string of waterfront homes in Massachusetts and Florida, faced a shareholder revolt over his ‘exorbitant’ pay package of almost £35 million. Orkambi is the only current therapy that slows the inevitable progression of CF. It could benefit up to half of the 10,500 Britons with the condition.

CF causes lungs to produce too much mucus, reducing their effectiveness. Around half of sufferers die before the age of 47, but Orkambi has been shown to boost lung function by 42 percent.

Catherine Upstone, from Brackley, Northamptonshire, whose daughter Cerys, 14, is one of those who could benefit from Orkambi, said: ‘The two sides need to get back around the table.’

But NHS England boss Simon Stevens recently told MPs that Vertex must first drop its price.

‘Right now, the company is a very long way from acting responsibly,’ he said. ‘The implication of the kinds of prices that are being talked about would either be to deny patients those drugs or to rip off British taxpayers, and neither of those situations is acceptable.’

Vertex’s chief commercial officer Stuart Arbuckle said the firm had made NHS England ‘the best offer in the world, which provides all eligible patients immediate access to our current and future medicines. However, the NHS wants our existing medicines and those coming down the line in the next year for no extra money – this is essentially asking for our medicines for free.’

NHS England said: ‘If Vertex believes they are offering a reasonable deal, they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair.’

Original article here.

Positive Data from the CARE CF 1 Clinical Study of Oral Lynovex in Cystic Fibrosis Exacerbations

NovaBiotics Ltd (“NovaBiotics”) announces that its oral therapy for cystic fibrosis (CF), Lynovex®, has met the study objectives of the CARE CF 1 clinical trial.

CARE CF 1 assessed the effects of two weeks of Lynovex treatment as an adjunct to standard of care therapy (SOCT) in CF, compared to placebo plus SOCT. This trial was designed to determine whether the inclusion of Lynovex capsules alongside SOCT lessened the clinical impact of exacerbations in adults with CF, as measured by symptom severity and levels of bacteria and inflammatory mediators in sputum and blood.  CARE CF 1 was a 6-arm study with the primary objectives of determining the optimal dose and regimen of Lynovex in patients with exacerbations of CF-associated lung disease and to further evaluate the safety and tolerability of Lynovex in exacerbating CF patients.  Continue reading Positive Data from the CARE CF 1 Clinical Study of Oral Lynovex in Cystic Fibrosis Exacerbations

Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

By Jennifer Prince

A marine omega-3 compound comprising a docosahexaenoic acid (DHA) sn1-monoacylglyceride (MAG-DHA) may act as an anti-inflammatory for subjects with cystic fibrosis, according to a new pilot study1 published in the journal Marine Drugs. In the study, MaxSimil (Neptune Wellness Solutions; Laval, QC, Canada) increased omega-3 red blood cell levels, helped moderate the ratio of arachidonic acid (AA) to docosahexaenoic acid, and reduced key inflammatory biomarkers in subjects with cystic fibrosis. Continue reading Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

Vertex Pharmaceuticals opens expanded San Diego research center with focus on cystic fibrosis

By Bradley J. Fikes

Vertex Pharmaceuticals opened its new San Diego research center Monday, starting a new chapter in a decades-long quest to not only treat but cure cystic fibrosis.

In 18 years, three drugs for the lung-ravaging disease have emerged from Vertex’s San Diego center and more are in the pipeline.

The first, Kalydeco, was approved in 2012. It is the first drug that treats the underlying cause of the disease. The second, Orkambi, was approved three years later. And the third, Symdeko, was approved in February.

These drugs can benefit about half of all patients with the incurable disease. In the next several years, Boston-based Vertex hopes its drugs can help nearly all patients live longer, healthier lives.

Cystic fibrosis is caused by a genetic defect that allows a buildup of thick mucus in the lungs, and other internal organs. This mucus clogs airways and promotes the growth of bacteria. The average lifespan of patients is 37 years, up from 20 years in 1980. Treatments include antibiotics to fight lung infections and mucus-thinning drugs.

The new 170,000 square-foot building on Torrey Pines Mesa more than doubles the company’s space. The center includes cell culturing equipment to grow lung cells from patients, to be used for drug screening. A 4,000 square-foot incubator suite will serve outside collaborators.

Asides from cystic fibrosis, the staff will work on other serious diseases.

Among the speakers Monday morning was a veteran in the fight against cystic fibrosis: Jennifer Ferguson, who has two children with the disease, Ashton and Lola. Both her children are taking Vertex drugs, and both were present with her at the event.

With these drugs and the promise of better therapies ahead, she says Ashton and Lola have a good chance of growing up and leading their own lives. She urged all Vertex employees to think of themselves as part of a team to cure the disease.

Ferguson, of San Diego, found out about the work from the Cystic Fibrosis Foundation. The foundation had invested $30 million in startup Aurora Biosciences to find therapies.

In 2001, Vertex purchased Aurora for $592 million in stock, the same year Ashton was diagnosed. The research went on under Vertex, and Ferguson became quite familiar with the research team.

“The Cystic Fibrosis Foundation asked me to come speak, to show them what it’s like to have a little child with CF,” she said. “So I came here about 17 years ago with him as a 6-month-old.”

At that time, many cystic fibrosis patients never reached adulthood.

“I had a hard time keeping it together,” Ferguson told the audience of that long-ago visit.

“But I looked in the staff’s faces — and some of you are still here — and I thought, I’m going to put my faith and trust in your hands, in your brains. And I was able to let go of my worry, because you were on the case.”

Ferguson started visiting every few years to check on what progress was being made, first with Ashton, and later including Lola. She also raises money for the Cystic Fibrosis Foundation.

Both her children have shown improvement since starting the Vertex drugs, Ferguson said. But they still need to go through a daily regimen of clearing out their lungs.

From medications, the research frontier has advanced to investigations into a cure. That means fixing the genetic defect, which can come in several variations, inside living patients.

That cure might come from the hot new gene editing technology called CRISR. In 2015, Vertex allied with startup CRISPR Therapeutics to develop curative therapies.

This post was originally published on The San Diego Union-Tribune

7 Things Your Partner with CF Probably Isn’t Telling You

By Hannah Buck

Being in a committed, loving, long-term relationship is a distinctly intimate experience. It is the most intimate experience of humanhood many would argue. To see a person walking by and say to them, “Hey, you’re fairly OK-looking. Would you like to hold hands for forever and accrue debt until we die?” is to truly know companionship. That, and watching each other poop.

Chronic illness makes dating a thoroughly more vulnerable experience, and not just for the patient. You see, sickness affects everyone involved. It accelerates everything. Sickness makes daily life complicated (e.g. planning dates that aren’t physically taxing or one partner relying more on the other for help with everyday chores) and the future even less promising than it usually is. When you or your partner has a condition like cystic fibrosis, the bleak reality of your situation is sorely evident. It’s inescapable. One of you will die much sooner than the other. And with that intense reality flashing its lights 24/7, it can be tempting to hold things in.

I don’t speak for all people with CF in writing the following list — but by sharing what I’ve withheld in past romantic relationships, I hope to make you laugh, open your eyes, and help you become a better partner to the person whose hand you like to hold. Enjoy.

Things your partner with CF probably isn’t telling you

1. They’ve been wetting your bed for a while.

Have you ever rolled over in the middle of the night to feel a damp spot on the mattress? Has your girlfriend been known to spontaneously wash your sheets and comforter out of the goodness of her heart? Yeah, sorry to break it to you, it’s not because she’s an angel. It’s because she has coughing-induced, premature incontinence, and she doesn’t want you to know.

2. Their antibiotics give them diarrhea.

It just happens, OK! We don’t ask for this! Antibiotics have one mission: to kill. This includes good gut bacteria, which unfortunately messes up our tummies. Make your partner’s day by surprising them with a bottle of probiotic-rich kombucha to get things back on track.

3. They’d prefer if you looked away during their cough attacks.

Coughing ain’t cute. Yes, yes, I know you love them, but try to put yourself in your partner’s shoes. If you were red in the face, foaming at the mouth, hunched over like the Notre Dame character, and spewing phlegm like a swampy sprinkler, would you want the love of your life to gaze longingly upon you? Probably not. Give ’em space.

4. They wish you visited them in the hospital more.

They just feel too guilty to say it out loud. It is unspeakably lonely to sit in a small room and face the same wall every day. Please, even if they insist they’re fine, be there for them. Stop by. Make the time. If you can’t do that, text, call, or video chat. While 24 hours pass by in a snap in the outside world, in the hospital, the hours drag like you wouldn’t believe.

5. It makes them really happy when you randomly flex your CF knowledge.

Showing your partner that you care about them enough to not only learn about their disease but about how it’s treated is an instant way to grow closer. For many of us, the only people we have to confide in about this part of our lives is our medical team, our family, and occasionally other CFers (but only online). Take the time to learn what’s what — get the medication names right, make yourself an ally, and demonstrate that the two of you are teammates in this fight.

6. Explaining what’s “wrong” with them to other people makes their life so much easier.

With this one, I want to repeat my disclaimer: I am speaking for myself, and every person with CF is different. Please talk to your partner before taking this advice.

With that said, I have always found it to be an incredible relief when my partner discreetly says, “She has something called CF, so she coughs a lot. It’s normal. So anyway … ” and then changes the subject when I have a cough attack around people who don’t know me. Explaining myself is something I’ve had to do my entire life, so having someone else do it for me is a treat I savor every time.

7. They don’t feel worthy of your love, and they feel guilty about loving you.

I hope this one isn’t true for you guys. In my case, it is, and I suspect it’ll be a lifelong battle. Having an incurable illness is a heavy burden to bear, but when you’re born with it, there’s no other option. Putting it on another person, though, that’s different. That isoptional. And it can feel impossible to justify exposing the person whose hand you like holding to that level of lifelong pain.

This post originally appeared on CF News Today.

Broadway’s biggest stars come together to raise money for Cystic Fibrosis

What do you get when Broadway’s biggest stars, such as Javier Muñoz and Gideon Glick, come together in the recording studio? One heck of a song. And one heck of a message.

Joined by Broadway veterans Laura Osnes, Christy Altomare and actress Sarah Levy, Muñoz and Glick have banded together for a new campaign — called the “Anyway” campaign — for an original song to help raise money for Emily’s Entourage.

At the center of the Entourage is Emily Kramer-Golinkoff: a 33-year-old daughter, sister and granddaughter who was diagnosed with Cystic Fibrosis when she was just a few weeks old. Her parents, Liza and Michael, have done everything in their power to raise Emily as if she was a normal child.

As the fatal disease only affects 70,000 people worldwide, funding for a cure is limited. Emily and her family are even more restrained by her specific mutation, which means medical funding is even rarer. And with a life expectancy of only 35-37 years for her kind of CF, time’s ticking.

Six years after the Kramer-Golinkoffs decided to take matters into their own hands, they’ve raised $3 million to drive high-impact research and speed up breakthroughs to research not only Emily’s mutation, but many other diseases including muscular dystrophy, inherited blood disorders and certain cancers.

They’ve also built a network of family, friends, and strangers from around the world, all of whom have been welcomed into the Entourage. Each Entourage member has been more inspired by Emily’s story than the next.

Take Elizabeth Phillipson-Weiner, from Emily’s hometown of Philadelphia, Pennsylvania and composer of “Anyway.” She took a simple journal entry from a songwriting retreat, turning lyrics like “when the going gets tough I ask questions” into a prolific melody.

Elizabeth explained to AOL Lifestyle. “The song wasn’t just cathartic for me, but could actually ring true for a lot of other people. I immediately thought of Emily.” Working with co-writers, producers and engineers, that melody was soon turned into a demo.

But as inspired as Elizabeth was by Emily’s story, the Entourage required “star power” to bring this project alive.

“I cold contacted agents and managers, I asked friends and friends of friends and friends of friends of friends,” said Elizabeth. “We did hear ‘no’ quite a bit, but whenever I became discouraged or frustrated I thought about who and what I was doing this for.”

This story was originally published on AOL.com

Surviving Home IVs As a Mom

By Janeil Whitworth

I’m no stranger to home IV antibiotics.

Actually, if I am being completely honest, I think we have gotten a little too friendly over the past decade or so. I prefer to do home IVs because I am one of those patients who goes a little nuts after being trapped in the hospital for too long. Seclusion and unlimited access to cable are not beneficial to my physical or mental well-being. I need the opportunity to be with people just as much as I need the comfort of my own bed to rest and heal completely. I’ve even gone as far as learning to access my own port-a-cath to create even further distance between the hospital and me. Plus, I enjoy the luxuries of home IVs such as creating my own schedule, eating my own food, using my own toilet paper, and coming and going as I please.

Releasing control

I’m positive I’m not the only one who struggles to release control over my disease while experiencing an exacerbation. Feeling the façade of freedom slipping away post-IV-talk, I involuntarily tighten my grip on my independence in a desperate attempt at normalcy.

In-patient admission? No way. Home IVs? Ok, I can do that. Thankfully, you can travel on home IVs, celebrate your bachelorette party on home IVs, and even graduate college on home IVs. But is it possible to successfully care for a 9-month-old baby on home IVs?

After the past few weeks of increased shortness of breath and unwavering fatigue, I agreed it was time for IVs. The reality of home IVs as a mom suddenly hit me as I exhaustedly said for the millionth time that day, “Please, don’t eat mommy’s IVs, buddy.”

I knew this was not going to be easy with a mobile, energetic baby crawling every which way, leaving a trail of alcohol pads in his path. (I think this might be the fatigue talking, but the crunchy hospital bed and one-ply toilet paper are starting to seem a little better right now.)

The last time I did home IVs, my son was 4 months old, so it made it a tad easier as he was just a chubby and adorable blob. I would set him down, hook myself up to the antibiotic-filled Eclipse ball, and he wouldn’t move. Those were the days.

There’s been very little rest this week, and admittedly, I tried my hardest to do it all even if I was feeling absolutely poisoned. In my defense, I felt my independence as a mom was slipping away once again, and the impulse to hold on tighter completely crept up on me. This way of thinking will not benefit me in the long run, neither as a mother, nor as a patient. A week has passed since I accessed my port to begin treatment, and I can see more clearly now that I am in desperate need of a different rhythm.

Finding a different rhythm

I am going to attempt to take my own advice for the remainder of treatment, including: 1) Ask for help; 2) Be easy on myself when it comes to everything else; 3) Remain grateful for the privilege of staying home. My normalcy in the coming weeks will look a little different. My parenting style will be altered as well. I need to accept that this is the price for staying home with my baby, while simultaneously being the nurse and patient. Everything needs to be taken in stride as I release my grasp and let the rest, antibiotics, and baby hugs heal me. I think this is the key to surviving home IVs with my health, sanity, and pride intact. I hope.

This blog was originally published on CF News Today.

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.

From The New York Times:

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.

New York’s Medicaid program says Orkambi, a new drug to treat cystic fibrosis, is not worth the price. The case is being closely watched around the country.

A wave of breakthrough drugs is transforming the medical world, offering hope for people with deadly diseases despite their dizzying price tags.

But what if it turns out that some of these expensive new drugs don’t work that well?

That’s the quandary over Orkambi, a drug that was approved in 2015 for cystic fibrosis and was only the second ever to address the underlying cause of the genetic disease. Orkambi, which is sold by Vertex Pharmaceuticals, costs $272,000 a year, but has been shown to only modestly help patients.

Now, in a case that is being closely watched around the country, New York state health officials have said Orkambi is not worth its price, and are demanding that Vertex give a steeper discount to the state’s Medicaid program. The case is the first test of a new law aimed at reining in skyrocketing drug costs in New York’s Medicaid program.

The high price of prescription drugs has ignited a populist furor, and in May, the Trump administration unveiled a set of proposals to address the issue. But while the ideas at the federal level are still mostly theoretical, some states have begun tackling the issue themselves. Earlier this year, Massachusetts asked the federal government for permission to limit its coverage of drugs in an effort to secure larger discounts from drug makers. Other states, like California and Vermont, have passed laws requiring drug companies to turn over certain financial details if they raise prices significantly.

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“There’s a number of states that are really trying to push forward and say, we need to be thinking very differently about how we’re paying for drugs,” said Matt Salo, the executive director of the National Association of Medicaid Directors. “We need the ability to say that there are some drugs that are just not priced in a rational way.”

Orkambi held great promise for people with cystic fibrosis when it was approved three years ago. A similar drug, Kalydeco, approved in 2012, was viewed as groundbreaking because it was the first to try to counteract the genetic defect that causes cystic fibrosis. The disease, which affects about 30,000 Americans, leads to a buildup of sticky mucus in the lungs and can lead to death by respiratory failure by the time many people are 40.

But while Kalydeco, also known as ivacaftor, was found to be effective, it was only approved for a sliver of patients with the disease — those who had certain genetic mutations. Orkambi, which combines ivacaftor and another drug, lumacaftor, was approved for mutations that covered nearly half of cystic fibrosis patients, but studies showed it was not as effective as Kalydeco.

Since Orkambi’s approval, several countries have balked at paying for it, including Britain, France and Canada.

In the United States, private insurers and Medicare plans have generally covered Orkambi. Medicaid programs, which cover health insurance for the poor, are required to cover all drugs.

Still, many insurers require patients to pay thousands of dollars out of pocket, and even though Vertex offers assistance, not everyone qualifies.

Lora Moser, 40, is covered by Medicare because she is disabled, and said she had to stop taking Orkambi in January because she could not afford the first month’s payment of more than $3,000 required by her insurer, Humana. A spokeswoman for Humana said that for high-cost drugs like Orkambi, the insurer helps patients identify outside assistance programs to cover out-of-pocket costs.

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Ms. Moser said a nonprofit group that had provided financial assistance declined to renew her grant because, she said she was told, her annual household income was too high.CreditTamir Kalifa for The New York Times

A nonprofit group that had provided assistance the previous year to Ms. Moser declined to renew her grant because, she said she was told, her annual household income was too high. She said her income is about $600 above their limit.

“I’ve never felt more destitute and hopeless as I do right now, from a medical standpoint,” Ms. Moser said.

A spokeswoman for Vertex, Heather Nichols, said more than 99 percent of cystic fibrosis patients who are eligible to take Orkambi in the United States have “broad access” to the drug.

“Vertex has a longstanding commitment to supporting access for all eligible patients, and we will continue to oppose any attempts to restrict patient access to these transformative medicines,” Ms. Nichols said.

Despite its lukewarm reception, Orkambi has been a boon for Vertex. In 2017, the drug was its top-selling product, bringing in about $1.3 billion in sales, a considerable sum for a product that is only approved to treat about 28,000 people worldwide.

Dr. Steven D. Pearson, the president of the Institute for Clinical and Economic Review, which evaluates the cost-effectiveness of drugs, said the problem is that in the United States, drug companies control the prices, especially in the case of newly approved drugs like Orkambi.

“Our system is set up not to distinguish very well between those drugs that are fairly priced and those that are not,” he said. Dr. Pearson’s institute concluded that Vertex’s cystic-fibrosis drugs should be discounted by as much as 77 percent. “That gives the incentive to the company to overreach, and that’s part of why our system is so out of whack,” he said.

In April, Orkambi became the test case for the New York law when a state board ruled that the drug was not worth its cost, recommending that it be discounted from the list price by roughly 70 percent — an amount that was influenced by work done by Dr. Pearson’s institute. New York’s law, passed in 2017, allows the state to ask manufacturers for a deeper discount if the state’s Medicaid drug budget exceeds a certain amount.

Under federal law, state Medicaid programs get a rebate of at least 23 percent. New York officials said that they identified 30 drugs this year that were priced too high, and that those products’ manufacturers agreed to deeper discounts, resulting in about $60 million in annual savings. Vertex, which is based in Boston, was the only company that refused, the state said. New York officials did not identify the manufacturers that agreed to steeper discounts.

For now, at least, Vertex appears to have the upper hand because federal law requires the state to cover Orkambi, although the state can limit its use. Under its new law, New York could also demand that Vertex disclose details about how it sets its price, including how much goes toward research and development or to other areas, like marketing. But even if Vertex complied, that information would not be made public because it is considered proprietary.

Ms. Nichols, the Vertex spokeswoman, said the company had no plans to agree to a discount below the 23 percent required by law.

And Donna Frescatore, the director of New York’s Medicaid program, said she was reluctant to limit the use of Orkambi for those who need it. “It’s certainly a balance with our ability to get fair pricing for this medication,” she said.

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Ms. Moser prepares to administer medication through a nebulizer at her home in Leander, Tex. CreditTamir Kalifa for The New York Times

But despite the impasse, Mr. Salo said big states like New York are major buyers of prescription drugs, and companies may see an interest in taking those states seriously. “I see this as being of very, very widespread interest,” he said. “A lot of other states are kind of watching and saying, ‘How is that going to work?’”

The debate over Orkambi may soon become moot — earlier this year, the Food and Drug Administration approved a new cystic fibrosis drug, also made by Vertex. Symdeko, as the drug is called, treats a similar population as Orkambi, but has been proven to be more effective. It carries a list price of $292,000 a year, and some analysts, including Geoffrey Porges, of Leerink, say they believe Symdeko will eventually replace Orkambi.

Given the arrival of Symdeko, some analysts said New York would be smart to negotiate a package deal for all three of Vertex’s cystic fibrosis drugs, similar to a deal recently made with Ireland. Ms. Frescatore said that’s an approach that she would consider.

“You don’t want a patient being forced to take Orkambi because it’s cheaper,” Mr. Porges said. “You want the right patient to get the right medicine.”

Katie Thomas covers the business of health care, with a focus on the drug industry. She started at The Times in 2008 as a sports reporter. @katie_thomas