Pile of colorful pills in blister packs

The Age of Orphans

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By Sarah Rickwood

The 2020s are the age of orphan medicines.

Orphan medicines are available for a larger number of diseases and patients than ever before, a testament to the success of legislation established decades ago encouraging the development of these medicines in both the U.S. and European Union.

Orphan medicines were the majority of European Medicines Agency (EMA) approvals for the first time ever in 2016 (59%) and the majority of U.S. Food and Drug Administration (FDA) approvals for the first time ever in 2018 (58%).

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person cuts salami sausage on a wooden cutting board.

Tackling Salami Slicing and Indication Stacking in Orphan Drug Innovation Incentives

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By Sven Bostyn

The development of orphan drugs, so named for the rare diseases they treat, has been incentivized through regulation in the European Union. The primary reward is 10 years’ market protection (or exclusivity).

But are these incentive mechanisms working as they should? To date, only 131 orphan drugs have been brought to market. Findings from the European Commission’s long-awaited evaluation of the orphan drug system in Europe, 20 years after its inception, suggest there may be cause for concern.

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Blister pack of pills, but instead of bills dollar bills are rolled up in the packaging

Mortal Sins of Orphan Drug Development: How to Save the Lost Souls

Join the authors on Friday, September 17, 2021 for the 2021 CeBIL Symposium. Register here!

By Jakob Wested and John Liddicoat

In a working paper from November 2020, the EU Commission finds a significant inefficiency in the EU orphan drug regulation (a pan-EU piece of legislation): that it does not contain a provision to safeguard the affordability and accessibility of orphan medicines.

The working paper then entertains the idea of inserting such a provision into the regulation. But, is the orphan drug regulation the right place for this type of law?

Diagnosing the problem behind orphan drug pricing is the key issue to address before jumping to consider ways to address excessive pricing.

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Picture of Ivermectin tablets.

Legal and Ethical Analysis of Court-Ordered Ivermectin Treatment for COVID-19

By Jennifer S. Bard

A judge in Ohio ruled on Monday that a hospital in the region must administer ivermectin to a patient very sick with COVID-19 in their ICU, despite the decision by the medical staff, in agreement with recent statements by the U.S. Food and Drug Administration (FDA) and Centers for Disease Control and Prevention (CDC), that ivermectin is not an appropriate treatment, as it has been shown not to work against COVID.

The patient’s prescription came from a doctor who has no authority to treat patients at this particular hospital, although he is licensed to prescribe medicine in Ohio.

This case tracks a swelling interest, which some ascribe to the efforts of a group called America’s Front Line Doctors, among people for the anti-parasitic medication as both a treatment and prophylactic for COVID-19 — despite warnings from the medical establishment that it doesn’t work, and, if taken in the form normally given to farm animals or at the dosages being suggested, can be harmful.

The Ohio ruling is just the latest of several successful law suits (see similar cases in New York and Illinois) to order hospitals to administer ivermectin to hospitalized COVID-19 patients, despite the objections of the treating physicians.

There is also evidence of a global trend, as evidenced by the order of a court in South Africa to allow the prescription of ivermectin for COVID-19, something that was previously not permitted by the country’s drug regulatory agency.

This trend of courts ordering that treatments requested by hospitalized patients be made available by that hospital — so long as they are prescribed by a physician — opens the door to substantial administrative, legal, and ethical chaos. This post analyzes some of the most pressing legal, regulatory, and ethical concerns.

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Abortion rights protest following the Supreme Court decision for Whole Women's Health in 2016

How Social Movements Have Facilitated Access to Abortion During the Pandemic

By Rachel Rebouché

Before the end of 2021, the U.S. Food and Drug Administration (FDA) will reconsider its restrictions on medication abortion. The FDA’s decision could make a critical difference to the availability of medication abortion, especially if the Supreme Court abandons or continues to erode constitutional abortion rights.

Under that scenario of hostile judicial precedents, a broad movement for abortion access — including providers, researchers, advocates, and lawyers — will be immensely important to securing the availability of remote, early abortion care.

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LONDON, UNITED KINGDOM- 1 APRIL 2015: A newspaper rack holding several international newspapers, such as The International New York Times, USA Today, Irish Times, Londra Sera and Corriere Della Sera.

Monthly Round-Up of What to Read on Pharma Law and Policy

By Ameet SarpatwariBeatrice Brown, Alexander EgilmanAviva Wang, and Aaron S. Kesselheim

Each month, members of the Program On Regulation, Therapeutics, And Law (PORTAL) review the peer-reviewed medical literature to identify interesting empirical studies, policy analyses, and editorials on health law and policy issues.

Below are the citations for papers identified from the month of July. The selections feature topics ranging from a discussion of issues related to FDA’s approval of aducanumab for the treatment of Alzheimer’s disease, to an analysis of the communication of survival data in cancer drug labels, to an evaluation of public-sector contributions to novel biologic drugs. A full posting of abstracts/summaries of these articles may be found on our website.

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Globe and vaccine.

Access-to-Medicines Activists Demand Health Justice During COVID-19 Pandemic

By Brook K. Baker 

It was apparent from the outset of the COVID-19 pandemic that a business-as-usual approach — perpetuating the biopharmaceutical industry’s intellectual property-based monopolies and allowing artificial supply scarcity and nationalistic hoarding by rich countries — would result in systemic failure and gross inequity.

The world had seen it all before, from the Big Pharma blockade of affordable antiretrovirals to treat HIV/AIDS, to the hoarding of vaccines by the global north during the H1N1 bird flu outbreak in 2009 and its stockpiling of Tamiflu.

Activists in the access-to-medicines movement quickly mobilized to combat the threat of vaccine/therapeutic apartheid.

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Hundred dollar bills rolled up in a pill bottle

To Address the Overdose Epidemic, Tackle Pharma Industry Influence

By Liza Vertinsky

A recently released government report estimates that 93,000 people died from drug overdose in 2020. This estimate reflects a jump in the death toll of almost 30% from 2019 to 2020, with opioids as a primary driver.

In response, President Biden has called for historic levels of funding for the treatment and prevention of addiction and drug overdose.

Transforming mental health and addiction services is a critical part of tackling the overdose crisis, but it is not enough, on its own, to address this epidemic, or to prevent a future one. We must also alter the conditions that fueled expanded use, and abuse, in the first place. As I argue in Pharmaceutical (Re)capture, a forthcoming article in the Yale Journal of Health Policy, Law and Ethics, this includes a change in how we regulate markets for prescription drugs.

To truly combat the epidemic, I suggest, we have to understand how pain became such a lucrative business and how regulators failed to protect the public health as the market for prescription opioids grew. Then, we need to put this understanding to work in the redesign of pharmaceutical regulation.

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Person typing on computer.

COVID-19 and the New Reproductive Justice Movement

By Mary Ziegler

The COVID-19 pandemic has transformed advocacy for reproductive rights and reproductive justice in what previously had been called an endless, unchanging, and intractable abortion conflict.

The pandemic — and the stay-at-home orders it required — finally shifted the movement’s focus to abortion access, rather than abortion rights, as exemplified by its emphasis on medication and telehealth abortion.

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