What were the results?
Over a lifetime, the model showed that patients who received ropeginterferon alfa-2b-njft had more years alive (0.4), and higher QALYs (0.4), and higher cost (141,783).
What do the results mean?
The results suggest the benefits of treatment with ropeginterferon alfa-2b-njft outweigh the costs for a broad range of patients with PV.
Unfortunately Besremi didn’t work for me, I had to stop it immediately after 15 months because my hemoglobin dropped to 4 and I needed blood transfusions I was very critical . Please keep an eye out for your hemoglobin (I was doing the highest dose 500mcg). I don’t want it to happen to anyone else, good luck and God bless to all of you who are lucky to go into into remission.
At 500mcg, it is not surprising the cytoreduction was too severe. I am maintaining a complete hematologic response at 150mcg, but it is causing mild lymphopenia and borderline neutropenia. If I went much higher, my immune system would be seriously compromised. Our treatment needs vary. Many of us would not benefit from nor tolerate the max dose of Besremi. I am a strong believer in using the minimum dose necessary for the desired hematologic response.
Wishing you success in your next steps.
I’m so happy for you Hunter5582 ! I waited so long for this medicine to come to the US I’m so disappointed and I can’t do it anymore. I also thought it was a very high dose for me also but my mpn specialist thought I needed it because my platelets would not go down and still made me do Hydrox with it for all this time (15 months ). I developed hemolytic anemia from this. I’m being advised to start Jakafi at the end of the month when I’m done with all my steroids that I’m on for the treatment of this episode I had. Stay well and thank you again for all you do for us here. God bless you.
Jakafi is a logical next step having tried Besremi and HU. I expect you already know that the combination of Besremi and HU is usually avoided due to the risk of excessive myelosuppression.
Avoid/Use Alternative - ropeginterferon alfa-2b + hydroxyurea use alternative or monitor CBC: combo may incr. risk of serious infection, myelosuppression (additive effects) epocrates.com/online/drugs/...
Suggest reviewing the treatment goal for your case of PV. The primary risk factor is erythrocytosis, not thrombocytosis. There is not a linear increase in thrombosis risk as platelets rise above 450. There is a linear increased risk when the erythrocytosis is too high. It may be that you would tolerate a higher level of platelets providing the erythrocytes are at your individual goal. Noting that the benefits of any medication have to outweigh the adverse effects, perhaps a different goal is warranted in your case. If you wish to do so, perhaps you can consult again with your MPN Specialist about what the targets should be as you initiate treatment with Jakafi.
Wishing you all the best as you move forward.
That publication was a direct, biased advertisement from the manufacturer. However, you just provided a real-world-experience testimonial to the contrary, further proving that the very, very expensive medicine is not necessarily better for the welfare of the patient and in any way cost effective for the treatment of polycythemia vera. Haha!
Hi Kristin Marie,
It’s important to keep an open mind and not dismiss a research paper solely based on the affiliation of the authors. There may be cases where researchers from the same drug company conduct high-quality and unbiased research that contributes to the advancement of science and society.
Yes, everyone is entitled to his or her own opinion, whether the opinion is biased or unbiased.
The following is the advice from Scottish Medicines Consortium on besremi following a resubmission. The excerpt is from the weblink: scottishmedicines.org.uk/medicines-advice/ropeginterferon-alfa-2b-besremi-resubmission-smc2563/
ropeginterferon alfa-2b (Besremi®) is not recommended for use within NHSScotland.
Indication under review: as monotherapy in adults for the treatment of polycythaemia vera without symptomatic splenomegaly.
In a phase III study, ropeginterferon alfa-2b failed to demonstrate non-inferiority to hydroxycarbamide in treatment-naïve patients who required cytoreductive therapy and in patients who had a partial response to hydroxycarbamide.
The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Medicine details
Medicine name: ropeginterferon alfa-2b (Besremi)
SMC ID: SMC2563
Indication: As a monotherapy in adults for the treatment of polycythaemia vera without symptomatic splenomegaly.
Pharmaceutical company: AOP Orphan Pharmaceuticals AG
BNF chapter: Malignant disease and immunosuppression
Submission type: Resubmission
Status: Not recommended
Date advice published: 10 July 2023
Not a single haematologist on this Scottish Consortium…
The Scottish Medicines Consortium (SMC) is a committee of clinicians, pharmacists, NHS board representatives, the pharmaceutical industry and the public¹. Members of the committee consider a broad range of evidence in order to decide which medicines should be accepted for use by NHSScotland¹. The SMC has around 25 members³, including:
- Dr Scott Muir, the chair of the SMC and a consultant in respiratory medicine at NHS Lanarkshire⁴.
- Dr Alan MacDonald, the vice chair of the SMC and a consultant in public health medicine at NHS Fife¹.
- Dr Frances Macdonald, the vice chair of the New Drugs Committee (NDC) and a consultant in palliative medicine at NHS Greater Glasgow and Clyde¹.
- Dr Andrew Walker, the health economist lead for the SMC and a senior research fellow at the University of Glasgow¹.
- Three public partner members who represent the views of patients and carers: Ms Aileen Bryson, Mr John McFarlane and Ms Sandra Webster¹.
- Representatives from 14 NHS boards across Scotland, including doctors, pharmacists and senior managers¹.
- Representatives from the Association of the British Pharmaceutical Industry (ABPI), who provide input from the perspective of the pharmaceutical industry¹³.
You can find more information about the SMC and its members on their [website](^1^).
(1) Who we are - Scottish Medicines Consortium. scottishmedicines.org.uk/ab....
(2) Attending a Scottish Medicines Consortium Meeting - Information for .... scottishmedicines.org.uk/me....
(3) New SMC Chair appointed. healthandcare.scot/stories/....
(4) Who we are - Scottish Medicines Consortium. scottishmedicines.org.uk/ab....
(5) SMC | Scottish Medicines Consortium. scottishmedicines.org.uk/Home.
This drug is not available in UK in part due to its perceived value for the money or the lack thereof.
It should also be noted that Jakafi and Pegasys are both available in UK; apparently, both these drugs had provided acceptable justification of the treatment’s costs in relation to its health benefits.
Therefore, the same can be true for this new, super-expensive interferon if the manufacturer is willing to lower the annual price of US$190,000 to justify its cost vs benefits profile.
Previously, Anton Health had published an article which mentioned that analysts had expected the annual cost per patient per year (PPPY) to be in the $40,000 range. Here is the weblink: antonhealth.com/fda-approves-new-tx-for-polycythemia-vera-besremi/
»Analysts expect the annual cost per patient per year (PPPY) to be in the $40,000 range ».
Still worth it, if it spares the need of a SCT.
«. According to some sources, the average cost of an allogeneic SCT in the United States ranges from $200,000 to $500,000«
If you read this paper carefully, you'll find that the comparator arm in the study is hydorxycarbamide followed by "ruxolitinib", the latter of which was never dosed in their proud and conti clinical studies.
Actually , pegasys should be made a comparator in their cost-effectiveness study, however, in that case the manufacturer will lose big time. Haha.
If you read the paper even more carefully, you’ll notice they are comparing « on-label » medications, not off-label.
« Comparing two on-label medications in a scientific study is preferred because it allows for a more controlled and accurate assessment of their effectiveness and safety within their intended uses. Comparing an off-label medication with an on-label one introduces variables related to different indications, potential biases, and varying patient characteristics that could confound the results and make them less reliable. This practice helps ensure that study outcomes can be more confidently attributed to the medications being compared rather than external factors »
There you said it.
This paper is biased in selecting only the on-label medications to draw its analysis, absolutely for marketing purposes. This relates to a relatiely small percentage of PV patients, maybe less than 20%, as most PV patients are on hydroxycarbamide.
In real life, when a hematologist or an MPN specialist prescribes medications for the PV patient, she is making the recommedations for what is right for her patient, not really minding whether the medications are off-label or on-label.
Love the additional commentary on this interesting study by kristinmarie and Manouche . You both make excellent points.
Noting that hydroxyurea and Pegasys are both off-label for PV in the USA does mix the conversation up a bit. A head-to-head comparison of Besremi (ropeginterferon) vs Jakafi (ruxolitinib) would be the only cost-benefit comparison for on-label PV drugs in the USA. That masks the debate since hydroxyurea is in the NCCN guidelines as a first-line option for PV even though it is off-label. Pegasys is not addressed in the NCCN guidelines even though it is in common use. Regardless, Pegasys is a valid comparator for Besremi as well.
A more nuanced study would have compared all of the treatment options in common use: venesection-only, hydroxyurea, Pegasys, Jakafi. We will soon need to add Rusfertide to the mix of drugs to compare. We will have to see what the cost of rusferide will be.
To cost compare for the USA. (Note that costs can vary between plans)
Hydroxyurea 500 mg (60 ea): $25.00
Pegasys 180 mcg/0.5 mL (1 kit, 4 syringes): $4,177.00
Jakafi all doses (60 ea): $14,308.00
Besremi 2 -500mcg syringes: $14,232
The reality is that hydroxyurea is preferred by many insurance formularies because of the cost. Doctors in many healthcare systems are expected to prescribe the cheapest drugs available. Some doctors will buck the system they work for, but not all. Doctors in private practice have more latitude to prescribe based purely on individual patient needs/preferences, but must contend with insurance formularies when prescribing. It can take significant additional hours of work behind the scenes to get a more expensive drug authorized. This additional cost is not reimbursed.
Fortunately, Besremi was added to the NCCN guidelines as a first-line treatment option for PV. Hopefully this will make authorization easier for many people with PV. I expect that was the main purpose of this study. It is in a real sense marketing to the insurance companies and healthcare systems.
The bottom line is that insurance companies and healthcare systems are by their nature cost-driven. Increasing profits and/or reducing costs will always affect healthcare decisions. That is why we need to be our own best advocates. It is also why we have to support our providers, who are increasingly experiencing burn-out due to the challenges faced in providing healthcare. Providers who are willing to advocate for the best choice for their patients regardless of cost deserve our respect and support.
The cost-effectiveness study was based primarily on the on-label medication, however, you might want to read the Pharmaessentia August 4 new release with the misleading title of "New Study Demonstrates Ropeginterferon Alfa-2b-njft Is a Cost-Effective Treatment Option for a Broad Range of Patients with Polycythemia Vera".
The weblink: finance.yahoo.com/news/study-demonstrates-ropeginterferon-alfa-2b-120000137.html
One might conclude that our friends from PharmaEssentia are a tad "optimistic" about what the results actually mean in terms of "cost-effectiveness." One might even hypothesize that the conclusions are something less than totally objective.
I think the "cost-effective" conclusion needs a more nuanced and comprehensive evaluation. Besremi needs to be compared to all treatment options in current use. This would need to include comparing Besremi to Pegasys in particular.
I am certainly very pleased with how I have responded to Besremi. This is my own experience-based self-study with a N (sample size) = 1.
Besremi vs aspirin-only - Besremi much more effective.
Besremi vs HU - Besremi more effective and much easier to tolerate. Quality of Life much better.
Besremi vs venesection - Besremi more effective and easier to tolerate. Quality of life better.
Besremi vs Pegasys - based on 8 months PEG - 19 months Besremi - no apparent difference in efficacy. Improved Quality of Life equivalent. Adverse effects similar but Besremi is at a relatively higher dose (PEG=45mcg, BES=150mcg). Tolerance comparison is inconclusive due to inadequate data.
Understanding the financial imperatives that drive research and how new drugs are developed is essential in interpreting data and how it is presented. "The expected cost to develop a new drug—including capital costs and expenditures on drugs that fail to reach the market—has been estimated to range from less than $1 billion to more than $2 billion." Congressional Budget Office cbo.gov/publication/57126#:.... It is quite common for any new drug to cost in excess of $1 billion dollars to develop and get approved. This makes new drugs for rare diseases particularly expensive to develop, The average I have heard quoted for rare disease drug development = $1.3 billion.
So, I can understand where a drug company might want to present an expensive drug in the most optimistic light. They have a lot invested in it. It is up to us to be discriminating consumers when we review how data is presented.
What is interesting is that some insurors have started to authorize Besremi while refusing to authorize Pegasys. FDA label does matter. Besremi is making it onto the foruimlaries, albeit at a very high tier. NCCN guidelines matter. Conversely, all insurance companies seem willing to authorize HU, even though it is off-label for PV. Cost also matters.
Thank you for the opportunity to have an informed discussion about an issue that affects all of us with MPNs. Wishing you all the best on your MPN journey.
Somehow come across this article. While in principle I do agree with Manouche's point that articles with the manufacturer's involvement are not necessarily biased, I would be more conservative in this particular case. Especially after seeing how this company runs its business and promoting their product. I remember a while ago there was someone on this forum showing how the manufacturer manipulating FDA data to demonstrate that it product has fewer or less severe side effects.
This is, of course, not to say the drug is not safe or not effective. I am just concerned about the way the manufacturer promotes the products.
This is a super expensive interferon with no added benefit. In Europe, this thing is rarely prescribed because it is denied by insurance in several countries. Reportedly, the yearly cost for Besremi is almost 200K, whereas Pegasys is only around 50K annually. What justifies for its high cost? None. Can't do with data manipulation, the manufacturer has not proven it to be any better than exisiting therapy.
To Ggrana3192
Sorry it didn't work out for you. It didn't for me either, I got a permanent autoimmune and neuro condition from it after getting a flu vaccine. I can never try IFN again.
But till that it worked great. It's shame your Dr put you on that dose, I was on 140 and it was already too high. Did your Hb get that low all at once or did it decrease as your dose went up? If the latter Dr should have controlled you dose more carefully. What was your PLT reading?
Is the hemolytic anemia an irreversible condition?
I'm now on Rux and it's working fine. It is great for calming the immune system.
Interesting study though the statistical analysis is a bit complex. The bottom line is that Besremi results in longer and better quality life for (some) people with PV so it is worth the money.
Besremi interferon = 3 1/2 times more expensive than 18 year old Pegasys interferon, but usually not clearly superior in regards to either efficacy or side effects. Clearly inferior in regards to affordability.
Ropeginterferon alfa-2b versus phlebotomy in low-risk patients with polycythaemia vera (Low-PV study) PharmaEssentia Initiates Pivotal Trial of Ropeginterferon alfa-2b to Treat Essential Thrombocythemia
Ropeg versus HU/BAT results after 3 yearsEvent-free survival in patients with polycythemia vera treated with Besremi versus best available treatmentBesremi Progress in US
