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True Breakthroughs

But when will the CREDENCE results on Invokana be published?

You may remember that the CREDENCE study was stopped early July 16, 2018 --the “Phase 3 CREDENCE (Canagliflozin and Renal Events in Diabetes with Established Nephropathy Clinical Evaluation) clinical trial, evaluating the efficacy and safety of INVOKANA® (canagliflozin) versus placebo when used in addition to standard of care for patients with chronic kidney disease (CKD) and type 2 diabetes (T2D), is being stopped early based on the achievement of pre-specified efficacy criteria.”

OK then, but WHEN will we know the results (usually released at a major conference).

Would you like to know the answer?

April 15, 2019 at the World Congress of Nephrology in Melbourne, Austrailia.

We of course, will update our current Essential Guide to Prescription Drugs, Update on canagliflozin, CREDENCE for Invokana eBook at that time. The beauty of breaking science is that it will highlight the 4,401 patients who were in the study AND bring the benefits of those patients to a broader patient population while helping prevent problems in those living with diabetes.

Remember, as the lead CREDENCE researcher said: "Nearly half of all people with type 2 diabetes will develop chronic kidney disease, causing a high risk of kidney failure and cardiovascular disease, and impacting their quality and length of life, even with the current best available care. This huge unmet need is why it was so important for us to initiate the landmark CREDENCE renal outcomes trial over four years ago," said Vlado Perkovic, M.B.B.S, Ph.D., F.A.S.N., F.R.A.C.P., CREDENCE Steering Committee co-chair, Professor of Medicine, University of New South Wales Sydney, and Executive Director, The George Institute for Global Health Australia. "We have accepted the advice of the Independent Data Monitoring Committee to stop the CREDENCE trial early due to demonstration of efficacy, and look forward to sharing the findings as soon as possible."

See the True Breakthrough criteria below

True breakthrough Criteria: What criteria will we use?

  1. The medicine is the first in a new medicine family (class)…what the FDA would call a novel therapeutic entity (visit A good example here would be evolocumab (Rapatha), the first member of the PCSK9 inhibitor family which was approved.
  2. The medicine is 20% or more effective in treating or preventing a prevalent disease or condition than prior medicines. For example, the new prescription fish oil (Vascepa) which was found to prevent heart attacks by 25% in people who were already being treated with statins.
  3. The therapeutic concept targets a novel path or uses a novel approach to modify or prevent or cure disease. An example here would be signal transduction inhibitors such as Gleevec (actually turns off a gene to cure stomach cancer) or immune based therapies such as ipilimumab (Yervoy) for melanoma. CRISPER technology seeking gene-based cures.
  4. New guidelines which radically change current practice, incorporating new approaches to monitoring therapy or preventing disease. A good example here are the 2018 American Diabetes Association standards of care.
  5. A company making a medicine develops a program or radical pricing which confers new access to care or enhances outcomes (results) by 15% versus other approaches or therapies. The latest example here is the company Amgen and the medicine Rapatha. The company decided to lower the cost of the medicine by 60%, which is expected to give huge increases in access to a very effective cholesterol medicine.
  6. A new medicine results in CURES of a particular disease or condition where none was previously available. A great example here are the hepatitis C treatments (Harvoni, others) which can result in CURE of the hepatitis C.
  7. A laboratory, clinic or point of care test that improves the ability to diagnose or predict a disease or condition which either could not be previously reliably predicted.
  8. A medicine, vaccine or mechanical device that PREVENTS a disease or condition to a significantly greater degree than previously possible or which addresses a disease or condition previously perceived as a natural course of aging or which was not able to be prevented. A great example here is the new Shingles vaccine.

Real medicine, real research, really important. The Essential Guide to Prescription Drugs is always there for you!


  1. FDA
  2. NIH Updated September 24, 2017. Accessed September 10, 2018.
  3. The Use of Essential Drugs. World Health Organization,
  4.  Accessed 10 31 2018.  
  5. Conference materials accessed 1/12/19: World Congress of Nephrology.


Have you heard about the REDUCE-IT study?

For those attending the American Heart Association meeting in Chicago By James Joseph Rybacki, Pharm.D. 1/8/19

On November 10-14 2018, you could have heard a pin drop in the large conference room when Dr. Deepak Bhatt presented the results. The results showed a relative risk reduction of 25% in Major Cardiac Adverse Events (MACE). This study looked at more than 8,000 people who were already taking statins, had heart disease and may also have been diabetic.

It’s not my first rodeo and even I was impressed by these results. Interestingly, many of you know I have a history of heart disease and suffered an early heart attack in 2005. Despite this, and despite the new REDUCE-IT data, my insurance carrier REFUSED to pay for Vascepa, although my doctor sent in two requests saying that the medicine was warranted.

The problem stems from the product labeling (although huge numbers of prescriptions are written off-label in the US). Who will pay for the drug? For now, me. I have been absorbing the two hundred dollar expense because I think that the data are simply that good. Will the label for Vascepa change? This remains a decision for the FDA. In the interim, I think US insurance companies who attempt to deny heart patients this valuable medicine are short sighted and are WRONG. We have a new video on Vascepa on

What on earth is the controversy over PCSK9 medicines for cholesterol.??

By James Joseph Rybacki, Pharm.D. 1/8/19

Hmmmm, it comes down to money, or it used to, or, it still does. Let’s take a closer look. The challenge for the two novel cholesterol medicines in the PCSK9 family (evolocumab Repatha and Alirocumab or Praluent) has NOT been lack of use because they don’t work, it has been a failure of insurance companies to pay the roughly $12,000 dollars per year that they cost. Let me repeat that. COST, NOT results have been the key reason that these extremely effective and safe medicines have not been widely used.

A study released at the Heart Association meeting in Chicago Nov 11, 2018, showed that the ODYSSEY Outcomes trial found Praulent is cost-effective when the price is $6,319 a year when the willingness-to-pay threshold is at a usually accepted $100,000 per QALY (Quality Adjusted Life-year). The author, Deepak Bhatt from the Brigham and Women’s hospital expressed his hope that this analysis might provide evidence for what the price should be, versus market pricing that uses expense that might be tolerated.

Interestingly, the Repatha manufacturer (Amgen) lowered the price of Repatha to $5,850 per year last October. It will be interesting to see how Repatha sales are impacted by this novel pricing.

Did you think that Twitter would become mainstream medicine?

By James Joseph Rybacki, Pharm.D. 1/8/19

I wasn’t so sure when I decided to call myself Rybacki knows. Sounded pompous at first, then had a certain ring to it. Thousands of tweets later, I think of it in the same light at LinkedIn.

I remember reading “Medicine in the age of Twitter in the NY Times in 2009. At that time Pauline Chen, MD was reflective and it was noted that more than 60% of Americans went on line for medical information. Yikes.

If we jump to 2017, we find Vinay Prasad challenging his medical colleagues via Twitter and doing a righteous job of it as well. I’ve found myself roundly criticized at times, but then, if you are saying something worthwhile, this is bound to happen from time to time. Is Twitter the appropriate place for patient discussions or consultations, definitely no, but Twitter does have a place in mainstream medicine IF you manage by data and accurately quote the facts.

By James Joseph Rybacki, Pharm.D. 1/8/19

Even when patients are treated to blood pressure and cholesterol goals, they still have heart attacks and some of them die. This sobering fact has led to an emerging residual cardiovascular risk, residual inflammatory burden and a number of other explanations.

The CANTOS or the Canakinumab Anti-Inflammatory Thrombosis Outcomes Study took a look at the idea that inflammation had a role in cardiac events. The results did indeed show that 150 mg of canakinumab worked better than a sugar pill at preventing bad (adverse) heart (cardiac) events.

You may not have heard about canakinumab unless you’ve been ready nerdy research or have recently attended a heart meeting. This experimental medicine is a MAB (monoclonal antibody) that works against an inflammatory molecule called interleukin-Beta. The goal of CANTOS was to see how placebo stacked up against canakinumab in people with a history of heart attack (MI) and an elevated high sensitivity C-reactive protein levels (HsCRP).

It appears that the evidence is growing (swelling) for some kind of role of inflammation, but the exact cause of the swelling and the ideal way to lower the swelling and heart attack risk have yet to be clearly decided.

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