Change in Chicago: Boomerang 2
In 1983, the year before Henry Waxman got the Waxman-Hatch Act on the Statute Books he got the Orphan Drugs Act passed. Here’s how it happened.
In a wonderful 2016 self-publication, Abbey Meyer, a self-styled Connecticut housewife, outlines a story that began when her eldest son David was born in 1968. The book is downloadable for free and is well written and compelling.
When David was only a few years old he began to show signs of what her doctor called tics. As problems grew at school, she became increasingly concerned for him.
Then one Sunday in a newspaper she read an article about Tourette Syndrome. This led to clinical appointments and treatment. The standard drug, haloperidol, turned David into a zombie. He was enrolled in a clinical trial of a new drug pimozide, which was better, but supplies were cut off in 1976 when McNeil pharmaceuticals opted not to develop it further. Her doctor used the term orphan drug to explain what was going on.
An Orphan Drug was one that was likely to end up being used by less than 200,000 people – which the pharmaceutical industry figured was their break even point. Unless that many people took the drug, they claimed they were unlikely to make a return on their investment. Around 1980 concern was growing that this might mean no new drugs for Multiple Sclerosis, or Epilepsy or Lou-Gehrig’s Disease (Amyotrophic Lateral Sclerosis – ALS).
Meyer was astonished. She became active. Among the first people she tied up with was Marjorie Guthrie, the wife of Woody Guthrie who was campaigning for orphan diseases. A group of women mobilized pretty quickly. Muriel Seligman from Los Angeles had a son Adam with severe Tourette’s. She approached her Congressman, Henry Waxman. Waxman convened a hearing in June 1980 inviting all pharmaceutical companies. None turned up. There was no-one at the hearing apart from a few families with rare diseases and a reporter from the Los Angeles Times.
On the back of an LA Times article, Meyer was contacted by Jack Klugman who ran the Quincy MD show a widely watched medical series on TV. Klugman bought the idea of an episode that would have a boy (an actor playing Adam Seligman) testify at a Congressional Hearing in very emotional terms about the damage caused by lack of treatment.
The show Seldom Silent, Never Heard ran in March 1981. Its a must watch. There are 5 minute clips out there showing the key scene when Adam Seligman makes a pitch to Congress – but I can’t find the link at the moment.
Thousands of letters flooded in. Waxman convened a follow up hearing at which Quincy (Klugman) testified in person. There was standing room only. The Wall Street Journal laughed at Waxman and the idea that Congress might take advice from actors. But Nancy Kassebaum introduced a bill in the Senate, which Waxman introduced in the house that passed both houses in December 1982 and was signed into law by Ronald Reagan in January 1983.
A few days later, Meyer and others formed the National Organization for Rare Diseases (NORD) and began cataloguing all the conditions that might qualify – there were over 6000.
By this stage she had met Bill Haddad. Haddad, as Chair of the Generic Pharmaceutical Industry Association, was chasing a Bill to regularize the position of generic drugs – that later became Hatch-Waxman. Among other things he had made it clear that if the branded industry didn’t bring orphan drugs to the market, the generic companies would.
Haddad provided funds for an office for NORD in Danbury Connecticut. When the landlord heard what NORD was up to, he asked whether this meant AIDS patients might be visiting the premises. Meyer hadn’t thought of this possibility. The landlord told her she would have to go elsewhere but after a later “meal” with one of her supporters he made a several thousand dollar donation.
AZT turned out to be the first drug approved under Orphan Drug provisions, with almost all other drugs for AIDS and its associated infections being approved in the same way.
A key contact Meyer got from Haddad was Agnes Varis, the owner of AgVar a generic company. Varis was connected to everyone in the generic industry and the Democratic party.
At this stage rare diseases were assuming a shape fronted by ALS, multiple sclerosis (MS) and hemophilia. A drug called copaxone had shown benefit for MS but Meyer couldn’t persuade a branded company to pick it up. Among other things the patent status was obscure. It had been discovered in the Weizman Institute in Israel.
Varis strong-armed Teva, a small generic company based in Israel and the US to pick it up. It was approved under the Orphan Drugs Act and went on to blockbuster status, earning Teva over $1 Billion per year, and making Teva the leading generic company.
Get on the Train
Meyer’s book is a compelling read. But its difficult to avoid a slight feeling of deliverance from a ghetto on trains that turn up to transport us East to safety.
As of 2016, the branded drug industry is a $1,275 Billion dollar industry half of whose profits ($638 Billion) come from drugs brought to market in the standard way. The other half ($637 Billion) comes from drugs availing of the Orphan Drug provisions which allow for an easier ride through FDA and a longer period of monopoly.
The orphans have included Merck’s Vioxx, Lilly’s Cialis, Astra’s Crestor, Bristol Myers Squibb’s Abilify, Botox and many others among the best-selling drugs of recent years.
While it is possible to close some loopholes that lead to blockbusters being named orphans, the precedent of allowing high prices for drugs that will supposedly only ever occupy niche markets has laid a business model for personalized medicines. If genetic tests ever permit a tailoring of medicines to specific patient groups, the orphan argument will apply.
For most of this period, the Wall Street Journal had Henry Waxman as one of its prize targets claiming that he was to progress what the Troll under the bridge was in the Billy Goats Gruff.
But without mentioning him, as early as 2005 WSJ was trumpeting the fact that Orphan Drugs Act had been the savior of the pharmaceutical industry – not that an industry earning $638 billion from other sources really needs saving.
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This clip shows David, and what he was up against with the Officials quite startled .. and his parents ..
Quincy M.E. Seldom Silent Never Heard Quincy Piano Scene!
https://www.youtube.com/watch?v=xZyaGEIqvRs
Ed Silverman casts his eye over the $$$s in Orphan drugs, with loophole ..
Orphan drug sales will outpace all other meds, thanks to high prices
March 2, 2017
By: Ed Silverman
http://www.coteorphan.com/news/2017/3/2/orphan-drug-sales-will-outpace-all-other-meds-thanks-to-high-prices
“the data, which was released to coincide with Rare Disease Day, also emerges amid growing controversy over orphan drugs. Large price tags are drawing attention and there is growing concern that some drug makers are exploiting loopholes in a US law, which has prompted one lawmaker to start a probe.”
Interesting blog, here – yet another unsavoury aspect of the very murky world of BigPharma …….. and a particularly interesting link to Ed Silverman’s article, thanks Annie.
Silverman’s article makes specific reference to the new drug, Spinaraza, which is priced at an enormous $750,000 for first year of treatment and $350,00 thereafter.
My partner’s little grandson (aged 19 months) has recently been diagnosed with SMA (Spinal Muscular Atrophy) and Spinraza, which has had impressive results in clinical trials, could considerably enhance his quality of life.
The SMA Trust is now battling away with the NHS to try to make this drug available to babies and children like my partner’s grandson, but so far, it seems to be an uphill battle due to the prohibitive cost.
It is so very sad to see the parents of this very cute little boy first have to come to terms with the diagnosis that their baby has SMA Type 2 (and face realisation that your little child is never going to be able to walk and may well suffer serious respiratory problems due to muscle weakness) yet also learn that a new and effective drug is now available (Spinraza – FDA approved December 2016 and EMA approved April 2017) but is too expensive and out of reach – a very hurtful ‘double whammy’ if ever there was one.
At the same time I read (on various Facebook groups, Twitter etc) about people incarcerated and medicated against their will and kept on secure wards costing the NHS absolutely £ thousands ….. such wasted budgets that could be so much better spent on providing help to these poor little children who face such huge life challenges.
We can but hope that the NHS ‘powers that be’ in charge of making decisions about commissioning this orphan drug, Spinraza, will see fit to find the budgets necessary to allow treatment ….. it could make so much difference to his precious little life.
Or, better, still BigPharma (Biogen in this instance) play fair in pricing of orphan drugs (pigs might fly, eh?)
The ‘Orphan’ Appointment ..
Without a drug involved, GSK is involved in a court case ..
“you could have gone to any company but GSK”
https://www.thetimes.co.uk/edition/business/pascal-soriot-threatened-protege-over-joining-rival-glaxosmithkline-rx8nv9tkt
Comment
Nicholas Beale
9 hours ago
Restrictive covenants and gardening leave are commonplace in industry especially at this level. And disputes are generally settled amicably, with Chairman to Chairman discussion in especially sensitive issues. It seems odd that this has come to court – is there more going on here than the story suggests?
Luke Miels is President, Global Pharmaceuticals at GlaxoSmithKline.
https://www.vbprofiles.com/people/luke-miels-5887009c2cd3f4b692000225
Er, not quite ..
Commenting on the appointment,Emma Walmsley said: “Luke brings a combination of excellent R&D insight and a strong track record of commercial execution.
“We are now entering a critical period of commercialisation for our new pharmaceutical products and, over the next two to three years, we have important data to come on our early-stage pipeline. Luke will bring a strong new voice to the decisions and choices we will have to make for our Pharmaceuticals business.
Change in Manila: Boom Boom
Wrongful use of prescription medicine is the most pressing issue in the industry,
http://news.abs-cbn.com/business/07/12/17/tax-reform-a-boost-for-ph-healthcare-glaxo-smithkline
Baxter said, adding GSK was working with doctors and allied professionals to address this.
“he can’t stop the train”
What’s the @EFPIA Disclosure Code and why should you care?
#pharmadisclosure
https://twitter.com/GSK/status/885165260793139202
Archer @SArcher45 24m24 minutes ago
Replying to @GSK @EFPIA
Stop peddling heroin or as you call it “opioids”. People are dying. Disclose that.
Slate .. (King and Spalding) ..
http://www.slate.com/articles/news_and_politics/jurisprudence/2017/07/christopher_wray_will_not_be_trump_s_stooge.html
Change in C: G1, G2, G3 …
Ex-GSK CEO Andrew Witty goes biotech, taking a board seat at G1
Witty had his ups and downs during his 9-year stint at the top of the pharma giant, certainly, but he persisted in maintaining a focus on ethics and corporate performance
https://endpts.com/ex-gsk-ceo-andrew-witty-goes-biotech-taking-a-board-seat-at-g1/
all I heard back was that he “has nothing further to add.”
Wiki:
G1
Witty later claimed that he knew nothing about the China fraud and tried to pass the blame onto subordinates
G2
Illegal promotion of prescription drugs, its failure to report safety data
https://en.wikipedia.org/wiki/Andrew_Witty
G3
In March 2016 Witty announced that he was to stand down as chief executive
Senior Reporter, @BuzzFeedNews.
Mike HayesVerified account @michaelhayes 3h3 hours ago
Just in: Sessions, DOJ announces “largest health care fraud takedown in American history.” 412 defendants, including 56 doctors charged.
6:40 AM – 13 Jul 2017
July 13th, 2017 | 09:30 AM – 11:00 AM EST
Department of Justice, July 13, 2017
PLEASE STAND BY.
https://recapd.com/w-c88e15/
Stars in Your Eyes .. **********
“cannot resist”
http://www.pharmafile.com/appointment/514614/not-long-after-leaving-gsk-andrew-witty-dips-toes-back-water
Only a short while after leaving his position as CEO of GSK, it seems Andrew Witty cannot resist getting back into the industry, as he takes up a board position at newly-fledged biotech, North Carolina-based, G1 Therapeutics.
The appointment is a massive coup for the biotech that recently completed a $105 million IPO and the addition of industry-veteran to its board will only increase confidence in the company.
The biotech’s lead candidate is a CDK4/6 inhibitor that is designed to be used in combination with chemotherapy for NSCLC lung cancer and triple negative breast cancer.
Data on the drug’s performance in Phase 2 trials are expected to be released next year.
“We are thrilled to welcome Andrew to our board of directors,” said Mark Velleca, CEO of G1 Therapeutics. “Andrew led and oversaw the development, commercialization, and global market access for several leading pharmaceutical products at GSK. We anticipate leveraging *his wealth* of industry experience as we advance G1’s portfolio of potential best-in-class and first-in-class therapies.”
Witty’s experience will certainly be a bonus, after 30 year journey at GSK that resulted in him being appointed CEO and saw him lead the company in a *notable shift towards ethical practices* , including a drive to increase access to medicines in lower-income countries.
Witty will balance his new role with his position as a Director of Singapore Economic Development Board and his position on the Imperial College Commercialisation Advisory Board. He is also the Chancellor of the University of Nottingham.
“As someone who is passionate about the development of new and *meaningful medicines* , I am delighted to join the G1 board of directors,” said Witty. “The encouraging *early clinical data* generated by G1’s CDK4/6 inhibitors support their potential to *improve patient outcomes* in multiple oncology indications. I look forward to working with the G1 team at this exciting time of *corporate* growth.
Emma Walmsley, on her second poach, as presumably, she feels, her L’Oreal Experience does not quite cut the thrust ..
http://www.fiercebiotech.com/biotech/pfizer-s-medicinal-sciences-head-tony-wood-jumps-ship-to-gsk
Today is the anniversary of Stewart Dolin and capturing the mood is some exquisite music ..
,
http://fiddaman.blogspot.co.uk/2017/07/stewart-dolin.html#.WWpIdIWcGZ8
Almost all those linked between Paxil/Paroxetine/Seroxat have been made .. Sirs ..
Panorama asks if enough is known about this rare side effect
A Prescription for Murder?
and if doctors are unwittingly prescribing what could be a prescription for murder.
http://www.bbc.co.uk/programmes/b08zjyp1
Panorama
Is it possible that a pill prescribed by your doctor can turn you into a killer?
Over 40 million prescriptions for SSRI anti-depressants were handed out by doctors last year in the UK. Panorama reveals the devastating side effects on a tiny minority that can lead to psychosis, violence, possibly even murder.
With exclusive access to psychiatric reports, court footage and drug company data, reporter Shelley Jofre investigates the mass killings at the 2012 midnight premiere of a Batman movie in Aurora, Colorado. 24-year-old PhD student James Holmes, who had no record of violence or gun ownership, murdered 12 and injured 70. Did the SSRI anti-depressant he had been prescribed play a part in the killings?
Panorama has uncovered other cases of murder and extreme violence which could be linked to psychosis developed after the taking of SSRIs- including a father who strangled his 11-year-old son.
Panorama asks if enough is known about this rare side effect, and if doctors are unwittingly prescribing what could be a prescription for murder.
Wed 26 Jul 2017 21:00
BBC One
Reporter
Shelley Jofre
1.36
“There’s a lot of runway space out there for Seroxat
https://www.bing.com/videos/search?q=panorama+and+seroxat&qpvt=panorama+and+seroxat&view=detail&mid=B91E6C96E7D2A78E2263B91E6C96E7D2A78E2263&FORM=VRDGAR
let’s get the planes down …