Earlier this week the FDA approved ocrelizumab (Ocrevus) as the first disease-modifying treatment specifically for primary progressive multiple sclerosis (PPMS).
Made by Genentech, the biologic reduced the risk of 12-week disability progression in PPMS by 24% relative to placebo, and also proved to be an effective treatment for adults with relapsing-remitting multiple sclerosis (RRMS).
The drug's price tag is a costly $65,000 per year, but experts agree that it's comparable with other MS therapies, especially as the price of MS drugs has continued to increase over the years.
What's your opinion of the magnitude of benefit for this drug? Given the differences between clinical trial populations and real-world patients, would you expect to see this kind of risk reduction in routine practice?
, Johns Hopkins Bloomberg School of Public Health: Ocrelizumab represents an important new treatment choice for those with MS, especially the minority of patients that have primary progressive disease. With that said, the clinical benefits are modest and as is typically the case with new approvals, long-term safety and efficacy are unclear.
, Icahn School of Medicine at Mount Sinai: Ocrelizumab showed excellent effects on reducing disease activity and disability in relapsing MS; the effect in primary progressive MS was more modest, but this is the first agent to successfully alter the course of primary progressive MS. In relapsing MS, the experience in practice was similar to that seen in the clinical trials. For primary progressive MS, we will need to learn from experience.
, NYU Langone Medical Center: I think that the real-world benefits of this drug will be large, especially for patients with relapsing-remitting MS. It is similar to Rituxan [rituximab], which we have been using off-label for years. For patients with progressive MS the benefits will be more modest. It slows progression, but won't get patients out of wheelchairs.
, Yale School of Medicine: The drug is highly efficacious in patients with relapsing remitting MS and expect to see the same risk reduction in routine practice for selected patients with new onset, inflammatory disease. The efficacy of the drug is significantly more modest in primary progressive disease. While I agree with its approval, we need to better understand the mechanism of the neurodegenerative aspect of primary progressive MS which appears not to be mediated by the peripheral adoptive immune system.
, University of Chicago Medicine: This drug is in the same league as the best available drugs for RRMS; the FDA believes there is an effect on progressive MS; known side effects are minimal; and the price is not excessive (in relative terms). This is a quadfecta! And it should be effective for many types of "real world" MS because the trials spanned RR and PPMS.
, Perelman School of Medicine at the University of Pennsylvania: It's tremendous and we expect to see these significant anti-inflammatory effects in practice.
Is $65,000 per year a fair price for the stated benefit?
Alexander: Depends who you ask. One thing is certain – this price is going to be closely scrutinized, and rightly so. We need to be talking about what constitutes a "fair price" more often, instead of ducking from this question.
, University of Colorado School of Medicine: Fair is a challenging concept in the world of MS therapies. While the wholesale cost as quoted will certainly be competitive with all presently-approved medications, the other MS disease modifying therapies are subject to substantial and nontransparent cost reductions which shield the actual costs. That said, ocrelizumab will rank well in cost-benefit analyses, compared to other approved therapies.
, Cleveland Clinic: With MS therapies, we haven't figured out a good way to compare price and benefit. Nonetheless, the price of ocrelizumab appears to be approximately on-par with other approved MS therapies.
Hafler: As the interferons and Copaxone were originally priced at ~$15,000, it is hard to say what a fair price would be. However, I applaud Roche/Genentech in bringing the price below market hopefully allowing us to partner with insurers to use ocrelizumab as a first-line drug.
, Icahn School of Medicine at Mount Sinai: I regret the extraordinarily high price for all marketed disease-modifying therapies. Nonetheless, Genentech is to be commended for at least reversing what has seemed to be a never-ending and unwarranted escalation of prices for MS DMTs [disease-modifying therapy]. Ocrevus has been priced lower than virtually every other drug.
, UC San Diego School of Medicine: Genentech should be commended for reducing the annual cost compared to other disease modifying therapies.
, Washington University School of Medicine in St. Louis: While $65,000/year is high, ocrelizumab is one of the least expensive MS therapies. Roche clearly listened to many neurologists who were concerned about rising drug prices.
Reder: This is uniquely low for a first-in-class, highly effective MS drug. Some other new drugs attempted to use "unique mechanism, highly effective" as leverage to price their drug above prevailing rates. Of course, the ideal would be a drug as inexpensive as sunlight, but there are significant development costs for biologic drugs.
Prices for MS drugs in general have risen considerably over the years -- how much of a problem is that and what can be done about it?
Fox: Given the high cost of MS drugs, research should focus on identifying the patients who are most likely to benefit from therapy. If we can direct the right therapies to the right patients, then I think we can better manage the growing overall cost of MS therapies.
Corey-Bloom: I believe that Genentech's lower pricing of ocrelizumab will actually encourage other pharmaceutical companies to go the same route, especially if they want to maintain their market share. And I hope that clinicians caring for patients with MS will take every opportunity to encourage pharmaceutical companies to do so.
Alexander: There is a growing consensus that current levels and increases in drug prices are unsustainable, and MS products are an important part of this mix. Our current system doesn't price products based on value. While it's risky to make predictions, there is a lot of policymaker interest in many different potential solutions, ranging from Medicare price negotiations to price-gouging bills.
, Cleveland Clinic: The high price of MS medications is a significant contributor to the high overall cost of MS care. I anticipate that ultimately legislative action will be necessary to lower medication costs in the U.S.
Miller: Ultimately the inappropriate escalation of drug prices in the U.S. will have to be addressed through legislative action. Continued pressure from patients on their government representatives will be necessary, supported and orchestrated by not-for-profit organizations, such as the National MS Society, and by professional organizations, such as the American Academy of Neurology.
Corboy: The cost escalations for MS therapies over the last 15-years defy all logic and are a substantial problem. They result from a combination of pharmaceutical companies taking advantage of our free market system and the complete abdication of political leaders. Until the legislators and president of the U.S. try to actually improve healthcare, nothing will change.
: MS is now labeled as one of the most expensive conditions to treat and can become a significant issue with possible changes in healthcare laws. Using effective medications early on in the disease can help reduce the burden of disability of patients in the long run, which will ultimately be cost saving for society. Furthermore, the introduction of generic medications is required to bring down the price of disease modifying therapies.