Clinical Excellence or Death Sentence

American business media outlets are publishing stories which decry the recent decision by the British National Institute for Clinical Excellence (NICE) to not recommend paying for Yervy (ipilimumab), a new pharmaceutical treatment for inoperable melanoma.  Here is an excerpt from Bloomberg Businessweek (find it here):

Bloomberg Businessweek
October 14, 2011
UK medical group rejects new skin cancer treatment
By Maria Cheng

An independent British medical watchdog says the first treatment proven to help people with the deadliest form of skin cancer is too expensive to be used by the U.K.'s health care system, a recommendation critics called a potential death sentence.

The drug, Bristol-Meyers Squibb's Yervoy, has offered some hope to people with advanced skin cancers, though a recent study showed it only worked in a small segment of patients studied, and they lived just four months longer than patients given older medications.

The National Institute for Clinical Excellence, or NICE, advised Friday that at a cost of 80,000 pounds ($126,600) Yervoy "could not be considered a cost-effective use" of health funds. A final decision is expected next month after a public consultation.

In the U.K., most medicines are paid for by the government, as long as they're recommended by the cost-efficiency watchdog. The agency commonly rejects expensive drugs, including recently advising against new treatments for prostate cancer, breast cancer, and multiple sclerosis, though patients and doctors are increasingly protesting the decisions.

The government usually adopts NICE's recommendations, meaning doctors in the government-funded health service cannot prescribe Yervoy without NICE's approval.

In its decision, NICE said it was not convinced by the evidence, saying the data for Yervoy, which works by stimulating the immune system to fight cancer, did not compare it to older drugs used to treat melanoma. NICE also said the trial was too short to know how long the drug's effects would last and raised concerns about the drug's side effects, including diarrhea, rash, fatigue and nausea, which they said could affect a patient's quality of life.

"We need to be sure that new treatments provide sufficient benefits to justify the significant cost (the health care system) is being asked to pay," said Sir Andrew Dillon, NICE's chief executive, in a statement.

Patient groups and charities slammed the decision, labeling it a "death sentence" for people with advanced skin cancer.

AND. . .

here is an excerpt from the Wall Street Journal (find the article here):

The Wall Street Journal
October 14, 2011
U.K. Agency Rejects Bristol-Myers Skin Cancer Drug
By Jonathan D. Rockoff and Sten Stovall

Yervoy is expected to be a blockbuster for Bristol, with more than $1 billion in yearly sales. The therapy was approved in the U.S. in March and in Europe in July. It had $95 million in sales during the second quarter. Bristol CEO Lamberto Andreotti recently said the company was "very happy with the results so far" from the drug's uptake.

"The price of Yervoy reflects the value of Yervoy," Mr. Andreotti added, at the Pharmaceutical Strategic Alliances conference in New York last month.

AND. . .

here is a key conclusion from NICE (find the guidance document here):

National Institute for Health and Clinical Excellence (NICE)
October 12, 2011
Melanoma (stage III or IV) - ipilimumab: appraisal consultation document

Key conclusion

Ipilimumab is not recommended for the treatment of advanced (unresectable or metastatic) malignant melanoma in people who have received prior therapy.

The Committee was satisfied that ipilimumab meets the criteria for being a life-extending, end-of-life treatment and that the trial evidence presented for this consideration was robust.

The Committee acknowledged that few advances had been made in the treatment of advanced melanoma in recent years and ipilimumab could be considered a significant innovation for a disease with a high unmet clinical need.

Despite the combined value of these factors the Committee considered that the magnitude of additional weight that would need to be assigned to the QALY gains (quality-adjusted life years) for people with advanced (unresectable or metastatic) melanoma would be too great for ipilimumab to be considered a cost-effective use of NHS resources.

Dr. Don McCanne's question:

Should a $126,000 drug (Yervoy, ipilimumab) that produced only a very minimal benefit in a small segment of patients studied, yet caused significant side affects, be included in program that we finance? Is there no limit as to what we should add to coverage when our national health expenditures are already challenging individual, business and government budgets?

My comment:

Yervoy comes at an exceptionally high price, which Bristol-Myers believes wil be paid because people threatened by melanoma will find a way to make the payment, or make a third party make the payment.  Bristol-Myers is expecting to make an enormous profit from this drug.  NICE, however, has made the judgment that the price is too high for the taxpayers of Great Britain. 

The truth is that taxpayers in the US pay for the majority of health care costs.  And health care costs are pushing the federal government into massive budget deficits.  Surely, any fair judgment about this situation requires the conclusion that the taxpayers can not afford to pay whatever price suits the private sector's pursuit of the highest profits possible.  Some clinical interventions are simply not worth the price.  And the nonsense rhetoric about these judgments being a 'death sentence' needs to stop. 

How to make the judgments about what clinical interventions are worth the price is really the question.  The Utah Healthcare Initiative, noting that the State of Washington already has a Commission process effectively weighing in on clinical effectiveness, proposes that Utah form a health system commission where the decisions about what is covered will be made.  Further, we propose that clinical interventions not included in the Utah health benefit be made available for sale but without the financial support of the taxpayer or the premium payer.  In other words, those who wish to purchase Yervoy (assuming it was not included in the health benefit package) could do so using their own money, or other private sources of funding.

We do not have unlimited funds to spend on health care.  A limit must be set.  Let's all grow up and discuss this sensibly.

Dr. Joe Jarvis