I'm pretty triggered by anyone calling themselves a health professional in the other thread and saying that they haven't seen enough to say that the therapy works. They are either lying, misinformed, unqualified or all of the above.
As I have said before, I am a translational scientist/business analyst with a background as a physiologist.
The standard of care (SoC) for CHF is furosemide (or equivalent) or a heart transplant.
Furosemide is treating the symptoms of CHF by making a patient pee a lot to get rid of more fluid than they would normally to get rid of some of the load on the failing heart. This requires escalated dosing as the heart failure progresses/tolerance is built. This method has a significantly high associated morbidity and it does nothing for the breathlessness of the patient while they perform minimal exertion (like getting out of bed and walking a few metres) and they have to pee every half hour depending on the severity of their disease and the corresponding dosage. This presents its own problem as the patient losses a significant amount of salts that they need to stay on top of to prevent other significant issues. My own grandmother got to this stage and very seriously asked me to up her dosage of morphine to a stage where she would not wake up. I, of course, did not increase my grandmother's dosage - but the morbidity associated with this standard of care is crippling and a massive toll on the individual and their families.
A heart transplant has obvious shortcomings. You need a donor, you need to not reject the graft, and even then, a graft is usually only expected to last around 10 years. It also involves open-heart surgery with a significant theatre time (costs hospitals and surgeons money).
Facts About Heart Failure in the United States:
- About 6.2 million adults in the United States have heart failure.1 - In 2018, heart failure was mentioned on 379,800 death certificates (13.4%).1
- Heart failure costs the nation an estimated $30.7 billion in 2012.2 This total includes the cost of health care services, medicines to treat heart failure, and missed days of work.
Mesoblast is a minimum of 7 years ahead of all competitors for cellular medicines targeting heart failure.
Mesoblast has an extensive patent portfolio, that will require significant litigation even when someone catches up to them. The barrier for Mesoblast for MSB is not whether the product works or not. That is known as the results of a gold standard RCT in the US showed a statistically significant reduction in deaths by 60% in patients with class II heart failure. Estimations based on study recruitment rates is that Class II heart failure will be 30-60% of the total number of heart failure cases. Lets low ball this and say 30% (which is, as I say, low balling). 30% of 6.2 million is 1,860,000 people who could be benefiting and receiving Rexlemestrocel. This doesn't even take into consideration Class I patients who will also be inflammatory and class III patients who, while did not show a reduction in mortality, seemed to show an improvement in quality of life.
The barrier for Mesoblast is purely regulatory. And that is only a delay - will it be this year or 2 years time (reduced sample size requirements in a targeted study population).
If I go to Mr/Ms big pharma and say "Hey we got screwed by an unpredictable mechanism of the disease because we didn't show a reduction in 'decompensated non-fatal recurrent heart failure associated events' but we have a significant reduction in death in this population, a prevention of disease progression, and a reduction in heart attack, stroke, and death."
Do you REALLY THINK that big pharma - OR ANYONE ELSE!?!?! Is going to be like "Oh but that 'decompensated non-fatal recurrent heart failure associated events' p value tho"
Worst case scenario is that they pay for another Phase III, wait 2-5 (conservatively) years, and have a multi-billion dollar therapy.
And don't even get me started on CLBP I swear.
SMH
Gang Gang
(20min delay)