The FDA recently revved up its approval engine waving yet another specialty pharmacy checkered flag for Zokinvy (lonafarnib) from Eiger BioPharmaceuticals. Zokinvy is indicated for a rare genetic condition, Hutchinson-Gilford progeria syndrome, and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. It is an oral formulation. Prior to this approval only supportive therapies were available for affected patients.

Progeria is caused by a point mutation in a single gene called lamin A. This mutated gene produces an abnormal form of the lamin A protein called progerin.

Progeria and Progeroid Laminopathies are serious, ultra-rare diseases characterized by premature and accelerated aging in children. Globally, there are an estimated 400 children with Progeria and another 400 children with Progeroid Laminopathies (in total, probably fewer than 200 in the US). Untreated, most patients will die from heart failure, heart attack or stroke. There is no known cure and the average life expectancy for the untreated children is around 14 years.

Zokinvy is dosed by height and weight, with an average dose of 175 milligrams per day; a bottle of 50-milligram and 75-milligram capsules will cost $21,500 and $32,000, respectively. That translates into an annual cost of $252,000 – $384,000.

Given the very small patient population and very high cost, it is almost assured that this therapy will be launched through exclusive distribution.

---

FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies

SILVER SPRING, Md., Nov. 20, 2020 /PRNewswire/ — Today, the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. Zokinvy is not approved for use in patients with other progeroid syndromes or laminopathies.

“Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect on people’s lives,” said Hylton V. Joffe, M.D., M.M.Sc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “With today’s approval, Zokinvy is the first FDA-approved medication for these devastating diseases. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.”

Patients with Hutchinson-Gilford progeria syndrome and progeroid laminopathies experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. Before today’s approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.

Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. Compared to untreated patients, the lifespan of Hutchinson-Gilford progeria syndrome patients treated with Zokinvy increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of11 years. Zokinvy’s approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.

The most common side effects included nausea vomiting, diarrhea, infection, decreased appetite and fatigue.

Zokinvy is contraindicated for co-administration with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and certain cholesterol-lowering medications. Some patients treated with Zokinvy developed laboratory test abnormalities, such as changes in blood sodium and potassium levels, lowered white blood cell counts and increased liver blood tests. Routine blood laboratory testing should be obtained periodically. Eye toxicity was seen in animals so eye exams are recommended periodically and if there are new visual changes.

The FDA granted this application Priority Review designation. Zokinvy received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases, and Breakthrough Therapy Designation. In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children. The FDA granted the approval of Zokinvy to Eiger BioPharmaceuticals, Inc.

For a while we were seeing FDA specialty drugs approvals on a fairly regular basis….. then crickets. Perhaps the hiatus is over with the approval of two new products by TEVA Pharmaceuticals. Don’t get too excited….. they are generic versions of two old drugs.

We usually don’t cover generic approvals…. but it is worth mentioning these two as they are examples of a movement that is slowly picking up steam. In the next few years we will see more generic approvals….. as well as biosimilars that will finally see light of day once legal and patent challenges are behind them.

The other point of interest is that these approvals are in HIV which has seen very few new therapy approvals in recent memory.

So what are they?
The first is a new version of the successful 3 drug combo drug, Truvada. The very long generic name is Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate. As a generic it will come to market at a 70% discount to Truvada.

The second is a 2 drug combo equivalent to Atripla. It goes by Emtricitabine and Tenofovir Disoproxil Fumarate Tablets. Note, that it is the same as Truvada except that it leaves out the Efavirenz which may be supplemented by the physician for another drug.

How can generics impact pharmacy margins?
Specialty pharmacies that dispense the TEVA generic will see revenues drop since the generic price is about 70% lower than the brand. Often times, the spread for a generic is large enough for a pharmacy to realize the same or even greater net profit vs. the brand. It would be tough for the pharmacy to break even with that large a discount to the brand.

Click here to read the full TEVA press release

Are you up to speed on HIV Drug Classes, Single drug, Single pill, and Combination therapies? If not, take a few minutes to refresh your knowledge with this link.

Click here to read the article.

Teva Launches First Generic Versions of HIV-1 Treatments TRUVADA® and ATRIPLA® Tablets in the U.S.

October 02, 2020 — TEL AVIV & PARSIPPANY, N.J.–(BUSINESS WIRE)– Teva Pharmaceuticals USA, Inc., a U.S. affiliate of Teva Pharmaceutical Industries Ltd.(NYSE and TASE: TEVA), today announced the availability of the first Food and Drug Administration (FDA)-approved generic versions of TRUVADA® and ATRIPLA® tablets.

“As the global leader in producing and supplying generics, Teva is excited to launch these medications which further our commitment to delivering more treatment options to patients,” said Brendan O’Grady, Executive Vice President, North America Commercial, Teva. “These products, in addition to our more than 10 HIV-related medications already on the World Health Organization’s Essential Medicines list, represent Teva’s continued pursuit of treatments for HIV to improve health outcomes for the HIV community. During the COVID-19 pandemic, access to treatment is more essential than ever for those who are immunocompromised and at risk of developing more severe disease.”

With 1.2 million people currently living with HIV-1 in the U.S., Teva is committed to increasing access to critical HIV therapies. Despite significant advances in the treatment and prevention of HIV over the last two decades, there are still 12.6 million people globally who are unable to obtain treatment today. With the introduction of these new generic HIV treatment options, Teva strives to further increase access to important therapies.

These newly available generic medicines are indicated for:
Emtricitabine and Tenofovir Disoproxil Fumarate Tablets:
Treatment of HIV-1 infection when used with other anti-HIV-1 medicines in adults and children who weigh at least 37 pounds (at least 17 kilograms)
HIV-1 pre-exposure prophylaxis (PrEP) to reduce the risk of getting HIV-1 infection in adults and adolescents who weigh at least 77 pounds (at least 35 kilograms)

Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate Tablets:
Treatment of HIV-1 infection in people who weigh at least 88 pounds (40 kilograms), alone as a complete regimen, or in combination with other anti-HIV-1 medicines
Emtricitabine and Tenofovir Disoproxil Fumarate Tablets and Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate Tablets are both combination treatments available as a single pill with similar safety profiles to their reference products.

Once in a while it is good to believe in facts.
The brief article below deserves some serious attention as it includes some really positive facts about Cancer mortality rates.

Here’s the banner headline—-
24% decrease in deaths / 100k across the 15 most common tumor types (2000 thru 2016) across the 15 most common tumor types.

Now those are some impressive statistics.

What has been the primary drivers of the improvement in mortality rates over that period?

It is noteworthy that oncology therapy approvals even before 2010 were major contributors. However, we’ve seen multiple examples of specialty pharmacy cancer drugs with novel mechanisms of action come to market since 2010….. especially in the last few years.

The recently approved cancer therapies have been predominantly oral forms which also contribute to the improved mortality statistics as patients are more likely to remain adherent / compliant.

One last factor that deserves mention is the specialty pharmacy model itself which morphed to offer a distinct Oncology-specific model designed to support the unique needs of cancer patients, Oncologists, and even cancer therapy manufacturers.

---

Cancer Drug Approvals Associated with Reduction in Deaths

10 November 2020 — For the most common cancers, cancer drug approvals between 2000 and 2016 were associated with a reduction in deaths, according to a study published online Nov. 9 in the Journal of Medical Economics.

Joanna P. MacEwan, Ph.D., from Precision Health Economics & Outcome Research (PRECISIONheor) in Los Angeles, and colleagues examined the extent to which the approvals of new pharmacological therapies were associated with cancer mortality in the United States from 2000 to 2016. Cancer drug approvals across the 15 tumor types with the highest incidence were quantified. The annual tumor-specific cancer mortality, defined as the number of deaths per 100,000 U.S. population, was the primary outcome.

The researchers observed a 24% decrease in deaths per 100,000 population across the 15 most common tumor types between 2000 and 2016. Across the 15 most common tumor types, 10.2 new indications were approved per year. For colorectal cancer, lung cancer, breast cancer, non-Hodgkin lymphoma, leukemia, melanoma, gastric cancer, and renal cancer, cancer drug approvals were associated with significant reductions in deaths in 2016. Between 2000 and 2016, across the 15 most common tumor types, new cancer treatments were associated with 1,291,769 total deaths prevented.

“This study provides evidence that the gains in survival measured in clinical trials are translating into health benefits for patients in the real world and confirms previous research that has also shown that new pharmaceutical treatments are associated with improved survival outcomes for patients,” MacEwan said in a statement.

Specialty pharmacy under Medicaid is a bellwether for making observations on the broader specialty market and where the market is headed. Since Medicaid is state administered, the amount of reporting – and transparency – seems to be quite a bit more robust. The Magellan Rx team is one of the better sources of annual trend analytics and they’ve again done a great job with their 2020 Medicaid Pharmacy Trend Report.

The report also now includes a standard in-depth analysis of the top drug classes including six additional categories that provide a superior overview of classes with significant net dollar impact.

Key findings in this year’s Medicaid report include:
Specialty drugs accounted for 1.3 percent of utilization but 48.5 percent of 2016 net cost.
Traditional net spending on drugs decreased 0.4 percent from 2018 to 2019.
Unit cost, not utilization, drove specialty trend in 2019. The net cost per claim increased by $141.12, while utilization decreased by 0.9 percent.
Total net spend on specialty drugs increased by 2.4 percent suggesting that specialty drugs will account for 50 percent of total net spend for 2020.
Claim volume remains virtually unchanged

CLICK HERE TO ACCESS THE FULL REPORT (Select 2020 / Read the report)

---

Magellan Rx Management Releases Fifth Annual Medicaid Pharmacy Trend Report

November 13, 2020 — Magellan Rx Management, the full-service pharmacy benefits management division of Magellan Health, Inc. (NASDAQ: MGLN), released its fifth annual Medicaid Pharmacy Trend Report, the industry’s leading report exclusively detailing trends in the Medicaid pharmacy fee-for-service (FFS) space and the only detailed source examining Medicaid FFS gross and net drug spend trends.

“As a national leader in pharmacy benefit management, with more than 40 years of experience, we maintain a deep understanding of the complexities within the Medicaid space related to prescription drug costs and utilization trends,” said Meredith Delk, PhD, MSW, general manager and senior vice president, government markets, Magellan Rx Management. “The Medicaid Trend Report is one tool of many we deploy that provides value to our more than 25 government customers and Medicaid agencies across the country. We are delighted to release it for the fifth consecutive year.”

Developed through in-depth data analysis and supported by Magellan’s broad national experience managing Medicaid FFS pharmacy, the Medicaid Pharmacy Trend Report highlights the evolving landscape of Medicaid prescription drugs and anticipates the trends and challenges in the Medicaid FFS space.

“States are faced with inherent challenges related to the variability in the Medicaid program due to fluctuations in enrollment, enabling legislation and pharmacy program design,” said Chris Andrews, Pharm.D., vice president, value-based purchasing, Magellan Rx Management. “The Medicaid Trend Report clearly illustrates critical data-driven observations and helpful solutions that can assist states as they continue to explore and implement efforts to balance the growing cost of state Medicaid programs with state budget projections as they focus on achieving improved outcomes for Medicaid patients.”

The Magellan Rx Management Medicaid Pharmacy Trend Report includes data derived from Magellan Rx’s Medicaid FFS pharmacy programs in 25 states and the District of Columbia.

I’ve been ringing the alarm bell for several years now alerting the specialty pharmacy industry that it was under attack by health system owned specialty pharmacies. Many of my readers are sick of hearing that ‘the sky is falling’.

Today we are ready to say that a wall may have been hit. The article below details feedback from payers that they are now pushing back on health system owned specialty pharmacies.

Here are some payer comments—
A pharmacy is a pharmacy is a pharmacy.
They have the [same] ability to order a product.
They all have the [same] ability to dispense a product.
There is absolutely no difference between health-system specialty pharmacies and [independent] specialty pharmacies
Our [network] specialty pharmacy does everything a health-system pharmacy would do just as well.
There are “zero reasons” for health plans to work with hospital specialty pharmacies.

Now that’s different !

We believe that the incursion of health system owned specialty pharmacies has hit an inflection point. The largest health systems have used their prominence as ‘must have’ medical providers to bully their ways into payer pharmacy networks. Now that so many have been admitted to networks, payers are likely saying ‘enough is enough’.

So, is the danger to independent specialty pharmacies over?
No, not by a long shot.

As long as those big health system owned specialty pharmacies are in the payer networks they control a huge number of physicians that will write prescriptions that first get sent to the health systems’ pharmacies.

______________________________________________________________

Health Systems Versus Payors: A Duel for SP Care

Dueling misperceptions among health plans and payors about the value each brings to patient care can make it difficult for them to forge effective partnerships, according to a recent survey by Trellis Rx.

To create the report, 60-minute blinded telephone interviews with 10 hospital pharmacy leaders (i.e., directors, vice presidents, chief or specialty pharmacy managers) and 10 health plan leaders (i.e., medical directors, pharmacy directors, directors of pharmacy contracting), as well as nine subject matter experts: industry consultants and health-system pharmacy executives.

Many of the responses underscored the wide gulf that exists…….. CLICK HERE TO ACCESS THE FULL ARTICLE

By Gina Shaw
Specialty Pharmacy Continuum

There are almost always one or a few people within a specialty pharmacy that can wax on at length over the topic of compliance. Compliance is a broad term for many companies, but especially in the healthcare area.

Specialty pharmacies need to be very careful in ensuring that they are in full compliance with federal and state laws, Boards of Pharmacy requirements and a host of regulatory agencies. It is a full-time job.

The article below is one that those compliance ‘nerds’ should be able to write on their own. We offer it up for the rest of us that should know enough about this important topic to be dangerous. It is a bit of a long read but it is offered up in small digestible pieces. I always encourage people working in this industry to understand all of the working parts that are essential to ensure that they will survive and thrive in an increasingly competitive marketplace. So, give it a speed read at a minimum.

---

20 Elements of an Effective Pharmacy Compliance Program: A Nonexclusive List of U.S. Pharmacies’ Obligations Under Federal Law

Pharmacies operating in the United States are subject to a host of compliance obligations under federal law. These compliance obligations touch on all aspects of pharmaceutical practice, from drug ordering and inventory management to billing public and private payers. Despite the extraordinary scope of pharmacies’ compliance obligations, federal authorities do not excuse oversights. If an audit, investigation, or whistleblower complaint reveals evidence of shortcomings in any respect, pharmacy owners and pharmacists-in-charge can expect to face significant ramifications.

With this in mind, for pharmacies in the U.S., compliance needs to be a priority. Pharmacies cannot rely on the excuse of being overburdened with high demand, and they cannot expect to escape scrutiny if……….. CLICK HERE TO ACCESS THE FULL ARTICLE

Oberheiden P.C. © 2020
National Law Review, Volume X, Number 210

OK, this is not fake news….. the press release below details a new, exclusive-distribution arrangement for an oral therapy, Lysodren, with an indication for inoperable, functional or non-functional adrenocortical carcinoma.

Is this big news….. Not really.
Is this odd news…. Yes it is.
Why?
Because Lysodren (mitotane) was first approved in 1970….. yes, 50 years ago.

Mitotane is on the list of ‘Off-Patent, Off-Exclusivity Drugs without an Approved Generic’ as published in the Orange Book. Why it doesn’t have an approved generic is anybody’s guess….. there are lots of old brand drugs with no generics on that list waiting for someone to come along and relaunch an old cheap drug at 100+++ times the old price. Currently, Lysodren runs only $500+ per month of therapy.

The new FDA Label includes a black box warning for adrenal crisis…. which may be the primary reason that this drug is going limited distribution.

It appears that there is renewed interest in this old therapy. Over the decades it has been used routinely by veterinarians to treat carcinoma in animals.

AllianceRx Walgreens Prime named exclusive distributor for HRA Pharma Rare Diseases

FDA-approved prescription drugs Lysodren® and Metopirone® in the United States

Oct 29, 2020 — ORLANDO, Fla. and MORRISTOWN, N.J. and CHATILLON, France, PRNewswire. HRA Pharma Rare Diseases has named AllianceRx Walgreens Prime as the exclusive specialty pharmacy and clinical support provider for two of its rare disease drugs, Lysodren and Metopirone.

Lysodren (mitotane) is the only FDA-approved drug to treat inoperable, functional or non-functional adrenocortical carcinoma, a rare cancer of the adrenal glands. Metopirone (metyrapone) is used as a diagnostic test for adrenocorticotropic hormone (ACTH) insufficiency and in the diagnosis of ACTH-dependent Cushing’s syndrome. ACTH is a hormone produced in the pituitary gland in the brain.

“We are very excited to be the exclusive pharmacy to dispense these medicines,” says Tracey James, senior vice president of pharmacy services at AllianceRx Walgreens Prime. “HRA Pharma Rare Diseases recognizes our ability to clinically support patients throughout their treatment journey and link them to financial resources so they can obtain these life-changing medicines. HRA Pharma Rare Diseases and AllianceRx Walgreens Prime share the same goal of providing the best possible outcomes for these patients.”

Evelina Paberze, chief operating officer of HRA Pharma Rare Diseases, says, “At HRA Pharma Rare Diseases, we are dedicated to giving our patients improved access to essential treatments. The decision to make AllianceRx Walgreens Prime our exclusive specialty pharmacy and clinical support provider means our rare disease patients in the U.S. will get improved access to Lysodren and Metopirone.”

David Wright, chief executive officer of HRA Pharma, says, “At HRA Pharma, we constantly strive to provide accessible and affordable solutions to our patients. I believe making AllianceRx Walgreens Prime the exclusive pharmacy of our rare diseases affiliate is a step in the right direction to achieving this goal.”

For full prescribing information on the drugs listed above, including the black box warning for Lysodren®, visit dailymed.nlm.nih.gov.

We’ve often admonished people working in specialty pharmacy to be in the forefront of new developments. That’s easier said than done especially since the low hanging fruit in SP innovation has already been implemented.

The article below details something that is innovative under the SP umbrella, the concept of inserting Genetic Counseling into the SP model. The number of newly approved gene-based therapies is on the rise so the demand for counseling makes perfect sense. Why not insert it at the point of care where patients have the most interaction with pharmacists, at the point of dispensing.

This raises a serious question for specialty pharmacists…..
They already have to step up their game to master the clinical pharmacology of the new therapies. Do they also want to expand their role beyond clinical pharmacy to include the complex elements of genetic counseling? As the authors of the article below point out, genetic counseling must also include psychological counseling to assist patients make ethical choices about diagnostic testing and therapy selection. Does that mean SPs would staff a dedicated genetics counselor to work hand in hand with the pharmacist?

One area that the authors do not address is cost. Since it is likely to be complex and time-consuming, cost is a real concern. Who will pay a specialty pharmacy for counseling? Will payers compensate for the counseling if provided by a specialty pharmacist or a specialty pharmacy staff professional? If not, will a manufacturer of the genetic therapy step up to underwrite the counseling? That would make sense especially if the counseling resulted in improved adherence. We also believe that these uber expensive therapies will also be launched under value-based contracts with payers.

So, specialty pharmacies may want to think on this opportunity. Note, however, that virtually all genetic therapies approved so far have been launched through limited distribution. But, consider that offering genetic counseling might be the key differentiator that will propel your specialty pharmacy to be selected as a manufacturer’s limited distribution partner.

______________________________________________________________

The Expanding Roles of Genetic Counselors in Specialty Pharmacy Management

2020-10-29 — Pharmacy Times — Traditionally, the role of genetic counselors has been dedicated to caring for patients and families affected by genetic conditions. Today, genetic counselors apply their molecular and clinical expertise in diverse roles, many delivered via telehealth.

Recent rapid advances in gene-based therapies represent additional roles for genetic counselors throughout the health care system, including collaborations with pharmacy professionals. Here’s a look at some evolving changes and potential future shifts in how genetic counselors and pharmacists collaborate.

Prior authorizations (PAs) and specialty pharmacy
As health care practitioners prescribe genetic-driven therapies and seek PA from a health plan, pharmacy benefit managers play a critical role in determining if that request is appropriate for the patient. These prescriptions are often tremendously expensive. If they are misprescribed even 1% of the time, the result is potential harm for patients and a big financial waste.

As more therapies come online, an expert genetic counselor provides a valuable clinical and molecular review, as well as confirmation during the PA process. Beyond authorization, the majority of gene therapies fall to the specialty pharmacy at the point of dispensation.

Pharmacists in these environments may not have extensive molecular genetics training and often need to confirm that a gene-based therapeutic is appropriate and/or to educate patients. A genetic counselor can help patients clearly understand a drug’s mechanism of action and discuss whether it is a good fit with the genetic cause in their case.

Clinical genetic counseling services are typically covered by patients’ health insurance, including when delivered via telemedicine.

Navigating the testing landscape
Most pharmacists bring a level of genetic knowledge and understand the “why” behind a specific genetic therapy and the patient’s specific mutation. However, in some situations, lab reports and associated patient information could nudge a pharmacist outside of her or his comfort zone.

Lab reports generated in the United States are far from standardized. The same test result may look very different, depending on which lab it comes from.

Pharmacists, regardless of where they work in the health care system and patient treatment chain, may need added expertise in laboratory result interpretation and genetic nomenclature to precisely review molecular test results that may look different from one laboratory to another, even when dealing with the same disease and gene as a previous case.

In the landscape of genetics, there can also be different variants within a gene that have different impacts on a case. There is not a magical test that looks at all of those things equally and effectively, so added expertise helps.

Some variants, for example, are not normal or typical but are not likely problematic either. They fall into the gray zone of “variants of uncertain significance.”

It can be very challenging to understand how to interpret these results in the context of evaluating eligibility for a particular medication. In addition, sometimes a genetic diagnosis rests not solely on molecular results, but instead on a group of clinical features.

Clinical diagnostic expertise, in addition to the understanding and interpretation of molecular results, may be needed to establish a diagnosis. These are all skill sets that are in the genetic counselor’s toolbox—they are able to determine whether a patient meets diagnostic criteria for a particular indication or, alternately, if a diagnosis is sufficiently unclear and additional workup or testing to clarify the diagnosis may be indicated.

The patient education role
Through the years, the pharmacist role has evolved from dispensing to include direct care. Some pharmacists provide services such as glucose testing, immunizations, and blood pressure monitoring.

With closer patient contact, the educational role for the pharmacist increases as well. In the world of genetic therapies, they should expect the need to advance their knowledge base or to align with genetic counselors to add value to their role in the patient care process.

In addition to deep expertise in medical genetics, genetic counselors have extensive psychology training, and their bread-and-butter work involves explaining complex genetic information to patients who have minimal to no understanding of the subject. These skills can be of value whether working alongside pharmacists or providing professional education to them.

Let’s use the case of retinal dystrophy as an example, which causes blindness at a very young age. In 2017, Luxturna was approved for a very narrow subset of retinal dystrophy patients, those with a mutation in both copies of the RPE65 gene.

Luxturna offers a working version of the RPE65 gene that acts in place of the gene’s mutated version. This small subset of patients often experiences restored vision.

If a family member has retinal dystrophy but shares only 1 of their relative’s 2 genetic mutations, and is hopeful for that same outcome, it can be difficult for the patient and family to understand that their particular cause of the disease is not amenable to this treatment.

Luxturna is administered through in-patient settings, so the educational role of the genetic counselor would likely not be alongside pharmacists, but instead with the physician and patient leading up to treatment delivery. However, with information widely available on the internet, patients with retinal dystrophy and their families who are not offered the therapy may approach physicians, pharmacists, and other medical professionals on their care journey and request detailed explanations.

In these cases, the genetic counselor could step in and serve as a valuable resource. Bridging that gap between the patient’s and family’s hope and understanding is a key role for genetic counselors.

A key neutral party
Situations also arise in which a therapy may be a sizable cost to the patient but the benefits are only marginal. Genetic counselors are trained in facilitating decision-making, and are often the informational source that helps a patient understand, “What is the true value for me?”

At times, there is a need to filter out advertising, marketing, and other noise surrounding a particular therapeutic. The complex collection of players involved in patient care brings competing views.

The pharmaceutical companies may have a lot of bias and marketing sway. Physicians care about patients and will sometimes take risks to make them happy. Payers may not want to cover something if uncertainties exist.

Having a public health-minded independent party who will consider the best interests of the patient can neutralize these competing interests by focusing on facts, level-setting true benefits for them, and helping patients develop realistic expectations.

Experiences in patient-facing moments, behind the scenes during PA deliberations, and in the trenches of analyzing genetic tests all serve to drive genetic therapies forward and guide better future utilization. Genetic counselors can add significant value to this process.

Integration of genetics expertise will only become more important as a greater number of therapies come to market.

About the Authors
Gwen Fraley is Director of Clinical Policy at InformedDNA.
Heather Dorsey, is Evidence Review Manager, at InformedDNA

---

The article below, How Specialty Pharmacies Can Safeguard Against Fraud When Using Patient Assistance Programs, details yet another case including a specialty pharmacy. In this instance the SP was pinched by the feds for participating in a scheme to mickey with patient co-pay programs for Copaxone. Advanced Care Scripts, an Orlando based specialty pharmacy, was recently fined $3.5 million!

The details of the case are summarized in the article (link below)… but here’s the bottom line. If you want to avoid a big financial penalty, don’t step over well-defined lines of business behavior to collude with a manufacturer to violate laws.

Here are some best practices from the article that can keep specialty pharmacies (you) out of hot water related to patient assistance programs:
Carefully review data elements provided to drug manufacturers in data reports to ensure information about charitable support or status of a charitable support request is not provided.
Do not enter into contractual arrangements with drug manufacturers to coordinate charitable foundation support. Assisting patients in identifying such funding should be conducted as part of the pharmacy’s core services and not as directed by a drug manufacturer.
Train staff to avoid any coordination with pharmaceutical manufacturers on the provision of charitable funding to patients. This includes not only through contractual agreements but also by avoiding such discussions informally or in scheduled business reviews.
Ensure robust contractual language is in place clearly prohibiting pharmaceutical manufacturers from utilizing data provided by the specialty pharmacy to make charitable funding decisions.

One need not have been a lawyer to know when lines had been crossed in the ACS/TEVA case. Since $3.5 million is a whole lot bigger number than, say, $5000-$10,000 in legal fees, having your SP’s lawyer on speed dial might be the very next thing you do today.

Click here to read the complete article from Bass, Berry & Sims PLC

---

Anton RX Reports are copyrighted by Anton Health, LLC.

HIPAA…. We all know everything there is to know about HIPAA, right??
…… Wrong.

One aspect of HIPAA that gets almost all the attention is how to prevent breaches of private healthcare information. If your specialty pharmacy is accredited, you have been through a bunch of orientations pounding the requirements into your brain.

But, how many orientations have you survived that focus on what to do after a breach of private information? We’ve provided a link to a good, but lengthy, article for your reading pleasure….. Ensuring Transparency: Language to Avoid in HIPAA Breach Notifications. The article is well worth a read if you are a specialty pharmacy professional….. for HIPAA compliance officers this should be a must read.

The author correctly states, “Breach notifications [to patients following a breach] often contain wishy-washy wording… while it may meet the minimum legal requirements, it doesn’t really tell people what actually happened to their personal information.”

In terms of transparency when it comes to HIPAA, notifications must include a brief description of the breach, types of information involved, steps affected patients should take to protect against potential harm, and what the covered entity is doing to both investigate the breach – and to prevent future incidents. Notices must also include contact information.

Health care providers should also be aware that HIPAA is not the only regulation that should govern their response to a security incident. States, federal regulations, and even foreign laws like the General Data Protection Regulation, may also govern the appropriate response.

So, is that it? Oh, no it is not.

The primary requirement in the aftermath of a breach is to be transparent in the notice to impacted patients. The secondary requirement is to know what language to avoid when making those communications.

Here are some highlights—-
avoid language that creates more uncertainty or anxiety
focus on what is known, not what is unknown , do not engage in speculation
avoid unsubstantiated facts….. but state (when pertinent) that certain facts may not known or are being withheld for security, legal or other reasons and provide supplemental notifications when more facts are known and can be shared
avoid deflecting blame for the incident by accepting qualified responsibility
don’t attempt to place the blame on another entity, such as the threat actor or a third-party vendor.

The article finishes with a section on best practices.

Click here to read the full article