Medical Innovation

How Can Migraine Drugmakers Avoid the PCSK9 Debacle?

Posted on Friday, April 8, 2016

The Timmerman Report, By Luke Timmerman, April 4, 2016 One of the big ideas in biotech today is that you can prevent severe, chronic migraine headaches. This story has a lot of juicy ingredients: intriguing biology, bona fide medical value, and a potentially broad impact on millions of people. But if the drugmakers in this emerging category overplay their hand, and don’t pay careful attention to the new drug pricing reality, it could become a train wreck. Think about the PCSK9 drugs. Not long ago, biologists and cardiologists were raving about the cholesterol-lowering power of these antibodies from Amgen and Sanofi/Regeneron Pharmaceuticals. Both drugs won FDA approval to much fanfare last summer. Months later, sales of these drugs are barely a rounding error for either company. The vast majority of prescriptions (about 75 percent by estimates from Amgen) are being denied by payers via prior authorization procedures. And the denials keep coming on appeal. Payers want to see whether these $14,000 list-priced drugs are any better than cheap generic statins at what really counts – reducing heart attack and stroke. Until cardiovascular outcomes data rolls in from clinical trials in 2017, and data accumulates on their long-term safety, payers have tucked these drugs in a tidy little box. They...

Learn More

Real Endpoints’ RxScorecard™ predicted launch failure of major new anti-cholesterol drugs

Posted on Thursday, February 11, 2016

Analysis from 2015 showed that because PCSK9 inhibitors had not proved more than incremental value for most patients, payers would dramatically restrict their usage. Poor Q4 sales show that’s exactly what happened. Westport, CT, February 11, 2016 In a press release issued July 7, 2015,  Real Endpoints LLC (RE) predicted that the two soon-to-be-launched PCSK9 inhibitors from Amgen and Regeneron/Sanofi would not get substantial market uptake. RE based this conclusion on its proprietary tool RxScorecard™, which assesses the relative value of marketed and pipeline drugs from a patient’s and payer’s point of view. RE defines value as a full assessment of a drug’s efficacy, safety, convenience, adherence and economic attributes in comparison to other therapies in the indication. RxScorecard indicated that while both products offered potential efficacy benefits, they demonstrated little incremental value for most patients because of their lack of meaningful outcomes data at launch and their expected high pricing. As a result RE said last July that “Payers will have an extraordinary opportunity to control costs in this class.” That’s exactly what they did. Now that Q4 sales are in for both Praluent and Repatha, the magnitude of their launch failure is clear. Praluent sold only $7 million in Q4; Amgen has not released data on Repatha,...

Learn More

‘DrugAbacus’, a Comparative Cancer Drug Pricing Platform Powered by Real Endpoints’ RxScorecard™, is Launched

Posted on Friday, June 19, 2015

Westport, CT, June 19, 2015 – Real Endpoints (RE) is pleased to announce that its RxScorecard™ is the information technology platform supporting Memorial Sloan Kettering Cancer Center’s DrugAbacus – an interactive tool for considering the basis of cancer drug prices. Conceived by Dr. Peter B. Bach, Director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering (MSK), DrugAbacus was launched at www.drugabacus.org. MSK’s DrugAbacus generates a dollar-value for cancer drugs available in the United States (beginning in 2001 with Gleevec) based on a user’s settings for six different domains of potential value including the treatment’s survival benefit, side effects, and the incidence of the condition targeted. Memorial Sloan Kettering licensed the Real Endpoints RxScorecard platform for research purposes so users can generate “Abacus prices” and compare them with actual prices of these drugs at the time of launch in a visual and intuitive format. “We believe RxScorecard is the only tool available that provides a 360 degree comparison of the multiple components of a drug’s value in an independent, objective and systematic approach. We developed the IT platform to make it very easy for users to access our analysis, and are delighted that Dr. Bach selected this platform for DrugAbacus,” said Julie Eskay Eagle, RE’s Vice...

Learn More

Wall Street Journal publishes article on new cancer pricing tool from Memorial Sloan Kettering using RE’s RxScorecard technology

Posted on Friday, June 19, 2015

June 19, 2015 – Memorial Sloan Kettering Cancer Center, one of the nation’s top cancer hospitals, has created an interactive calculator that compares the cost of more than 50 cancer drugs with what the prices would be if they were tied to factors such as the side effects the drugs produce, and the amount of extra life they give patients. Please click here to access the article....

Learn More

Evidence and Endpoints in Cancer: Can Both Payers and Regulators be Satisfied?

Posted on Wednesday, March 27, 2013

How can oncology drug developers design their trials to satisfy both regulators and payers, and thereby maximize both access and commercial success?  It’s an increasingly urgent question as cancer programs continue to dominate pipelines, and drugs bills. Yet “there’s a fundamental tension” between the clear evidentiary base required to get a drug approved, and the emerging, sometimes inconsistent data sought by payers to justify funding that drug, notes Roy Baynes, SVP Oncology Therapeutics at Gilead Sciences Inc. Above all, he emphasizes the need for close engagement with payers, at least in Europe, from an early stage. Collaborating prospectively with governmental payers may help address the reality that “individual sponsors have little leverage,” he says.  (In the US, it’s not at all clear to us that payers want much to do with pharmas before products get very close to market.  And in oncology specifically, payers still can’t really say “no” to paying for products approved by the FDA; European governments have no such inhibitions.) There are some rules that now beginning to hold across the board, though, notwithstanding often considerable differences in evidentiary standards across treatment-type, positioning, and disease area. Having a “meaningful comparator is obviously important,” point out Baynes, who will be speaking at NextLevel Pharma’s PharmAccess Leaders’ Forum in...

Learn More

The Drug Assessment Process: Inadequate and Costly

Posted on Wednesday, January 16, 2013

The more I study it, the more I believe the process of drug assessment by payers and providers needs some serious attention (we’ll pay plenty at the Real Endpoints Symposium, March 11-12). But driving action requires concern — and so, who cares?  Answer is: anyone interested in improving the dismal economics of healthcare.  As we noted here, drug-spend inflation is skyrocketing, thanks largely to the cost of new drugs and, in particular, new specialty drugs.  And the best mechanism we have for controlling those costs is the process, led by Pharmacy & Therapeutics committees, for assessing the value of drugs and establishing utilization guidelines.  But best, in this case, doesn’t mean adequate. Most obviously, the process is redundant.  Several hundred payers and several thousand hospitals all have P&T committees, with associated clinical pharmacy specialists and other research infrastructure, looking at the same materials about the same drugs…and by and large coming up with pretty similar answers. It’s slow and under-productive.  Based on our admittedly unscientific survey, P&T committees at most mid-sized and smaller plans meet quarterly and make decisions on, maybe, 13-15 new drugs per year.  They don’t have the time or bandwidth to re-assess new data on existing drugs that could change their formulary status. It’s oddly limited. ...

Learn More

Reimbursement “Furies” Real, But Won’t Avenge Pharma Til 2017, Says Citi

Posted on Tuesday, December 4, 2012

“Beware the Three Furies,” warns Citi analyst Andrew Baum.  In a report for pharma investors published Nov. 29., Baum turns to classical mythology to describe shared savings models, drug pathways and ACOs — the forces that will soon dominate US health care plans. He’s chosen an interesting analogy: The Three Furies were goddesses of vengeance, who punished the wicked for their crimes; they’re also described as “tormenting those who have yet to atone for their sins”. So we, and plenty of others, agree that pharma should be nervous. “Reimbursement, not R&D pressure,” is the is the biggest risk facing pharma investors, writes Baum. But although “alarmed” by the drivers, and potential consequences, of US healthcare cost containment (particularly given the dire state of the EU), Baum reckons drug firms’ earnings won’t feel the full brunt of US pricing pressure “until at least 2017.” He argues that for the next five years, the revisions to the US healthcare system will have the greatest impact on medtech, hospitals, and diagnostics, with less focus on pharmacy-related costs.  Even in the case of drug pathways, singled out as the most important long-term structural risk to the biopharma industry “meaningful adoption will remain slow” in Baum’s view. To back that up, he cites research by...

Learn More

Want Better Access? Then Take Some More Risk

Posted on Tuesday, November 20, 2012

Biopharma is an industry seeped in an above-average concentration of risk. Scientific, clinical and regulatory uncertainties add to more typical commercial and market-driven risks. Given that, you’d think pharma execs would be a little more willing to stick their necks out and embrace (or at least explore) change. Some are. They get that payer cost-pressures and pipeline productivity challenges are forcing new, make-or-break approaches to clinical development, payer interactions, and commercial positioning. Yet toward the by-now-rather-less-radical idea of more closely integrating regulatory and HTA requirements, the pharma sector’s attitude is “somewhat capricious,” notes Mel Walker, recently appointed VP Market Access at Otsuka Pharmaceuticals Europe, after six years at GSK. That even a few companies still resist this idea seems odd, since it’s pharma (along with patients) that has most to benefit from a more harmonized regulatory-HTA process. Some drug industry execs are worried that putting regulators in the same room as HTAs — an idea that has been piloted in Europe, in the context of scientific advice, since 2010 — “may lead to HTA somehow influencing regulators to say ‘no’ to more products”, illustrates Walker, who will chair a discussion on HTA/regulatory convergence at the PharmAccess Leaders’ Forum in Berlin later this month. Alasdair Breckenridge, chairman of the UK regulator...

Learn More

Drug Approvals Need Shades Of Grey

Posted on Monday, November 12, 2012

FDA’s Endocrinologic and Metabolic drugs advisory committee on Nov. 8 voted 8-4 in favor of recommending Novo’s latest insulin degludec (Tresiba) for approval. The detailed debate and deliberation underscored how unsuitably black-and-white the drug approval process is.  In the end, FDA (just like the European Medicines Agency), will say either ‘yes’ or ‘no’.  Yet the difference between acceptable risk and unacceptable risk among drug treatments isn’t a well-defined one. Nor is the threshold or criteria separating insufficient data from sufficient data. Now, as it happens, in Tresiba’s case the committee also unanimously voted in favor of a post-approval CV outcomes trial, reflecting lingering uncertainties about the drug’s risks.  But that’s unusual — and the conditions, timelines and the potential impact of the results remain unclear. What’s needed is a drug approval system in shades of grey — incremental steps on the way to full approval, for drugs that aren’t definite rejects, nor dead-cert approvals (e.g. most drugs). Programs including FDA’s accelerated approval and EU’s conditional approval go some way towards that, but only rarely. (Just 6 drugs were approved conditionally by EMA between 2009-2011, less than 10% of the total) A handful of projects run by regulators and academics around the world have been seeking, for several years now, to define and progress new, adaptive approaches to...

Learn More