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Local mother, daughter ‘getting loud’ for CF and the need for lifesaving medication – BradfordToday

Posted: May 30, 2020 at 9:48 pm

Just a few short months ago, the Cystic Fibrosis (CF) community was making strides in their fight to have life-saving 'orphan' drugs approved in Canada.

Then COVID-19 hit, and their efforts were put aside as the government grappled with the pandemic.

Local resident, Beth Vanstone, mother of CF Warrior Madi Vanstone, spoke with BradfordToday earlier this week to discuss the challenges the CF community is now facing and the tremendous effort being made to have the government to approve orphan drugsin Canada, not only for CF patients, but for all Canadians with rare conditions and diseases.

What are orphan drugs? And why are they critical to so many Canadians?

Orphan drugs treat rare diseases. They tend to be hugelyexpensive,due to the lengthy research involved in development, and the small percentage of people needing them. There is no chance of mass-production or mass-consumption, to bring down the price.

Eighteen-year-old Madi is currently on the medication Kalydeco, another orphan drug,to treat her CF.

Madi was diagnosed with CF at nine months of age, and struggled throughout childhood with multiple hospitalizations due to her illness, often missingschool.

Six years ago, when Kalydeco was released, Madi was able to take the drug as part of a drug trial - and the Vanstones launched their battle with theOntario government, to have themedication Kalydeco covered by OHIP. Without coverage, the cost to the Vanstones - even with private insurance and a discount from the pharmaceutical company - would have come to approximately $60,000 per year.

The government did approve coverage of the medication, which she is still on today. Kalydeco has substantially improved the quality of her life.

But their fight isnt over. Madi and her mom have been working tirelessly to help other CF patients and other Canadians with rare diseases gain access to the orphan drugs they require.

There are currently three new CF modulators (drugs) from the pharmaceutical company Vertex -Orkambi, Symdeco and Trikafta -with very little access to Canadians.

Since the COVID-19 pandemic, CF Canada has reached out to the government and Vertex Pharmaceuticals to allow CF patients access to all current and future CF modulators.

In a letter to the federal government,CEO of CF Canada Kelly Glover noted that the pharmaceutical company Vertex had recently made an offer to the pan-Canadian Pharmaceutical Alliance (pCPA), and urged the government to negotiate on the offer right away.

Cystic fibrosis is a fatal disease," Glover wrote. "Similar to COVID-19 it affects primarily the respiratory system. Similar to COVID-19, it kills people. Unlike COVID-19 its much more efficient, just slower. Eighty five percent of people with CF will die from respiratory failure, but CF will ultimately kill them all, as compared to only about 1-2% for COVID. But instead of dying in weeks, CF slowly deprives everyone with CF of quality of life. Our 4,300 patients spent a total 26,500 days in hospital in 2018. Thats 72 years! Seventy-two years worth of hospital resources that could be better spent taking care of people with COVID-19.

But the pCPA has indicated that it is not negotiating a deal for two of the three CF modulators, Orkambi and Symdeco, but isonly considering negotiating for Trikafta, once it is approved by Health Canada. Currently Trikafta can only be made available in compassionate cases if the patient meets specific criteria.

Our community is so vulnerable, having underlying respiratory issues already, said Beth, worried about the additional impact of COVID-19.

Everyday our people have to be six feet a part, everyday if theres somebody sick we cant go to them. Our kids cant touch things because they can get sick and end up in the hospital, she said.This is our life. Everyones getting a little test of what its like to have CF.

A response from the pCPA Governing Council stated, The pan-Canadian Pharmaceutical Alliance (pCPA) recognizes that this is a challenging time, especially for cystic fibrosis patients during the COVID-19 pandemic.

The Council noted that they have held numerous meetings with Vertex, hoping to reach an agreement to improve access to drug treatments for cystic fibrosis patients. The alliance continues to encourage Vertex to submit Trikafta to Health Canada and if approved, to then submit it to Canadian Agency for Drugs and Technology in Health (CADTH) or lInstitut national dexcellence en sant et en services sociaux (INESSS) for a Health Technology Assessment (HTA) review, the response stated.

The cPCA declined to providedetails of the discussionswith Vertex due to confidentiality reasons, but noted, the pCPA continues to engage with Vertex with the goal of bringing new cystic fibrosis medications to the Canadian market."

Beth and the rest of the CF community point to a disparity: governments are providing unlimited funding forCOVID-19 patient care, but there is alack of funding of treatment for CF patients who suffer breathing issues everyday.

We will spend whatever, and shut down our country, to save people from COVID, said Beth, yet it's too expensive to pay for the medications for CF patientsthat could ease the suffering, and reduce hospitalizations.

You cant help but take it a little personal, she said. Its really frustrating that the government doesnt seem to care.

She also worries that,with the new rules for the Patented Medicine Prices Review Board (PMPRB) set to come into effect this July, it will be even more difficult for companies like Vertex to market in Canada, due to the high price of orphan drugs.

The new rules and regulations for lowering the cost of medications for patients across Canada are supposed to take effect in July 2020. The PMPRB has said itwill be publishing an updated version of its draft pricing Guidelines for public consultation in the coming weeks.

This document is responsive to stakeholder feedback, including concerns from patient advocacy groups about the impact of the pending changes on access to promising new drug therapies, said Communications Advisor for the PMPRB, Jeff Wright.

Beth Vanstone is currently working with the group CF Get Loud, which has joined forces with CORD (Canadian Organization for Rare Disease) to oppose the PMPRB changes.

They (CORD) represent the rare disease community, and of course innovative therapies are so expensive for them as well, so they are going to be cut off from medications as well, she explained.

Simcoe-Grey MP Terry Dowdall has been working with the Vanstones and the CF community, to gettheir voices heard. He recently raised his concerns in the House of Commons, in an effort to have Patty Hajdu rescind the changes to the PMPRB.

"You are seeing how money can become available for certain issues," he explained, referring to the government spending on COVID-19, "Health is number one, we really need to take care of Canadians here in our homeland."

He belives the longterm costs of keeping CF patients in hospitals and on disability, far outweigh the costs of their needed medications.

"We're going to keep pushing it and Ihope one day its not an issue," he said. "I'm hoping the government does the right thing."

We are pushing that the changes be rescinded or an implementation date be changed to 2022 where they are apparently coming up with a rare disease strategy, Beth said.When they have a path set up for rare diseases in 2022, implement the changes at that time so orphan drugs can stream off on their own.

She is hoping that way, future drugs for rare diseases will not have to go through the same restrictive criteria.

What the PMPRB is doing right now is deciding for all Canadians what medications they will have access to, and its a very small percentage of whats available to everyone else in the world, Vanstone said. And instead of improving negotiations with pharmaceutical companies,theyre taking that opportunity right out, she said.

Wright, responding for PMPRB, stated thatIf any business decisions affecting Canada are being contemplated by pharmaceutical companies as a result of the draft Guidelines, it would be premature to take them until such time as the document is finalized.

He added that manufacturers don't need the PMPRBs prior approval of their price in order to launch drugs in Canada.

Beth is worried that, with all the emphasis on cost-savings,Canadians are not seeing the 'bigger picture' andthe implications of the PMPRB, especially for orphan drugs.

These drugs are available everywhere else. Noother country is doing something like this, she fumed. Its a war against big pharma, and patients are the casualties. Its heartbreaking."

The Vanstones and the rest of the CF community feel like they are on the wrong side in a losing battle.

I really have always thought people came first in Canada, until getting involved in this kind of scene and seeing that its not really true, Beth said.

As for the PMPRB, I think it was a really poorly thought-out plan, and now theyve dug their heels in, she said, noting that the plan may be a good idea for general medications for things like a cold, but not for those whoneed innovative life-saving medicines for rare diseases.

On June 3, the CF Get Loud community will be hosting a virtual Town Hall for patient advocacy, calling on Federal Health Minister of Canada Patty Hajdu and other MPs to rescind the changes in the PMPRB, so that the drugs can be submitted to help save patients lives.

Bethencourages anyoneinterested in learning more about the future of Canadas health care system in relation to new and innovative medications, to tune in.

Its not just going to be about CF," she said. "Its about our (health care) system and whats happening.

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Former Nats CF Denard Span mulling retirement: ‘Maybe this is it’ – NBCSports.com

Posted: at 9:48 pm

A former Nationals leadoff man and fan favorite has reportedly come to terms with the possibility of his baseball career reaching its conclusion.

Centerfielder Denard Span played in Washington from 2013-15, hitting .292 with 62 stolen bases and 207 runs scored as the teams primary hitter at the top of the lineup. Following stints with the San Francisco Giants, Tampa Bay Rays and Seattle Mariners over the next three seasons, Span has spent the last year and a half as a free agent.

I havent announced it, officially, but maybe this is it, Span told the Minnesota Star Tribune in a story published Saturday. I didnt play last yearnot because of an absence of contact from teams. The offers that came my way; they didnt seem like fair value for my services.

I spent this past offseason getting in shape, getting ready to play in 2020, and there were two or three minor league offers. I wasnt opposed to starting in the minors, but these didnt seem right as far as having a chance to move up to the big club if I was doing well in Triple-A.

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Span, a former first-round pick, started his career with the Minnesota Twins in 2008. He played five years in Minneapolis before traded to the Nationals just after the 2012 season. Washington was in desperate need of both an everyday centerfielder and a leadoff man, and Span checked off both boxes.

As a sparkplug that ignited the Nationals offense, Span quickly earned the appreciation of fans in D.C. His best season came in 2014, when he led the NLwith 184 hits and placed 19th in MVP voting. When the Nationals clinched a playoff berth last September, Span commented on the teams Instagram page to say his time in D.C. was the best 3 years of my career.

A day after he signed a free-agent contract with the Giants over the 2015-16 offseason, the Nationals moved on by acquiring Ben Revere from the Toronto Blue Jays in exchange for reliever Drew Storen. Revere kick-started another round of musical chairs at the center field position in D.C. until Victor Robles took over the job full-time last season.

Span admitted to the Star Tribune that while hed be open to returning to the majors, there likely wont be a situation that will entice him enough to do so.

I know that if the season ever would get started, he said, I still would have the ability to help a team. But 36-year-old outfielders who havent played in two yearsnot happening.

Im very satisfied pouring my life into our family, to [my wife] Anne, a wonderful person, and our two boys.

Stay connected to the Capitals and Wizards with the MyTeams app.Click hereto download for comprehensive coverage of your teams.

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Boy with cystic fibrosis bounces his way through lockdown – Kent Online

Posted: at 9:48 pm

A four-year-old boy who hasn't been able to leave the house since the pandemic hit due to his cystic fibrosis is bouncing his way through lockdown.

Theo Powell, from Tunbridge Wells, has been getting involved in a national challenge - Bounce 4 CF for the Cystic Fibrosis Trust, taking to his trampoline in a dragon costume and bounding around his garden in a yellow sack - the brand colour of the trust.

He is one of 10,500 people in the UK who have the condition which causes thick, sticky mucus to build up in the lungs, and is therefore in the vulnerable group during the coronavirus crisis.

Trampoline sessions are part of Theo's daily exercise and physiotherapy routine, as the movement helps to dislodge the mucus and improve his breathing.

So, encouraged by his parents, Caroline and Laurence, the Nivan Lodge Pre-School pupil decided to also use this as a way to raise money for a cause which the family has been involved in for some time.

Due to Theo's condition, the family have had to stay home since advice for the shielding group was issued by the Government., and do not know when they will be able to go out again.

Rev Powell, vicar at King Charles the Martyr Church priest in charge of St Mark's Church, has been holding services via Zoom and Mrs Powell has been trying to come up with inventive ways to keep Theo entertained during lockdown.

Mrs Powell, 31, said: "Every day gets quite similar but we are trying to mix it up when we can - camping in the garden, building stuff - we're keeping busy."

Although, she says Theo is used to not being allowed out due to frequent hospital trips.

"He's been in hospital quite a lot - he spent a month and a half there in total last year and when he's there, he's not allowed out of the room in case of cross contamination," said Mrs Powell.

Her husband added: "This strict shielding lockdown is a difficult situation for the whole family.

"Theo has just been accepted at the local school but at the moment we are not sure whether he will be able to start with his cohort."

The family are also pushing to get Theo a life-extending therapy called trikafta on the NHS.

Last year, hundreds shared their delight as Orkambi, said to slow decline in lung function, was made widely available after several years of campaigning. However, it does not treat all cystic fibrosis patients, with trikafta helping others whose DNA is not compatible with Orkambi, such as Theo.

In addition, Mrs Powell is working on a separate Instagram campaign, @wearyellow2020, encouraging people to post photos of them wearing yellow in support of Cystic Fibrosis Trust on Friday, June 19.

Along with her friend, Laura Fine, Mrs Powell is running a raffle on the day, with other games and fun activities planning for the week running up to 'wear yellow day'.

To support Theo in his Bounce 4 CF campaign, click here, and to get involved in the family's Instagram campaign, click here.

Read more: All the latest news from Tunbridge Wells

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How Five Feet Apart a story about Cystic Fibrosis is relevant today – stopthefud

Posted: May 14, 2020 at 6:07 pm

BY: HILARY PEARSON

One thing most of us have learnt during this pandemic: germs can spread as far as six feet when someone coughs or sneezes. They can land on surfaces, such as a doorknob, or in another persons eyes, nose, or mouth. Thats why social distancing guidelines state we should be standing six feet apart to limit the spread of the Corona virus.

For people with Cystic Fibrosis (CF), social distancing has always been a way of life. Being close to others with the disease puts them at increased risk of getting and spreading dangerous germs and bacteria, a term referred to as crossinfection. Not only are these dangerous germs difficult to treat, but they can lead to worsening symptoms and faster decline in lung function for those with CF.

Thats why its important for people with CF to stay at least six feet away from others with CF and anyone with a cold, flu, or infection.

Ive listened to this audiobook, Five Feet Apart, a few times now.

Stella Grant a teen living with CF describes her experience living with chronic illness:

Counting out exact doses of multiple medications. Being extra careful to not forget one. Careful to not accidentally overdose. Careful to take them at the specified time. Missing out on social events due to a flare up of symptoms. Going to the hospital because of catching a cold. Were basically doctors by the age of twelve.

I feel this. All the work that goes into my fight with my disability. Chronic pain. Chronic fatigue. Chronic migraines. Chronic asthma. Memory loss. Aphasia. Photophobia. Phonophobia. Post concussion syndrome. Brain damage.

I cant begin to understand what CF individuals have to go through. I dont know their fight. But I can begin to connect with the things we feel. Like the isolation. The complicated relationships. The chronic everything.

Even if you dont have a chronic condition, I still recommend reading this book.

We are all fighting in this pandemic. We can all begin to relate to those who need to always keep social distancing in mind.

We can all begin to see each other.

Hilary is a Toronto-based non-fiction writer and UofT masters student. Hilary is recovering from TBI, PCS and spends much of her free time on FaceTime with Isla, her baby niece.

This post originally appeared on her Instagram, @halite_brain_beads

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Two reasons why the market is missing a trick with CF Industries Holdings Inc – Stockopedia

Posted: at 6:07 pm

Buying quality merchandise when it's on sale is a stock market strategy used by some of the greatest investors in the world - including Warren Buffett. After theturmoil we've seen in 2020, there are signsthat stocks likeCF Industries Holdings Inc(NYQ:CF) could fit that bill. But how can you tell?

TheCF share price has moved by-37.3%over the past three months and its currently trading at24.59.

In volatile conditions,many investors are keen to buy what they think are cheap stocks- but this can be a mistake. It's importantknow the difference between a genuine bargain and a value trap - and often, the quality of the stock makes all the difference.

The good news is thatCF scores well against some important financial and technical measures.It's a large-cap share with strong exposure to twovery influential drivers of investment returns: high quality and a relatively cheap valuation.

To understand where that shows up,here's a closer look:

GET MORE DATA-DRIVEN INSIGHTS INTO NYQ:CF

Good quality stocks are loved by the market because they'remore likely to be solid, dependable businesses. Profitability is important, but so is the firm'sfinancial strength. A track record of improving finances is essential.

One of the stand out quality metrics forCFis that it passes8of the9 financial tests in the Piotroski F-Score. The F-Score is a world-class accounting-based checklist for findingstocks with an improving financial health trend. A good F-Score suggests that the company has strong signs ofquality.

While quality is important, no-one wants to overpay for a stock, so an appealingvaluation is vital too. With a weaker economy, earnings forecasts are unclear right across the market.But there are some valuation measures that can help, and one of them is the Earnings Yield.

Earnings Yieldcompares a company's profit with itsmarket valuation (worked out by dividing itsoperating profit by its enterprise value). It gives you a total value of the stock (including its cash and debt), which makes it easier to comparedifferent stocks. As a percentage, the higher the Earnings Yield, the better value the share.

A rule of thumb for a reasonable Earnings Yield might be5%, and theEarnings Yield forCFis currently8.06%.

In summary, good quality and relatively cheap valuations are pointers to those stocks that aresome of themost appealing to contrarian value investors. It's among these shares that genuine mis-pricing can be found. Once the market recognises that these quality firms are on sale, those prices often rebound.

Finding good quality stocks at attractive prices is a strategy used by some of the world's most successful investors. If you want to find more shares that meet these rules, you can see a comprehensive list on Stockopedia's StockRanks page.

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Two reasons why the market is missing a trick with CF Industries Holdings Inc - Stockopedia

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