Novartis Pharmaceuticals Health Policy Monthly Update : October

Presidential Candidates Release Proposals to Impact Drug Prices

Democratic candidates for President Hillary Clinton and Sen. Bernie Sanders introduced separate proposals that would impact pharmaceutical pricing and potentially inhibit innovation. The proposals include providing Medicaid-level rebates in Medicare Part D; allowing importation of drugs from other countries; reducing the exclusivity period for biologics; requiring negotiation with the federal government for Part D rebates; preventing certain patent settlements between innovator and generics companies; requiring pharmaceutical companies to invest a specific percentage of revenue in R&D; removing tax deductions for direct-to-consumer advertising; and pushing drug companies to price based on the value of treatments assessed via comparative effective analysis.
 
House Speaker Resigns, Government Shutdown Averted

John Boehner, Speaker of the House of Representatives, announced that he will resign from Congress, effective October 31. With his resignation, Speaker Boehner was able to put forth a bill to avert a partial government shutdown that would have begun October 1, 2015 if Congress had not passed legislation to provide funding to keep the government functioning. Previously, Speaker Boehner had faced threats to his leadership position if he put forward the bill, which included funding for Planned Parenthood. On September 30, legislation providing funding for the government until December 11 was passed by both the House and Senate and was signed by President Obama.
 
NCQA Releases Health Plan Ratings

The National Committee for Quality Assurance (NCQA) released a new health plan rating system, using a 1.0 to 5.0 scale with 5.0 indicating higher performance. This new rating system replaces their previous health plan ranking system. Evaluating more than 1,000 plans, including commercial, Medicaid, and Medicare Part D plans, the new rating system assesses three major performance categories, consumer satisfaction, prevention, and treatment and it provides a simple way for consumers to gauge the quality of care being provided by a health plan. The new plan rating system shows that Maine, Massachusetts, New Hampshire, New York, Pennsylvania, Rhode Island, Vermont, Michigan, Minnesota, and Wisconsin had the highest percentage of plans receiving a 4.5 or 5.0 rating.

 
CMS Announces Medicare Advantage Value-Based Insurance Design

On September 1, 2015, the Centers for Medicare & Medicaid Services (CMS) announced the Medicare Advantage Value-Based Insurance Design (VBID) Model, which will test the hypothesis that giving Medicare Advantage plans flexibility to offer targeted extra supplemental benefits or reduced cost sharing to enrollees who have specified chronic conditions can lead to higher quality and more cost-efficient care. The VBID Model will begin January 1, 2017 and run for five years. CMS will test the model in seven states: Arizona, Indiana, Iowa, Massachusetts, Oregon, Pennsylvania, and Tennessee. Upon approval from CMS, eligible Medicare Advantage plans in these states can offer varied plan benefit design for enrollees who fall into the following clinical categories: diabetes, congestive heart failure, chronic obstructive pulmonary disease (COPD), past stroke, hypertension, coronary artery disease, and mood disorders.
 
Medicare Advantage and Part D Markets Largely Stable from 2015-2016

On September 21, 2015, CMS announced that premiums for Medicare Advantage (MA) plans will remain stable in 2016. CMS estimates that the average MA premium will decrease by $0.31 next year, from $32.91 on average in 2015 to $32.60 in 2016. Enrollment in MA is projected to increase to approximately 17.4 million enrollees, which represents about 32 percent of the Medicare population. Earlier this year, CMS announced that the average basic Medicare prescription drug plan premium in 2016 is projected to remain stable at $32.50 per month. The Annual Election Period for Medicare health and drug plans begins on October 15, 2015 and ends December 7, 2015.
 
CMS Announces the Enhanced Medication Therapy Management Model

On September 28, 2015, the Center for Medicare and Medicaid Innovation (CMMI) announced a five-year model to test approaches to improve Medicare Part D beneficiary medication use. The Part D Enhanced Medication Therapy Management (MTM) Model will test whether changes to the Part D program can help to better align the interests of plan sponsors with those of the federal government. Eligible basic stand-alone prescription drug plans (PDPs), upon approval from CMS, can vary the intensity and types of MTM items and services based on beneficiary risk level and seek out a range of strategies to individualize beneficiary and prescriber outreach and engagement. An initial five-year performance period will begin January 1, 2017 in five Part D regions spanning 11 states: Region 7 (Virginia), Region 11 (Florida), Region 21 (Louisiana), Region 25 (Iowa, Minnesota, Montana, Nebraska, North Dakota, South Dakota, Wyoming), and Region 28 (Arizona).

 
HHS Releases Latest Exchange Enrollment Numbers

The U.S. Department of Health and Human Services (HHS) has released their latest enrollment numbers for both the federal and state exchanges. As is to be expected, actual enrollment is down slightly from the March report. Almost 9.9 million people had paid their first month’s premium as of June 30, slightly above the projected enrollment of 9.1 million people. Of those paying their premiums, 84%, or 8.3 million, received premium tax credits and 5.5 million people also received cost sharing subsidies. Premium tax credits averaged $270 a month. Approximately 423,000 people had their 2015 coverage terminated for failure to provide the necessary documentation of citizenship or legal immigrant status. More than 6.7 million people enrolled in a silver tiered plan, 2.1 million enrolled in bronze or catastrophic, almost 700,000 selected a gold tier and 332,000 picked platinum. In early September, CMS reported that Medicaid and CHIP enrollment had reached 72 million. The increase in both public and private insurance programs has dropped the national uninsured rate to below 10%.

 
HHS Issues Proposed Rule Regarding ACA’s Non-discrimination Provisions

HHS issued a draft rule providing clarity to the Affordable Care Act’s prohibition on discrimination in insurance coverage on the basis of race, color, age, national origin, sex, and disability. The proposed rule covers consumers’ rights under the ACA, obligations of covered entities, the inclusion of gender identify discrimination as a form of sex discrimination, requirements for effective communication to those with disabilities, and language assistance for those with limited English proficiency. Discriminatory practices in benefit plan design, marketing and cost sharing are prohibited. The proposed regulations apply to health insurers participating in the federal and state-based exchanges, Medicare Advantage and Medicaid programs. These protections are extended to all individual and group products sold by a participating insurer. As drafted, the proposed regulations are a step forward in eliminating discriminatory practices that prevent patients with chronic conditions from accessing necessary medication but opportunities for more specific language in the final regulations remain.

FDA Puts Strict Limits on Oral Ketoconazole Use

By John Gever, Deputy Managing Editor, MedPage Today

SILVER SPRING, Md. — Oral ketoconazole (Nizoral) should never be used as first-line therapy for any type of fungal infection because of the risk of liver toxicity and interactions with other drugs, the FDA said Friday.

The agency ordered a series of label changes and a new medication guide for patients that emphasize the risks, which also include adrenal insufficiency. It noted that the restrictions apply only to the oral formulation, not topical versions.

Late Thursday, the chief advisory body for the FDA’s European counterpart went further. The EU’s Committee on Medicinal Products for Human Use (CHMP) recommended that member nations pull oral ketoconazole from their markets entirely.

Both the FDA and the CHMP cited studies indicating high risks of severe, acute liver injury in patients taking the drug. Studies using the FDA’s adverse event reporting system and a similar database in the U.K. indicated that liver toxicity was more common with oral ketoconazole than with other anti-fungals in the azole class.

The FDA also said that oral ketoconazole “is one of the most potent inhibitors” of the CYP3A4 enzyme. This effect can lead to sometimes life-threatening interactions with other drugs metabolized by CYP3A4, and also to adrenal insufficiency, since the enzyme also catalyzes release of adrenal steroid hormones.

“This accounts for clinically important endocrinologic abnormalities observed in some patients (particularly when the drug is administered at high dosages), including gynecomastia in men and menstrual irregularities in women,” the FDA said.

The only indication for oral ketoconazole still supported by the FDA is for use in life-threatening mycoses in patients who cannot tolerate other anti-fungal medications or when such medications are unavailable.

In such instances, the FDA said, physicians should assess liver function before starting the drug. It is contraindicated in patients with pre-existing liver disease, and patients should be instructed not to drink alcohol or use other potentially hepatotoxic drugs.

Adrenal function should also be monitored in patients using the drug.

The CHMP also indicated the topical formulations of ketoconazole should stay on the market, but it found no basis for keeping the oral form available for any purpose.

“Taking into account the increased rate of liver injury and the availability of alternative anti-fungal treatments, the CHMP concluded that the benefits did not outweigh the risks,” the panel indicated in a statement.

It recommended that physicians stop prescribing oral ketoconazole and that they should review alternatives in patients currently receiving the drug. The committee also said that patients now taking oral ketoconazole “make a non-urgent appointment” with their physicians to discuss their treatment.

From MedPage Today

Notes on the Magic Foundation Cushing’s Conference, 2012

Here are links to all the posts in order, although some still need to be edited.  I posted these directly from the meeting hall with no rereads or rewrites.  If anyone has anything to add, edit or delete, please let me know!

 

  • Magic Conference: Understanding your Pituitary Gland in Health and Disease

Dr. Frohman will present an overview of the pituitary gland. He will cover general aspects of pituitary function and testing and also review the types of pituitary disease that occur, including pituitary tumors and Sheehan’s Syndrome. Many people ask and wonder if Growth Hormone Deficiency can be inherited. Dr. Frohman will also briefly address that concern.

 

http://cushingshelp.blogspot.com/2012/07/magic-conference-understanding-your.html

 

  • Magic Conference: Testing and Diagnosis Process for Pituitary Disorders

Pituitary disorders can be difficult to diagnose in many cases. Dr. Salvatori will discuss the different testing and diagnostic procedures to determine the pituitary disorder. MAGIC receives many calls asking about diagnostic procedures. This segment will be helpful in understanding what procedures are used today to provide the best treatment available.

http://cushingshelp.blogspot.com/2012/07/magic-conference-testing-and-diagnosis.html

 

  • Magic Conference: Managing Medications and Aftercare of Treatments

It is crucial to monitor your treatments and aftercare of treatments when living with a pituitary disorder. Dr. Salvatori will discuss the importance of these issues so you will be aware of how to manage your pituitary disorder. A simple diagnosis does not mean that medications may be altered or changed in the future. This segment will assist you with information on how to manage your future.

http://cushingshelp.blogspot.com/2012/07/magic-conference-managing-medications.html

 

  • Magic Conference: Cushing’s Disease, Are We Closer to Medical Therapies?

A significant proportion of patients with Cushing’s Disease are not cured by primary surgical treatment, the disease is prone to relapse and significantly damages quality of life. Adjuvant radiotherapy is an increasingly unattractive option for clinicians who wish to spare their patients hypopituitarism and other potential complications. Some pharmacological options are currently available but tend to have dose-limiting side effects. New agents recently approved or under investigation will be discussed and strategies to select the optimal drug or drug combination for individual patients reviewed.

http://cushingshelp.blogspot.com/2012/07/magic-conference-cushings-disease-are.html

 

  • The Trip So Far…

MaryO’s personal experiences and thoughts

http://cushingshelp.blogspot.com/2012/07/the-trip-so-far.html

 

  • Cushing’s Help Turned Twelve During the Conference!

Twelve  years ago I was talking with my dear friend Alice, who runs a wonderful menopause site, Power Surge, wondering why there weren’t many support groups online (OR off!) for Cushing’s and I wondered if I could start one myself.  We decided that I could.

This website (http://www.cushings-help.com) first went “live” July 21, 2000 and the message boards September 30, 2000. Hopefully, with this site, I’ve made  some helpful differences in someone else’s life.

http://cushingshelp.blogspot.com/2012/07/today-we-twelve.html

 

  • Magic Conference: Treating Cushing’s Disease with Surgery: Ways of Achieving a Cure

Dr. McCutcheon will discuss the ways of treating Cushing’s disease from the surgical perspective, including the different ways of getting to the pituitary, the chances of success, and the potential complications. In addition, special nuances and pitfalls ofsurgery in Cushing’s (as opposed to other types of pituitary tumor) will be addressed.

http://cushingshelp.blogspot.com/2012/07/magic-conference-treating-cushings.html

 

  • Phil and the Zebra Undies

Dr. McCutcheon is a surprise participant in a gift to Phil.

http://cushingshelp.blogspot.com/2012/07/phil-and-zebra-undies.html

 

  • Real Talk: Psychological Process of Illness, part 1

This Segment will be broken into two sections. Part I will be provide an open opportunity for participants to ask relevant questions around the emotional/mental issues in living with a chronic illness. Participants will be able to openly talk about depression, anxiety, trauma, and other processes that occur when living illness.

 

Due to confidentiality issues, most of this segment is not shared.

http://cushingshelp.blogspot.com/2012/07/real-talk-psychological-process-of.html

 

  • Real Talk: Psychological Process of Illness, part 2

Part II will focus on seeing ourselves as survivors of illness and the process of staying empowered through illness that impacts us in such a powerful way. Principles of empowerment and how one can turn adversity into opportunity will be discussed. This segment should create an environment that is non judgmental and motivating.

 

Due to confidentiality issues, most of this segment is not shared.

http://cushingshelp.blogspot.com/2012/07/real-talk-psychological-process-of_21.html

 

  • The Rest Of The Trip

Wind-up and heading home

http://cushingshelp.blogspot.com/2012/07/the-rest-of-trip.html

Eventually, all these posts will be edited, cleaned up and typos corrected.;

Korlym™ (mifepristone), the First Approved Medication for Patients with Endogenous Cushing’s Syndrome, to be Available by April 11

Corcept Therapeutics (NASDAQ:CORT) announced that it would be ready to ship Korlym to patients by April 11th, three weeks ahead of the company’s previously announced launch date. “Cushing’s syndrome is a life altering and life threatening disease,” said Joseph K. Belanoff, M.D., the company’s Chief Executive Officer. “We have worked hard to bring this first-in-class treatment to patients as quickly as possible.”

On February 17, 2012, the U.S. Food and Drug Administration (FDA) approved KorlymTM (mifepristone) 300 mg Tablets as a once-daily oral medicine to control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. Physicians and patients seeking more information can visit http://www.korlym.com.

Korlym is distributed in 300 milligram tablets to be taken once each day. The wholesale acquisition price of Korlym is $0.62 per milligram. The FDA-approved labeling instructs physicians to titrate each patient’s Korlym dose to clinical efficacy by assessing tolerability and degree of improvement in Cushing’s syndrome manifestations. In the first six weeks, these manifestations may include changes in glucose control, anti-diabetic medication requirements, insulin levels and psychiatric symptoms. After two months, assessment may also be based on improvements in cushingoid appearance, acne, hirsutism, striae or decreased body weight, along with further changes in glucose control.

Patient Assistance Programs 
“Our highest priority is that every patient who is prescribed Korlym will receive it,” said Dr. Belanoff. To that end, the company has launched a comprehensive financial assistance and patient support program. A dedicated team of Corcept case managers will help patients understand their insurance benefits and the financial and medical support programs available to them.

“Patients face tremendous challenges managing their illness – from finding physicians familiar with the disease to navigating the complexities of insurance reimbursement to paying for the cost of care,” said Dr. Belanoff. “We are determined that none of these barriers will keep patients from receiving the benefits of Korlym.”

About Cushing’s Syndrome 
Endogenous Cushing’s syndrome is a rare and life-threatening endocrine disorder that results from long-term exposure to excess levels of the hormone cortisol. This excess is caused by tumors that usually occur in the pituitary or adrenal glands that over-produce, or prompt the over-production of, cortisol.

Although cortisol at normal levels is essential to health, in excess it causes a variety of problems, including hyperglycemia, upper body obesity, a rounded face, stretch marks on the skin, an accumulation of fat on the back, thin and easily bruised skin, muscle weakness, bone weakness, persistent infections, high blood pressure, fatigue, irritability, anxiety, psychosis and depression. Women may have menstrual irregularities and facial hair growth, while men may have decreased fertility or erectile dysfunction. More than 70 percent of Cushing’s syndrome patients suffer from glucose intolerance or diabetes.

The treatment of an endogenous Cushing’s syndrome patient depends on the cause. The first-line approach is surgery to remove the tumor. If surgery is not successful or is not an option, radiation may be used, but that therapy can take up to ten years to achieve full effect. Surgery and radiation are successful in only approximately one-half of all cases.

If left untreated, Cushing’s syndrome has a five-year mortality rate of 50 percent.

An orphan disease, Cushing’s syndrome occurs in about 20,000 people in the United States, mostly women between the ages of 20 and 50.

About Korlym™ (mifepristone) 300 mg Tablets
Korlym is a once-daily oral medication that blocks the glucocorticoid receptor type II (GR-II) to which cortisol normally binds. By blocking this receptor, Korlym inhibits the effects of excess cortisol in Cushing’s syndrome patients.

The FDA has designated Korlym as an Orphan Drug, a special status designed to encourage the development of medicines for rare diseases and conditions. Because Korlym is an Orphan Drug, Corcept will have marketing exclusivity for the FDA-approved indication until February 2019.

IMPORTANT SAFETY INFORMATION


WARNING: TERMINATION OF PREGNANCY

 

See full prescribing information for complete boxed warning.

 

Mifepristone has potent antiprogestational effects and will result in the termination of pregnancy. Pregnancy must therefore be excluded before the initiation of treatment with Korlym, or if treatment is interrupted for more than 14 days in females of reproductive potential.

 

Contraindications

  • Pregnancy
  • Use of simvastatin or lovastatin and CYP 3A substrates with narrow therapeutic range
  • Concurrent long-term corticosteroid use
  • Women with history of unexplained vaginal bleeding
  • Women with endometrial hyperplasia with atypia or endometrial carcinoma

Warnings and Precautions

  • Adrenal insufficiency: Patients should be closely monitored for signs and symptoms of adrenal insufficiency.
  • Hypokalemia: Hypokalemia should be corrected prior to treatment and monitored for during treatment.
  • Vaginal bleeding and endometrial changes: Women may experience endometrial thickening or unexpected vaginal bleeding. Use with caution if patient also has a hemorrhagic disorder or is on anti-coagulant therapy.
  • QT interval prolongation: Avoid use with QT interval-prolonging drugs, or in patients with potassium channel variants resulting in a long QT interval.
  • Use of Strong CYP3A Inhibitors: Concomitant use can increase plasma levels significantly. Use only when necessary and limit dose to 300 mg.

Adverse Reactions

Most common adverse reactions in Cushing’s syndrome (≥ 20%): nausea, fatigue, headache, decreased blood potassium, arthralgia, vomiting, peripheral edema, hypertension, dizziness, decreased appetite, endometrial hypertrophy.

To report suspected adverse reactions, contact Corcept Therapeutics at 1-855-844-3270 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Drug Interactions

  • Drugs metabolized by CYP3A: Administer drugs that are metabolized by CYP3A at the lowest dose when used with Korlym
  • CYP3A inhibitors: Caution should be used when Korlym is used with strong CYP3A inhibitors. Limit mifepristone dose to 300 mg per day when used with strong CYP3A inhibitors.
  • CYP3A inducers: Do not use Korlym with CYP3A inducers.
  • Drugs metabolized by CYP2C8/2C9: Use the lowest dose of CYP2C8/2C9 substrates when used with Korlym.
  • Drugs metabolized by CYP2B6: Use of Korlym should be done with caution with bupropion and efavirenz.
  • Hormonal contraceptives: Do not use with Korlym.

Use in Specific Populations

  • Nursing mothers: Discontinue drug or discontinue nursing.

Please see the accompanying full Prescribing Information including boxed warning atwww.corcept.com/prescribinginfo.pdf

Please see the accompanying Medication Guide at www.corcept.com/medicationguide.pdf

About Corcept Therapeutics Incorporated
Corcept is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic and psychiatric disorders. Korlym, a first generation GR-II antagonist, is the company’s first FDA-approved medication. The company has a portfolio of new selective GR-II antagonists that block the effects of cortisol but not progesterone. Corcept also owns an extensive intellectual property portfolio covering the use of GR-II antagonists, including mifepristone, in the treatment of a wide variety of psychiatric and metabolic disorders. The company also holds composition of matter patents for its selective GR-II antagonists.

Statements made in this news release, other than statements of historical fact, are forward-looking statements. Forward-looking statements are subject to a number of known and unknown risks and uncertainties that might cause actual results to differ materially from those expressed or implied by such statements. For example, there can be no assurances that clinical results will be predictive of real-world use, or regarding the pace of Korlym’s acceptance by physicians and patients, the reimbursement decisions of government or private insurance payers, the effects of rapid technological change and competition, the protections afforded by Korlym’s Orphan Drug Designation or by Corcept’s other intellectual property rights, and the cost, pace and success of Corcept’s other product development efforts. These and other risks are set forth in the Company’s SEC filings, all of which are available from our website (www.corcept.com) or from the SEC’s website (www.sec.gov). We disclaim any intention or duty to update any forward-looking statement made in this news release.

CONTACT:

 

Investor Contact
Charles Robb
Chief Financial Officer 
Corcept Therapeutics
650-688-8783

New drug shows promise in patients with rare illness

Two in Montreal with Cushing’s disease were among 16 in international trial

By AARON DERFEL, The Gazette

Quebecers suffering from Cushing’s disease – a rare hormonal disorder – have some reason to be hopeful after a clinical trial conducted partly in Montreal has shown promising results of a new drug.

About 400 to 500 Quebecers have Cushing’s disease or syndrome, a disorder that produces tumours on the pituitary gland, leading to a spike in cortisol levels.

The excess cortisol can cause a wide range of problems, including obesity, hypertension and diabetes as well as sleep and mood disorders.

In some people, deposits of fat accumulate on the back of the neck and shoulders, an effect known as a “buffalo hump.”

Until now, surgery and radiotherapy have been the only options for many patients. However, a drug developed by Novartis Pharmaceuticals has been found to act on the tumours, cutting cortisol levels an average of 50 per cent.

In some patients – two of them from Quebec – cortisol levels returned to normal.

The drug, pasireotide, involves twice-daily injections. The treatment has yet to be approved by Health Canada.

“This study is very promising, especially for patients for whom complete surgical removal of tumours – the standard treatment for this disease – was not possible,” said Dr. André Lacroix, an endocrinologist at the Centre hospitalier de l’université de Montréal.

Lacroix and his colleagues tested the drug on four patients. In addition to the two who “experienced a complete regression of all symptoms” of Cushing’s disease, the two others reported drops in their cortisol levels and an improvement in their health, Lacroix noted.

The findings of the international study of 16 patients were published in the latest edition of the New England Journal of Medicine.

Pasireotide acts by binding to certain receptors on the tumours. Lacroix’s team has also carried out a separate study of another drug that targets a different receptor.

He suggested that using the two drugs together might prove even more beneficial, but this must be borne out by further research.

The ideal patients for the drug therapy would be those whose tumours are too small for surgery, Lacroix said.

Each year in Quebec, there are about 15 new cases of Cushing’s disease, and doctors at the CHUM treat about 150 patients.

aderfel@montrealgazette.com

© Copyright (c) The Montreal Gazette

 

 

Read more: http://www.montrealgazette.com/health/
drug+shows+promise+patients+with+rare+illness/6340316/story.html