Is the US setting the bar too low for new CF drug? Canada says it’s not worth it

From The Globe and Mail:

Canadian governments should not pay for a $250,000-a-year cystic fibrosis medication because it is not clear the drug actually improves patients’ health, according to a fresh review that has devastated some with the potentially fatal lung disease….

Screen Shot 2018-10-15 at 9.06.47 AMIn a report released on Thursday, the CADTH’s expert panel said its review of the Orkambi clinical trials and other studies found the drug produced only slight improvements for about one in four patients.

The report also said Orkambi’s Boston-based maker, Vertex Pharmaceuticals, would have to slash the drug’s price by more than 98 per cent to satisfy the agency’s value-for-money analysis.

“[Orkambi] is better than nothing. It’s better than placebo,” said Trevor Richter, director of the CADTH’s Common Drug Review, which oversees the review process for non-cancer medications. “But the benefit is really small. Not only is it small, but there’s a huge amount of uncertainty about what the actual size of that benefit is.”…

Vertex called the agency’s recommendation “deeply disappointing.”

Patients, who say the drug benefited them, are suing.

Mr. Richter of the CADTH said it is difficult but necessary sometimes to disappoint patients.

“We don’t enjoy delivering recommendations that are seen as negative,” he said. “In the end, we are an evidence-based review process. It’s rigorous and it’s transparent.”

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KHN: Trump Administration Sinks Teeth Into Paring Down #Drug #Prices

This story notes that CMS approved a Medicaid waiver from Oklahoma in June, but denied a Massachusetts request for a similar exception to existing rules. File_000

Oklahoma’s plan would expand that to negotiate additional prescription price reductions based on value-based purchasing agreements.

Still, CMS’ recent rejection of a related Massachusetts proposal makes it difficult to believe negotiating drug prices will really happen, said Sara Rosenbaum, a professor of health law and policy at George Washington University.

That proposal would have allowed Massachusetts’ Medicaid program to choose drugs 

based on cost and how well the medicines work.

“They have been very good and quite careful with their [Medicaid] program and so why not let them try this?” Rosenbaum said.

Full story below.

Three months after President Donald Trump announced his blueprint to bring down drug prices, administration officials have begun putting some teeth behind the rhetoric.

Many details have yet to be announced. But experts who pay close attention to federal drug policy and Medicare rules say the administration is preparing to incrementally roll out a multipronged plan that tasks the Centers for Medicare & Medicaid Services (CMS) and the Food and Drug Administration with promoting competition, attacking the complicated drug rebate system and introducing tactics to lower what the government pays for drugs.

downloadMark McClellan, director of the Duke-Margolis Center for Health Policy in Durham, N.C., and a former CMS administrator, said that although none of the initial steps has “fundamentally transformed drug prices,” there is “a lot going on inside the administration.”

Two HHS officials who are rolling out the plan, Dan Best and John O’Brien, described their efforts to Kaiser Health News not as a public relations strategy but a push to reform the system.

“This administration is trying to go after root causes” of high drug prices, said Wells Fargo analyst David Maris.

But others are not so optimistic.

Ameet Sarpatwari, an instructor in medicine at Harvard Medical School in Boston, said policies the administration has rolled out thus far “alone will not translate into meaningful cost savings for most Americans.”

Broadly, the strategy falls under a handful of steps:

1. Attacking The Rebates

Health and Human Services Secretary Alex Azar has said Americans “do not have a real market for prescription drugs” because drug middlemen and insurers get a wide range of hidden rebates from drugmakers, but those savings may not be passed on to consumers or Medicare. In July, the administration submitted a proposed rule that could change the way rebates are handled.

Details of the proposal have not been made public. But O’Brien, a deputy assistant secretary at HHS, explained during a recent conference on federal drug spending sponsored by the Pew Charitable Trust: “You don’t have to use market power to get rebates, you can use market power to obtain discounts, to actually lower the price of the drug on the front end.”

Umer Raffat, an investment analyst with EverCore ISI, said “it’s not clear [that drug prices are going down]” but the “rebate structure is changing.”

2. Bringing More Negotiation To Medicare

This week, CMS Administrator Seema Verma announced that Medicare Advantage insurers can use a step-therapy approach to negotiate better prices for Part B drugs — those administered in hospitals and doctors’ offices. These private plans will be allowed to require patients to first select the least expensive drug before stepping up to more costly drugs if the original medications aren’t working.

The administration is also looking at ways to introduce more competition into Part B drug purchasing. That idea was mentioned deep inside the annual Medicare outpatient payment rule released last month.

Peter Bach, director of Memorial Sloan Kettering’s Center for Health Policy and Outcomes in New York, pointed to the possible introduction of a competitive purchasing program in which a firm negotiates with drugmakers to buy their drugs and then sells them to the doctors and hospitals that will administer the medications. Bach said that helps ensure that hospitals and doctors can’t make more money by prescribing more expensive drugs.

Currently, Medicare pays the average sales price plus 6 percent to doctors or hospitals when they purchase drugs, a pricing mechanism that can benefit the providers if the drug costs go up. If there were a third party buying the drugs, it would “have a huge effect,” Bach said.

3. Paying For Value

Trump’s blueprint calls for CMS to encourage “value-based care” to lower drug prices, shifting from paying a set fee for drugs to basing payments on how well the patient does on them.

Louisiana’s Medicaid program could show the way. The state is working with CMS to explore a subscription-based model to pay for hepatitis C medicines. Louisiana would pay a fixed price to a drug manufacturer that would then get unlimited access to treat patients enrolled in Louisiana’s Medicaid program or in prison.

The program would move “from a big payment upfront to paying less over time based on actual outcomes,” said McClellan, who also serves on the boards of health care giant Johnson & Johnson and insurer Cigna.

CMS also approved a Medicaid waiver from Oklahoma in June. Medicaid programs are allowed to negotiate drug prices. Oklahoma’s plan would expand that to negotiate additional prescription price reductions based on value-based purchasing agreements.

Still, CMS’ recent rejection of a related Massachusetts proposal makes it difficult to believe negotiating drug prices will really happen, said Sara Rosenbaum, a professor of health law and policy at George Washington University.

That proposal would have allowed Massachusetts’ Medicaid program to choose drugs based on cost and how well the medicines work.

“They have been very good and quite careful with their [Medicaid] program and so why not let them try this?” Rosenbaum said.

4. Tackling Foreign Drug Costs

Pharmaceutical makers often sell their drugs at substantially lower prices in many foreign countries than they do in the United States. Trump emphasized in May that “it’s time to end the global freeloading once and for all,” saying U.S. consumers were paying part of the cost of the medicines that patients in other countries use.

He directed U.S. Trade Representative Robert Lighthizer to address the situation. Lighthizer’s office declined to comment.

When Sen. Todd Young (R-Ind.) asked during a Senate health committee hearing in June whether trade agreements with other countries should be used to “level the playing field,” Azar’s response was swift: “We absolutely believe we should be using our trade agreements to get them to pay more even as we have our job to pay less.”

Avalere Health President Matt Brow, who has been involved in talks with the administration, said it’s clear the focus on overseas pricing isn’t going away and the administration is “talking a lot about how to get the president what he wants.”

5. Increasing Competition

FDA Commissioner Scott Gottlieb has become the Trump administration’s lead proponent for increasing competition among drugmakers.

Competition resonates with Americans “because people see it every day in their experience in Costco and other places,” said Rena Conti, an assistant professor at the University of Chicago.

Gottlieb has announced plans to bolster the use of generic drugs and an “action plan” to encourage the development of biosimilars, which are copycat versions of expensive biologic drugs made from living organisms.

And to combat anti-competitive behavior in the market, Gottlieb said the FDA has passed along information to the Federal Trade Commission and hinted at potential action to come: “I think we’ve handed them some pretty good facts.”

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

 

Cambridge Mass. doc leads call for reform of “dysfunctional” pharmaceutical system.

Lead author Adam Gaffney is a pulmonologist and critical care physician at the Cambridge Health AllianceFile_000. Here’s the summary, from the BMJ:

Key messages

  • The US and Canadian pharmaceutical systems are dysfunctional

  • Costs are exorbitant, commercial goals distort drug development, misleading promotion fosters misuse, and medications are too often unaffordable for patients

  • We propose reforms that would provide universal drug coverage without fees at the point of use while reducing prices through negotiations with drug firms and, when needed, compulsory generic manufacture

  • Innovation would be enhanced through patent reform and by establishing new public agencies to fund drug development and clinical trials

  • Drug safety, efficacy, and prescribing quality would be improved by raising standards for approval and safety monitoring

  • While the proposals face formidable political obstacles, a popular mandate exists for pharmaceutical reform in both nations

Make your own #pharmaceuticals Thursday at the #CambSciFest

Probably not, but you never know. Maybe someone will get lucky.

Also know that today’s (Thursday) cancer mini-golf tournament is rescheduled

IMG_1114
2012 File photo

to Friday.

Be a Medicine Hunter

April 19 @ 1:00 pm – 4:00 pm
Novartis22 Windsor St. Cambridge USA 

Novartis scientists are hunting for new medicines that will change the practice of medicine and improve patients’ lives.

Meet our scientists on a self-guided journey where you will explore authentic laboratory tools and innovative scientific methods on your quest to discover new medicines. Collect stamps in your passport for each location visited along your discovery path and receive a completion badge at the end of your journey.

Making Medicines with Amgen

April 19 @ 1:00 pm – 4:00 pm
Amgen360 Binney Street
Cambridge, MA 02141 United States 
+ Google Map

Have you ever wondered how medicines are actually made? Come visit Amgen and make a batch yourself! Walk through a mock version of Amgen’s biologics manufacturing process, as you thaw and culture “cells”, purify and test them, and lastly, walk away with your final product—a “medicine” that can change the life of a patient in need. To ensure you have the best experience making medicines with Amgen, please pre-register for a time between 1:00 – 3:30 PM 

Neurodegenerative Diseases & Nanocapsules at Biogen Community Lab

April 19 @ 4:00 pm – 6:30 pm
Biogen Community Lab225 Binney St. Cambridge USA + Google Map

Biogen’s Community Lab will be hosting teens (and their parent or guardian) to adventure with us on two awesome rotating events. Event 1: Understanding Multiple Sclerosis, a devistating neurodegenerative diseases and how it affects patients all over the world, and how Biogen combats this diseases. Event 2: Ever wonder how nanocapsules are made, and how medicine delivery happens in your body. Well come join us here in the Community Lab and maybe we’ll answer some of those lingering questions about medicines and the drug…

From today’s Boston Globe: Partners and Trump, health costs and another expensive drug

STAT News reports that Partners Healthcare CEO Dr. David Torchiana was among the health care executive who met with President-elect Donald Trump Wenesday. 

Torchiana has been among those arguing that repeal of the law could disrupt millions of families who have relied upon it for insurance coverage.

“De-insuring 20 million people, many of whom were probably Trump vPHS_Logooters, is a pretty difficult thing to do politically,” Torchiana said in November, according to the Boston Globe. “Figuring out what the pathway is to altering the ACA and doing so in a way that actually doesn’t increase the cost of health care is pretty difficult. There’s not a simple policy solution to it.”

Torchiana did not reveal what he said about the Affordable Care Act to Trump. Partners spokesman Rich Copp told STAT that Torchiana was invited by the president-elect. “They had a conversation that touched on a wide range of health care policy issues, including affordability, access, quality and biomedical research,” Copp said.

$370,000 drug

Also,  Adam Vacaro of the buiness section reports that Biogen Inc.’s new drug “to treat a rare  genetic disorder in children will be priced at about $375,000 a year per patient — its most expensive medicine ever — the Cambridge biotech company said Wednesday.”

File_000Worth noting that drug companies were once so reluctant to make drugs for rare disease that Congress had to pass The Orphan Drug Act to entice them. It seems that now they just charge a lot of them.  Could it be that these rare diseases the low-hanging fruit for the developers of biologics?

Worth revisiting former Globe reporter Steve Heuser’s 2009 piece on how Genzyme sought out patients around the world with a rare condition and convinced governments to pay the full-price for the drug–  $160,000 a year.

Jose Gonzalez felt indescribably lucky that his little girl, wasting away beneath hibiscus vines in Central America, had been found. But for Genzyme, it wasn’t luck. It was another step in a remarkable business strategy: In countries from Colombia to Taiwan to Libya, the Cambridge firm has compiled an extraordinary track record of searching out patients like Tania, providing desperately needed treatment, and then successfully pressing their governments, even poor ones, to pay full price for the most expensive drugs in the world.

That strategy has helped Genzyme bring in more than $1 billion a year on Cerezyme alone, and to develop an arsenal of similarly expensive drugs for other rare diseases. And Costa Rica would soon learn a lot about Genzyme’s determination to be paid when it considered the cost of saving Tania Gonzalez, how it would drain resources from other patients, and decided that its answer was “no.”…

What (Genzyme)  won’t do is offer a discount. Although discounts are becoming more common on pharmaceuticals sold in lower-income countries, Genzyme follows a “full price or free” model, and works hard to be sure it’s the former.

“If you’re going to give on the price someplace, everyone’s going to ask for a deal, and then you’ve got a massive mess – so from day one, it was one price,” said Alison Taunton-Rigby, a former Genzyme executive who was with the company when it began setting its price policy.

Geoff McDonough, current head of the division that sells Cerezyme, says the policy allows the company to maintain the profit margin needed to research future drugs, while also keeping a door open for the truly impoverished.

“We’ve chosen a way here that’s been consistent across the company, and has met the specific needs and complexities of patients suffering from very rare diseases,” he said.

It is a balancing act, he says, that amounts to an “extreme example” of a discounting policy: the company currently provides the drug free to more than 300 patients worldwide.

Back to Partners, this time on costs

 letter from BU’s Alan Sager on the many confusing interpretations of health that is health care costs in Massachusetts:

David Torchiana,  president and CEO of Partners HealthCare, once again has recited Commonwealth Fund analyses of federal survey data showing that Massachusetts health insurance premiums are a lower share of median income than prevails nationally (“First, do no harm”).

But federal data on actual health spending contradict Torchiana. Massachusetts health spending per person was 36 percent above the US average, the highest in the world. After deducting Medicare and Medicaid dollars, private Massachusetts health costs per person were 40 percent above the US average, an excess of $11 billion over national average costs. Meanwhile, median income here was only 20 percent above the US average.

 

 

 

#STATnews asks: Are #Alzheimer’s numbers inflated?

CaptureIn her “Gut Check” column, STAT’s Sharon Begley notes that a NEJM report  from Boston University’s Framingham Heart Study claims that “The percent of people developing dementia each year is falling significantly… raising hope that some cases can be prevented and, possibly, that the worst forecasts of a “looming dementia crisis in the United States are overblown

The study notes that other evidence has the Alzheimer’s Association “sticking with its projection that, by 2050, the prevalence of Alzheimer’s will nearly triple, to 13.8 million Americans.

… The reluctance to dial down those forecasts might reflect “public health catastrophism,” added Dr. Jeremy Greene of Johns Hopkins, who, together with Jones, coauthored an accompanying perspective article on the Framingham study.

“It’s risky for advocates [to temper the most dire forecasts] for fear that it might bring a loss of funding,” Greene said.”

For more on this, see this list of stories on the topic of pharmaceutical support for non-profit patient groups. A bit dated, but USA Today offered an update in January.

Health charities say they work with drug companies in the search for cures. Corporate support is a standard form of fundraising for non-profits. The stories linked above question whether those relationships — and the dollars that come with them — bias these health groups. Will they challenge the efficacy of a new drug if they get tens of thousands from the drug’s  maker?

So, how much does the Alzheimer’s Association get from drug makers?  No one knows –non-profits are not required to report donations. Deep into its annual report, you’ll find a list of donors, but not exact amounts of donations.

$100,000-$249,000

Include:

Actavis

Amgen Foundation

AstraZeneca

Axovant

Biogen Idec

Eli Lilly

Forum Pharmaceuticals

Genentech/Roche

Janssen Pharmaceutical

Merck

Some of the same companies sponsored the group’s annual conference.

 

 

 

Massachusetts thread in NYTimes story on drug price rekoning

The New York Times reports:

As complaints grow about exorbitant drug prices, pharmaceutical companies are coming under pressure toHIT DEST disclose the development costs and profits of those medicines and the rationale for charging what they do.

So-called pharmaceutical cost transparency bills have been introduced in at least six state legislatures in the last year, aiming to make drug companies justify their prices, which are often attributed to high research and development costs.

One of them is Massachusetts. Also,Vertex comes up for the price of its CF drug — see recent post .

Finally, Tufts Center for the Study of Drug Development takes a knock for its oft-cited development cost reports.

Pharmaceutical executives do not typically tie the price of any particular drug to its development cost. But they do say that their sales have to recoup their investment in research and development if the companies are to stay in business.

They often cite the Tufts Center for the Study of Drug Development at Tufts University, which last year said companies spent an average of $2.6 billion to bring a drug to market, up from an estimate of $800 million in 2003. That includes the cost of failures. And almost half the figure is opportunity cost, the amount a company might have earned if it had invested money elsewhere rather than spending it on drug development.

Critics are skeptical of that figure, saying that the Tufts center gets funding from the pharmaceutical industry and uses data supplied by the drug companies, but does not disclose which drugs are used as the basis of the estimates.

Here’s what Tufts has to say about that:

Our Approach to Research

The Tufts Center for the Study of Drug Development (Tufts CSDD) conducts its research by working closely with government regulatory authorities and with drug developers and manufacturers of all sizes in the U.S. and abroad. Data are collected from the people who create it — pharmaceutical and biotechnology companies. They cooperate because they know Tufts CSDD will generate a comprehensive and objective picture of the drug development process, while strictly ensuring that individual company data are not disclosed. Adhering to stringent methods of quantitative analysis, Tufts CSDD validates its data and develops findings based on rigorous academic standards.

Worth noting here that not all their research is  from ad industry POV. For example:

Dr. Joshua Cohen utilizes his background in health economics to examine public policy issues that concern prescription drug reimbursement and market access. His areas of research include pharmacy benefits management as it relates to the Medicare prescription drug benefit, formulary regulations established by the Centers for Medicare and Medicaid Services, ethics and the distribution of pharmaceutical care resources, comparative effectiveness research, market access to biopharmaceuticals in the US and Europe, role of clinical and cost-effectiveness in clinical practice guideline development, drug development targeting neglected diseases, and decisions by drug regulatory agencies regarding prescription (Rx) to over-the-counter (OTC) switches.

  • Patient access to newly approved oncology drugs in US and Europe
  • Clinical, regulatory, and economic challenges facing pharmacogenomics
  • Prescription-to-over-the-counter switches in US and Europe
  • Trends in biosimilar market uptake