Doctor asking patient to fill out survey before medical treatment.

Key Considerations for Patient-Reported Outcome Measures

By Sharona Hoffman

Patient-reported outcome measures (PROMs) are questionnaires that patients fill out on tablets or other computers or devices. They ask patients to check boxes in answer to questions about their symptoms, treatment effects, and ability to function physically, emotionally, and socially. They thus may solicit very sensitive information about matters such as anxiety, depression, and sexual satisfaction. To illustrate, a query might be “in the past month, how often did you have a lot of trouble falling asleep,” and the patient is asked to check “never,” “rarely,” “sometimes,” “often,” or “always.”

PROM responses can be used for purposes of clinical care, research, quality improvement, Food and Drug Administration (FDA) approval of drugs and devices, and even insurance reimbursement. For example, insurers hypothetically could decide to decline coverage of particular treatments based on PROM responses indicating that many patients find them to be unhelpful.

I first became interested in patient-reported outcome measures because of an experience my husband had. Andy has Parkinson’s disease, and one of the neurologists he saw asked him to fill out a long questionnaire on a tablet computer before each appointment. This task was difficult for Andy because he had a hand tremor, and it was stressful because Andy worried that he would not have time to complete the survey before his appointment began. Moreover, Andy’s physician never referred to his responses and appeared never to look at them. Upon investigation, I found little to no analysis of PROMs in the legal literature, so Andy and I recently published a law review article about them.

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Operating room Doctor or Surgeon anatomy on Advanced robotic surgery machine.

Protecting Consumer Privacy in DTC Tissue Testing

By Adithi Iyer

In my last piece, I discussed the hypothetical successor of 23andme — a tissue-based direct-to-consumer testing service I’ve called yourtissueandyou — and the promise and perils that it might bring in consumer health information and privacy. Now, as promised, a closer look at the “who” and “how” of protecting the consumer at the heart of direct-to-consumer precision medicine. While several potential consumer interests are at stake with these services, at top of mind is data privacy — especially when the data is medically relevant and incredibly difficult to truly de-anonymize.

As we’ve established, the data collected by a tissue-based service will be vaster and more varied than we’ve seen before, magnifying existing issues with traditional data privacy. Consumer protections for this type of information are, in a word, complicated. A singular “authority” for data privacy does not exist in the United States, instead being spread among individual state data privacy statutes and regulatory backstops (with overlapping sections of some federal statutes in the background). In the context of health, let alone highly sophisticated cell signaling and microenvironment data, the web gets even more tangled.

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sample tube in female hands with pipette.

Why We Should Care About the Move from Saliva to Living Cells in Precision Medicine

By Adithi Iyer

The cultural, informational, and medical phenomenon that is 23andMe has placed a spotlight on precision medicine, which seeks to personalize medical care to each patient’s unique makeup. Thus far, advances in direct-to-consumer genetic testing have made saliva-sample sequencing services all the rage in this space, but regenerative medicine, which relies on cells and tissues, rather than saliva, now brings us to a new, increasingly complex inflection point.

While collecting and isolating DNA samples from saliva may offer a wealth of information regarding heredity, disease risk, and other outflows of the “instruction manual” for patients, analyzing cells captures the minutiae of patients that goes “beyond the book” and most closely informs pathology. Disease isn’t always “written in the stars” for patients. Epigenetic changes from environmental exposures, cell-to-cell signaling behaviors, and the mutations present in diseased cells all profoundly inform how cells behave in whether and how they code the instructions that DNA offers. These factors are critical to understanding how disease materializes, progresses, and ultimately responds to treatment. This information is highly personal to each patient, and reflects behavioral factors as well as genetics.

Regenerative medical technologies use cell- and tissue-based methods to recapitulate, bioengineer, and reprogram human tissue, making a whole suite of sci-fi-sounding technologies an ever-closer reality. With cell-based and other regenerative therapies entering the market (making up an entire FDA subgroup), it well worth considering how cell-based medicine can advance the world of personalized consumer testing. In other words, could a corporate, direct-to-consumer cell-based testing service be the next 23andMe? And what would that mean for patients?

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Mushrooms, capsules, and dropper bottle.

Psychedelics Are Cheap. Psychedelic Treatment Is Not.

By Vincent Joralemon

Psychedelics hold immense potential to address an array of conditions that are otherwise challenging to treat, but accessing these therapies can be costly, which means that potential benefits will be stratified along the lines of socioeconomic status.

This is an acute concern, because many with conditions that psychedelics may help to treat — such as post-traumatic stress disorder, postpartum depression, treatment-resistant depression, and alcohol use disorder — lack the resources to pay for effective health treatments.

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Hundred dollar bills rolled up in a pill bottle

Reasonable Pricing Clauses: A First Step Toward Ensuring Taxpayers a Fair Return on their Public R&D Investment

By Nikhil Chaudhry and Reshma Ramachandran

Earlier this month, the Department of Health and Human Services announced that it had successfully included a reasonable pricing provision in a $326M investment contract with Regeneron for development of a next generation monoclonal antibody therapy for COVID-19. This was the first time the Biden Administration had included such a provision as part of its research funding agreements with the private sector, demonstrating that it is indeed possible for the federal government to negotiate deals with pharmaceutical companies that ensure that products developed with public dollars are priced comparably to the global market.

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Medical Caduceus Symbol as Scales with backlight over Wall in dark room.

Gender-Affirming Care, Abortion, and the Politics of Science: A Response to Wuest’s ‘Born this Way’

By Aziza Ahmed

On August 21, 2023, the 11th Circuit issued a decision that allowed a ban on transgender care to go into effect in Alabama. The Alabama ban, formally called the Alabama Vulnerable Child Compassion and Protection Act, is one of the most extreme of the many bans on gender affirming care. The law defines sex as the “biological state of being female or male, based on sex organs, chromosomes and endogenous hormone profiles…genetically encoded into a person at the moment of conception…” and targets physicians who might undermine this notion of sex with criminal prosecution. Their punishment could be up to ten years in prison.

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Three blister packs of birth control pills on a pink background.

Opill’s FDA Approval: Implications for Pharmaceutical Regulation and Access to Care

By James René Jolin and Susannah Baruch

On July 13, 2023, the United States Food and Drug Administration (FDA) approved Opill (norgestrel) as the first daily oral contraceptive available for non-prescription use in the U.S. While the timeline for availability and price of Opill will ultimately be set by its manufacturer, Perrigo, this recent move represents a significant step forward in improving access to contraceptive health care. Indeed, shortly after Opill’s approval, the American College of Obstetricians and Gynecologists described the FDA’s decision as “a critically important advancement in the accessibility of reproductive health care.”

In response to this development, Petrie-Flom Center intern James René Jolin and Executive Director Susannah Baruch sat down to discuss its legal, regulatory, and public health implications. This interview has been edited for brevity and clarity.

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Silver Spring, MD, USA - June 25, 2022: The U.S. Department of Health and Human Services (HHS), U.S. Public Health Service (USPHS) and FDA logos are seen at the FDA headquarters, the White Oak Campus.

FDA Solicits Feedback on the Use of AI and Machine Learning in Drug Development

By Matthew Chun

The U.S. Food and Drug Administration (FDA), in fulfilling its task of ensuring that drugs are safe and effective, has recently turned its attention to the growing use of artificial intelligence (AI) and machine learning (ML) in drug development. On May 10, FDA published a discussion paper on this topic and requested feedback “to enhance mutual learning and to establish a dialogue with FDA stakeholders” and to “help inform the regulatory landscape in this area.” In this blog post, I will summarize the main themes of the discussion paper, highlighting areas where FDA seems particularly concerned, and detailing how interested parties can engage with the agency on these issues.

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