Close up on a pile of yellow pain pills

Addressing the Opioid Epidemic Starts with How We Treat Pain

As a nurse practitioner in a busy suburban emergency department, pain is my job. Pain is one of the most common reasons people come to an emergency department (ED). It could be abdominal pain, chest pain, back pain or even emotional pain, including depression or suicidal ideations. Pain is a driver for people seeking medical care. We have made pain into a vital sign, and we ask, “How would you rate your pain on a scale of 1 to 10?” a mandatory question for any patient who steps through our door.

This whole concept evolved circa 1987 when the Institute of Medicine urged healthcare providers to use a quantified measure for pain. It gained even more traction in 1990 when then president of the American Pain Society, Dr. Mitchell Max, called for improved means to assess and treat pain. The term “oligoanalgesia” gained popularity in the published literature, meaning that we weren’t giving enough pain medication to patients in the ED, in clinics or in any other healthcare setting. Healthcare providers responded. We asked about and we thought, more effectively treated pain to address this issue.

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close up of human eye

The Luxturna Debate: Why Ethics Needs a Seat at the Drug Pricing Table

By Clio Sophia Koller

Jack Hogan can now ride his bike home at dusk after an afternoon of playing with his friends. Is that childhood rite-of-passage worth $850,000?

Recently, the Health Policy and Bioethics Consortium convened by Harvard Medical School’s Center for Bioethics and the Program on Regulation, Therapeutics, and Law (PORTAL) at Brigham and Women’s Hospital met to discuss the implications of Spark Therapeutics’ new gene therapy treatment—along with its staggering price tag.

Luxturna, a novel therapy approved by the FDA last year, treats a rare form of inherited blindness known as retinitis pigmentosa. The therapeutic agent targets the RPE65 gene, associated with the disorder, and is shown to improve vision in a population with progressive vision-loss and an inability to see in dim light. Read More

hand reaching for blue pills

Should Non-opioid Analgesics have “Opioid Sparing” as a Label Claim?

Most meetings of the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC) are held to review a New Drug Application (NDA), usually for a novel opioid preparation. But, on November 15, the AADPAC discussed the concept and implications of “opioid sparing” as a potential product label claim for a non-opioid type of medication.

Although trends in outpatient opioid prescriptions appear to be heading in an encouraging direction (see figure below), the demonstration that a non-opioid medication could further decrease the overall use of opioids for acute pain has obvious implications for the amount of opioids prescribed and taken by patients who undergo painful surgical procedures. This will also decrease exposure in the community where prescribed opioids may become diverted for recreational use.

In recent years, some across-the-board measures (e.g. state laws that limit the duration of prescribed opioids) have attempted to reduce opioid prescribing, but as the FDA emphasized in their presentation at the meeting, these “one size fits all” strategies will not account for individual patient variation, and may lead to inadequate pain treatment in some patients. Read More

a stethoscope tied around a dollar bill, with a bottle of pills nearby

Drug Pricing Controls and the Power of Familiar Ideas

Eight in ten Americans think that prescription drug prices are unreasonable, according to a March 2018 Kaiser poll. That same poll found that more Americans considered passing legislation to lower drug pricing to be a top priority than passing legislation to improve infrastructure or to address the prescription painkiller epidemic, among other things.

Effectively addressing drug pricing is a complex task that will require the diligent efforts of many actors. On October 24, the Petrie-Flom Center held a full day’s programming to this important and timely topic. What I want to state here is a simple point—namely, that the very discussion of potential solutions can play a role in turning creative innovations into implementable solutions.

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Commentary: Do We Really Need a New, More Powerful Opioid?

The FDA’s Analgesic and Anesthetic Drug Advisory Committee (AADPAC), of which I am a member, met October 12 to discuss a controversial New Drug Application (NDA) for a powerful opioid called sufentanil, manufactured by AcelRx.

Like fentanyl, sufentanil is a short-acting synthetic opioid, but approximately 5 to 10 times more potent. In the midst of the current opioid crisis, why would anyone think that the availability of another powerful opioid is a good idea?

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hand reaching for blue pills

The Rotten U.S. Antiparasitic Drug Market

Recently, there has been a lot of media attention on galling price hikes of generic drugs.

Historically, the social contract in pharmaceutical pricing has been tolerating expensive brand-name drugs while they have been on patent (a government-granted monopoly), followed by allowing low cost generics to rush to market after patent expiration. Yet these norms are now being challenged in the setting of increased generic manufacturer consolidation and single-source generic drugs.

Probably the most well known example is the case of Martin Shkreli (the so-called “Pharma Bro”) and Turing Pharmaceuticals, which bought out the rights of pyrimethamine (Daraprim), a key treatment for Toxoplasmosis and other infectious diseases, raising the price from $13.50 per pill to $750 per pill.

Note that even the pre-price hike price is significantly more than people other countries pay. In the UK it costs only $0.66 per pill and in Australia it is $0.18 per pill.

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shopping trolley with medicine

Step therapy explained: An increasingly popular tool for cost control

News that the Centers for Medicare and Medicaid Services will allow Medicare Advantage programs to enact “step therapy” programs for drugs under Part B as part of an effort to combat rising drug prices has been making rounds in the health policy world recently.

Step therapy is used by all major private insurers and is aimed at curbing expenditures on expensive drugs. It requires that a patient to try a less expensive alternative treatment. Those who fail treatment with the less expensive drug would then be eligible for coverage of the more expensive treatment. Note that it is very similar to prior authorization, a ubiquitous policy tool in which a drug is approved for coverage only after ensuring certain clinical criteria are met.

What was once a relatively rare tool is now commonly used. I examined UnitedHealthcare’s list of step therapy drugs and there are now over 100 listings. This is an order of magnitude increase from the number of drugs listed just four years ago, when I first got interested in this issue.

Drugs listed for step therapy tend to be either new, extremely expensive therapies (e.g., 3rd-line biologics for rheumatoid arthritis, sofosbuvir for hepatitis C) or more expensive formulations of common drugs (e.g., extended release formulation of quetiapine).

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Will the EPO’s Enlarged Board of Appeal step into the CRISPR patent battle?

By  Jakob Wested, Timo Minssen & Esther van Zimmeren

Another version of this contribution has been published in Life Science Intellectual Property Review (LSIPR).

The Broad Institute is facing a formidable task in defending the revoked CRISPR patent claims in their pending appeal at the European Patent Office (EPO). Ultimately, some of the issues might still be referred to the Enlarged Board of Appeal. However, this might require a significant amount of legal and rhetorical agility.

“The Opposition Division’s interpretation of the EPC [European Patent Convention] is inconsistent with treaties designed to harmonize the international patent process, including that of the United States and Europe.”

This was the rather strong reaction of the Broad Institute after the EPO’s Opposition Division’s (OD) decision to revoke one of their CRISPR patents. It could, however, also be argued that the case presents a simple failure of the patent applicants to comply with the long-standing European practice to apply an “all applicants” approach when claiming priority under article 87 of the European Patent Convention.

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CMS Abandonment of Outcomes-Based Payment Deal with Novartis is a Missed Opportunity

Earlier this week, Politico broke the news that the Centers for Medicare and Medicaid Services (CMS) had withdrawn its outcomes-based payment deal for Novartis’ CAR-T therapy, Kymriah, without public acknowledgement.

The Food and Drug Administration’s approval of Kymriah in August of last year was accompanied by the announcement of a novel outcomes-based agreement with CMS, in which CMS would pay for Kymriah only if patients had responded to it by the end of the first month. Now, CMS has quietly backed away from that agreement. What does the deal – and its subsequent abandonment – tell us about CMS’ involvement in outcomes-based contracts going forward?

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