Growth Hormone: Drug companies are growing less generous in helping patients pay for meds

For 14 years, Encino resident Ed Wright received an expensive prescription medication for free through a drug-industry program intended to assist people with limited or fixed incomes.

Now he’s rationing his doses after a change to the program that imposed a $1,100 deductible before he can get a refill.

“I can’t afford that,” Wright, 75, told me. “When I run out in a few weeks, that’s going to be it.”

He isn’t alone. Industry watchers say soaring drug prices have prompted many pharmaceutical companies to rethink long-standing programs to help subsidize purchases or even give meds away for free.

“More and more people have become aware of these programs, and demand has gone up,” said David P. Wilson, president of PRAM Insurance Services, a Brea firm that helps employers with prescription-drug benefits.

This means trouble for patients who, like Wright, can’t handle sticker shock at the drugstore.

He suffered a head injury 17 years ago that damaged his pituitary gland. In 2003, he was diagnosed as having an abnormally low level of growth hormone, which caused him to suddenly become overwhelmed with fatigue.

That’s a potentially life-threatening condition if an episode should occur while driving, walking down stairs or performing some other physical activity.

Wright’s doctor prescribed the self-injected human growth hormone Humatrope, manufactured by Eli Lilly & Co.

The cost, however, was out of reach for Wright, even with Medicare Part D. According to the drug-pricing website GoodRX, a 6-milligram cartridge of Humatrope — a one month’s supply — runs about $700.

Luckily, Wright’s fixed income made him eligible for a program called Lilly Cares, which made the drug available free of charge. He and his doctor would renew the paperwork annually, and for 14 years Wright had no difficulty receiving the med.

That’s no longer the case with the new $1,100 deductible, which requires Wright to spend that amount on prescription drugs before he can access his free Humatrope.

Wright requires few other drugs, so the deductible is an almost insurmountable barrier to maintaining normal quality of life.

Most drugmakers offer what are known as patient assistance programs, through which the company may provide meds directly to patients at little or no cost. Or the company may assist with co-payments — the patient’s out-of-pocket expense that’s not covered by an insurer.

2009 study published in the journal Health Affairs found that most patient assistance programs run by drug companies were reluctant to disclose details of the number of people they serve or the program’s eligibility requirements.

These programs “exist to provide patients with access to a wide variety of medications,” researchers concluded. However, “many details about these programs remain unclear. As a result, the extent to which these programs provide a safety net to patients is poorly understood.”

Aaron Tidball, chief Medicare advisor for the Illinois consulting firm Allsup, which assists individuals and businesses in navigating the public insurance system, said Lilly Cares “has been more generous than some programs we’ve seen.”

He said that, until now, people who qualified for Lilly’s assistance were able to receive whatever specialty meds that were prescribed by their doctor without cost or co-pay.

It should be noted, though, that Lilly has structured its program so the company benefits as well. Rather than provide drugs directly to patients, as many companies do, Lilly donates its medications to a private foundation, the Lilly Cares Foundation, which in turn deals with the public.

This allows Lilly to deduct the value of its donated drugs from its taxes. According to the nonprofit foundation’s 2015 tax return, which by law must be made public, the Lilly Cares Foundation received more than $408 million worth of drugs from the company. That figure represented the “fair market value” of the meds.

“That’s obviously a lot more than the cost to produce the drugs,” observed Jeff Geida, a Los Angeles estate lawyer who specializes in nonprofit foundations and who examined the most recent Lilly Cares tax return at my request.

In other words, Lilly was able to reduce its taxable income for the year by $408 million, although the actual expense of manufacturing the donated drugs almost certainly was just a fraction of the deducted amount.

“It’s a very good deal,” Geida said.

To be sure, the company is still doing enormous good by making millions of dollars worth of drugs available to people in need. But the inflated figures highlight the lack of transparency surrounding the true cost of prescription meds.

Julie Williams, a Lilly spokeswoman, declined to answer my questions about the Lilly Cares Foundation. But she forwarded a statement from Steven Stapleton, the foundation’s president.

He said the foundation imposed the $1,100 deductible for Medicare Part D beneficiaries “after benchmarking our program with other similar programs, helping Lilly Cares to balance all the criteria for the program and to try to help as many people as possible.”

That’s just gibberish to my ear — and doesn’t address the fact that Lilly still helps itself to that whopping tax deduction while making it considerably harder for low-income people to receive assistance.

I called the foundation and spoke with a service rep, but she said she didn’t know why the deductible was put in place. Nor could she explain how it’s in the best interest of patients with limited incomes to have to spend $1,100 on drugs before being eligible to receive a needed medicine.

Stapleton said notifications were sent to program participants in the fall of 2015 and 2016, but Wright told me he couldn’t recall receiving any such notice. The office manager of his doctor’s practice said she too was caught by surprise.

Lilly Cares made the situation even more inexplicable when it sent a notice to Wright last month formally dropping him from the program. The only reason it gave was “inactivity,” which made no sense considering that he’s been using Humatrope steadily for 14 years.

Williams, the Lilly spokeswoman, said she couldn’t discuss an individual patient.

Wright told me that, after I started poking around, he received a call from a Lilly representative. She advised him to contact the Partnership for Prescription Assistance, an industry-sponsored service intended to help people find subsidy programs that can help cover the high cost of their meds.

Wright contacted four subsidy programs through the service. Each one turned him down.

Lilly says it’s balancing all the criteria for Lilly Cares, which undoubtedly will make the company more profitable.

Wright, and the many other patients in similar positions, are a secondary consideration.

From http://www.latimes.com/business/lazarus/la-fi-lazarus-prescription-drug-assistance-20170815-story.html

NDA for Macrilen™ for the Evaluation of Growth Hormone Deficiency in Adults

CHARLESTON, S.C.–(BUSINESS WIRE)–Aeterna Zentaris Inc. (NASDAQ: AEZS)(TSX: AEZS) (the “Company”) today announced that it has been notified by the U.S. Food and Drug Administration (“FDA”), that the Company’s New Drug Application (“NDA”) seeking approval of Macrilen™ (macimorelin) for the evaluation of growth hormone deficiency in adults (“AGHD”) has been accepted as a complete response to the FDA’s November 5, 2014 Complete Response Letter and granted a PDUFA date of December 30, 2017.

David A. Dodd, President and Chief Executive Officer of the Company stated, “We are pleased that the FDA has formally accepted our resubmitted NDA and that it is under active review with an end-of-year PDUFA date. We remain confident that the FDA will approve our NDA and, therefore, we are moving forward with our preparations to launch the product in the first quarter of 2018.”

The Company also announces that Mr. Kenneth Newport is no longer a member of the Board of Directors effective as of July 12, 2017.

About MacrilenTM (macimorelin)

Macimorelin, a ghrelin agonist, is an orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this patented compound and has significant patent protection left. The Company’s U.S. composition of matter patent expires in 2022 and its U.S. utility patent runs through 2027. The Company proposes, subject to FDA approval, to market macimorelin under the tradename Macrilen™.

About AGHD

AGHD affects approximately 75,000 adults across the U.S., Canada and Europe. Growth hormone not only plays an important role in growth from childhood to adulthood, but also helps promote a hormonally-balanced health status. AGHD mostly results from damage to the pituitary gland. It is usually characterized by a reduction in bone mineral density, lean body mass, exercise capacity, and overall quality of life as well as an increase of cardiovascular risks.

About Aeterna Zentaris Inc.

Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel pharmaceutical therapies. We are engaged in drug development activities and in the promotion of products for others. We recently completed Phase 3 studies of two internally developed compounds. The focus of our business development efforts is the acquisition of licenses to products that are relevant to our therapeutic areas of focus. We also intend to license out certain commercial rights of internally developed products to licensees in non-U.S. territories where such out-licensing would enable us to ensure development, registration and launch of our product candidates. Our goal is to become a growth-oriented specialty biopharmaceutical company by pursuing successful development and commercialization of our product portfolio, achieving successful commercial presence and growth, while consistently delivering value to our shareholders, employees and the medical providers and patients who will benefit from our products. For more information, visit www.aezsinc.com.

Forward-Looking Statements

This press release contains forward-looking statements made pursuant to the safe harbor provision of the U.S. Securities Litigation Reform Act of 1995, which reflect our current expectations regarding future events. Forward-looking statements may include, but are not limited to statements preceded by, followed by, or that include the words “expects,” “believes,” “intends,” “anticipates,” and similar terms that relate to future events, performance, or our results. Forward-looking statements involve known risks and uncertainties, many of which are discussed under the caption “Key Information – Risk Factors” in our most recent Annual Report on Form 20-F filed with the relevant Canadian securities regulatory authorities in lieu of an annual information form and with the U.S. Securities and Exchange Commission (“SEC”). Such statements include, but are not limited to, statements about the progress of our research, development and clinical trials and the timing of, and prospects for, regulatory approval and commercialization of our product candidates, the timing of expected results of our studies, anticipated results of these studies, statements about the status of our efforts to establish a commercial operation and to obtain the right to promote or sell products that we did not develop and estimates regarding our capital requirements and our needs for, and our ability to obtain, additional financing. Known and unknown risks and uncertainties could cause our actual results to differ materially from those in forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue our research and development projects and clinical trials, the successful and timely completion of clinical studies, the risk that safety and efficacy data from any of our Phase 3 trials may not coincide with the data analyses from previously reported Phase 1 and/or Phase 2 clinical trials, the rejection or non-acceptance of any new drug application by one or more regulatory authorities and, more generally, uncertainties related to the regulatory process (including whether or not the regulatory authorities will definitively accept the Company’s conclusions regarding Macrilen™ and approve its registration following the Company’s re-submission of an NDA for the product as described elsewhere in this press release), the ability of the Company to efficiently commercialize one or more of its products or product candidates, the degree of market acceptance once our products are approved for commercialization, our ability to take advantage of business opportunities in the pharmaceutical industry, our ability to protect our intellectual property, and the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult the Company’s quarterly and annual filings with the Canadian securities commissions and the SEC for additional information on risks and uncertainties. Given these uncertainties and risk factors, readers are cautioned not to place undue reliance on these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law.

Contacts

Aeterna Zentaris Inc.
Philip A. Theodore, 843-900-3211
Senior Vice President
ir@aezsinc.com

From http://www.businesswire.com/news/home/20170718006321/en/NDA-Macrilen%E2%84%A2-Evaluation-Growth-Hormone-Deficiency-Adults

Growth Hormone: Improving Patients’ Lives and Boosting Mature Product Portfolios

Jul 07, 2017
Volume 37, Issue 7

easypod—an automated drug delivery device manufactured by Merck KGaA, Darmstadt, Germany for its recombinant human growth hormone, Saizen—is the only electronic, fully automated injection device for growth hormone therapy. Its features include automated dose delivery and prescription tracking, which records injection history and any missed injections, and allows patients to know when to change their cartridge by displaying how much medicine is left in the device.

Speaking to Pharm Exec, Merck KGaA’s Chief Operating Officer of Biopharma, Simon Sturge, outlines the device’s development and highlights its position in the context of a changing treatment-adherence landscape that could bring benefits both to patients and mature product portfolios.

PE: Are digital interventions in patient adherence becoming more of a focus at your company?

STURGE: Absolutely. We are a major player in the area of diabetes, for example, and as we all know, lifestyle has a huge impact on the outcome of diabetes. How much we as a company should be able to offer a whole package that helps to support the lifestyle changes needed is a very important element of us preventing or delaying the onset of diabetes. In other areas, many people who are sick have a degree of depression. There are excellent apps that are reimbursed in some countries to help treat depression, and those sorts of things should be offered as part of a solution. We believe it is an essential part of our business to look holistically at the patient and bring to that patient as many practical things as possible to help them overcome their disease.

However, innovative drugs are also at the core of what we do. A few years ago, we established a clear strategy of driving innovation in the area of specialty products. This has taken quite some time from an R&D perspective, but it is now coming to fruition, with a focus on the areas of oncology, immuno-oncology, and immunology. We have a number of exciting innovative products coming to market, and what we’re also seeing is substantial growth on the portfolio of our established products, one of which is our growth hormone, Saizen.

PE: How much did you incorporate patients’ adherence behaviors in developing easypod?

STURGE: Quite a few of our products are biotech products that need to be given via injection. Understanding the patient need around that product, how they inject, what the issues are, particularly for children, has helped drive our e-health and digital platform. We have a number of different applications around our growth hormone product, but the most sophisticated is easypod. The device sends administration data such as time and dose to the cloud via a mobile device or home network, and then shares that data with the treating physician or carer, to be able to understand the usage of that product.

There are digital ways that you can track people and their activities, of course, but what we’ve found is that you can’t beat having somebody almost living with a patient. In some circumstances we do that. We use an external group, and they send an observer to stay with a family for several days to really understand the practical issues that surround the use of the product. It’s those kinds of insights that really help to provide solutions that are practical and that address genuine issues that the patient wants to overcome.

Adherence in using an injectable product in a chronic environment can be very low, as low as 25%, but we’ve seen in controlled studies that with easypod that we can take that up to close to 90%.

[Ramy Sourial, growth hormone franchise director at Merck KGaA, Darmstadt, Germany, adds: We worked with patient organizations and healthcare providers at different stages of planning the device, and we used focus groups and market researchers to identify the needs. During development, we conduct regular tests to check that we are on the right track. And when the product is on the market, we continue to improve the device, even small things like designing covers and designing smaller needles.]

PE: Can this higher adherence be sustained in a real-world setting?

STURGE: We’re moving to very elegant devices, more universal devices; physicians and caregivers are becoming a lot more comfortable using the data that is generated. Where the big transition needs to take place is still with the payers. The NHS (National Health Service) is one of the most sophisticated providers in terms of understanding usage of products on a more holistic basis and has a willingness to work with the pharma industry on pricing and payment mechanisms that ultimately link efficacy with payment. As governments, payers, and the industry work more closely together, this will be of benefit to all parties and especially patients.

Our responsibility as a pharma company is broader than just supplying the drug. We have worked with the NHS on schemes where they only pay if the drug is used. If adherence levels are low, they don’t pay. In some of the pilot schemes with the NHS in a real-world setting, we were getting those adherence rates of close to 90%; we think that is quite achievable in everyday use. But there’s always things you can add, adding digital gains into these things to encourage children to use these devices on a daily basis; it’s a dynamic process and our aim is to try and maintain these increased adherence rates.

PE: What would you say are the remaining challenges in patient adherence?

STURGE: One of the biggest challenges we face is data privacy, the different data privacy laws country by country. If you end up having to develop software that has to be different in every country, it becomes less meaningful. Respecting and understanding data privacy but having a broader global alignment on data privacy laws in our industry will help everybody.

It will remain a sticking point for quite some time; it’s a highly complex and politically emotive subject, for very good reasons. But our concern isn’t around data privacy, per se—it’s consistency of the regulations thereof.

 

Julian Upton is Pharm Exec’s European and Online Editor. He can be reached at julian.upton@ubm.com

From http://www.pharmexec.com/improving-patients-lives-and-boosting-mature-product-portfolios

Pediatric Endocrine Society Provides Guidance for Growth Hormone Use in Pediatric Patients

HealthDay News—Use of growth hormone in children and adolescents should be considered carefully, with assessment of the risks and benefits necessary for each patient, according to guidelines published in the January issue of Hormone Research in Paediatrics.

Adda Grimberg, MD, from the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, and colleagues updated guidelines on the use of growth hormone, focusing on idiopathic short stature (ISS), GH deficiency (GHD), and primary insulin-like growth factor-I (IGF-I) deficiency (PIGFD). The guidelines were written on behalf of the Pediatric Endocrine Society.

The researchers recommend use of growth hormone for children and adolescents with GHD. Prospective recipients of growth hormone treatment should receive guidance regarding potential adverse effects and should be monitored for these effects. Parents and clinicians should take a shared decision-making approach to treating patients with ISS, and assess the physical and physiological burdens for the child, while considering the risks and benefits of treatment.Follow-up assessment of benefit and psychosocial impact should be conducted at 12 months after initiation and dose optimization of GH. IGF-I therapy is recommended for patients with severe PIGFD. Diagnosis of PIGFD/GH insensitivity syndrome should be based on a combination of factors that fall into four stages.

Physicians with expertise in managing endocrine disorders in children should provide consultation for evaluation of GHD-ISS-PIGFD and manage treatment.

“The taskforce suggests that the recommendations be applied in clinical practice with consideration of the evolving literature and the risks and benefits to each individual patient,” the authors write. “In many instances, careful review highlights areas that need further research.”

Several authors disclosed financial ties to the pharmaceutical industry.

Reference

Grimberg A, DiVall SA, Polychronakos C, et al; on behalf of the Drug and Therapeutics Committee of the Pediatric Endocrine Society. Guidelines for growth hormone and insulin-like growth factor-I treatment in children and adolescents: growth hormone deficiency, idiopathic short stature, and primary insulin-like growth factor-I deficiency. Horm Res Paediatr. 2016;86(6):361-397. doi: 10.1159/000452150

 From http://www.endocrinologyadvisor.com/adrenal/growth-hormone-use-in-pediatric-patients/article/634909/

Growth Hormone Deficiency Following Complicated Mild Traumatic Brain Injury

Traumatic brain injury (“TBI”) is considered the main cause of hypopituitarism in adults and growth hormone (“GH”)deficiency is the most common pituitary deficit associated with TBI.

According to Cedars-Sinai, even after we stop growing, adults need growth hormone. Growth hormone plays a role in healthy muscle, how our bodies collect fat (especially around the stomach area), the ratio of high density to low density lipoproteins in cholesterol levels, and bone density. In addition, growth hormone is needed for normal brain function.

A recent study aimed to assess pituitary function and GH deficiency in adult patients at different time durations following complicated mild TBI, according to the Glasgow Coma Scale (GCS). The study also aimed to evaluate whether mild TBI patients with GH deficiency had developed alterations in the glycolipid profile.

Forty-eight patients (34 men and 14 women) with complicated mild TBI were included in the study. Twenty-three patients were evaluated at 1 year (Group A), and 25 patients at 5 years or longer after the injury (Group B). All patients underwent basal hormonal evaluation for pituitary function. GH deficiency was investigated by the combined test (GH releasing hormone + arginine). The glycolipid profile was also evaluated.

Researchers report that GH deficiency occurred in 8/23 patients (34.7 percent) of Group A and in 12/25 patients (48 percent) of Group B. In addition, two patients, one in each group, showed evidence of central hypothyroidism. Patients examined one-year or several years after complicated mild TBI had a similarly high occurrence of isolated GH deficiency, which was associated with visceral adiposity and metabolic alterations.

These findings suggest that patients with complicated mild TBI should be evaluated for GH deficiency even if several years have passed since the underlying trauma.

From http://www.natlawreview.com/article/growth-hormone-deficiency-following-complicated-mild-traumatic-brain-injury

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