The New Alphabet Soup of Market Access and Reimbursement: Part 1

Although I have never been a patient in a Clinical trial, a personal medical experience got me thinking about today’s newsletter topic. Years ago (when I was oh-so-young and naïve), while working in the lab during my early years in the industry, I was hit with an unbearable abdominal pain . . . one so bad I was rushed to the Doctor for a look. After a few palpations of my abdomen and questions posed by the Doc, he claimed it’s “probably just a kidney stone” that will take care of itself, and I was sent on my way. In hours the pain completely subsided, so I assumed he was right and I passed the stone (TMI?!). HURRAY, what an accomplishment!! Three months later the pain returned with a vengeance.

19Buckled over, sweating, nauseous, and vomiting (TMI?!), I insisted on a return trip . . . to a DIFFERENT doctor — this was not a kidney stone! Perhaps a kidney BOULDER?!?!

Long story short and an ovariectomy later, the pain was due to a large ovarian cyst and ovarian torsion. OUCH! Yes . . . my PRO (Patient-Reported Outcome-more below)was MORE IMPORTANT/ACCURATE in this case than the initial Doctor’s “diagnosis.”

Had I been more descriptive in my pain and symptoms from the start, perhaps an x-ray or c-scan would have been ordered, and three months of ovarian torsion leading to a “dead” ovary would have been prevented! Thankfully, my two kids are proof that you only NEED one ovary! 😉

At this point . . . I’ll stop with the grueling medical chat and introduce you to a member of our wonderful team of consultants, Jamie Banks, PhD, who can talk much more intelligently about PRO’s. Jamie is a health care consultant with more than 25 years of experience in drugs and medical devices. She earned her PhD in Health Economics/Social Policy from the University of Kent in Canterbury, UK, and Master’s Degrees in Clinical Evaluative Sciences from Dartmouth Medical School and Interdisciplinary Neurosciences from MIT. Her consult­ing services include evaluations in health economics, outcomes research, health policy, value dossiers, technology valuation, and communications/publications.

It’s Jamie’s turn!

A new vocabulary is being added to our industry-speak. The terms, FDA, NDA, RCT, CDER, 501(K), and PDUFA (etc., etc., etc.) are no longer enough; now there’s CEA, CER, HEOR, PRO, QOL, and SEALD, to name a few. So what’s going on?

As health care professionals, we converse in jargon, acronyms, and abbreviations. The tra­ditional terms relate to the regulatory process. The new ones relate to demonstrating value to health plans, payers and providers, patients/families, and society to optimize market access and reimbursement. It’s the new language; it’s here to stay, so we may as well start learning it.

In this issue (Part 1), I’ll introduce the world of “patient-reported outcomes” (PROs). Now, when you see PRO, QOL, HRQOL, SEALD, SF-36, EQ-5D, you’ll sound like an expert to your friends and colleagues. In subsequent issues, I’ll discuss terms related to health economics, outcomes research, and the growing field of comparative effectiveness research.

What is a PRO?

The U.S. FDAs 2009 guidance, Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims,defines a PRO as ” . . . any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” PROs are useful mainly to help clinicians and other decision-makers understand, in a comprehensive way, the impact of disease and treatment from the patient’s perspective. Their physician counterpart is usually referred to as a “clinician-reported outcome.”


Why Are PRO’s Important?

PROs provide different information on the patient’s health from the clinical exam. They have been shown to be powerful predictors of morbidity and mortality; in some cases, more powerful than objective health measures.

How often have you heard, “I don’t understand it. I just went to the doctor and she told me I’m fine; all my test results are normal. How come I feel so lousy?” (eh hem . . . Kelsey’s above story!) In fact, the doctor’s assessment often differs markedly from the patient’s assessment of his or her own health. Here’s an example.21

  • Your overweight neighbor regularly mows the lawn. It’s hot. He’s sweating, huffing and puffing, short of breath, barely able to stand when finished. In one scenario — he has a physical exam and receives a reassuring report: his blood pressure, heart rate, and lab results are all normal, although the doctor writes at the bottom, “Try to lose some of the ‘excess baggage’.” Alternatively, the patient fills out a question-naire at the doctor’s office and reports shortness of breath, profuse sweating, and occasional chest discomfort over the past month, clueing the doctor in to possible coronary disease.

How Are PROs Used?

PROs uses include:

  • Support for label claims (as primary/secondary trial endpoints)
  • Product differentiation
  • Outcome prediction
  • Dosing optimization

The FDAs 2009 guidance referenced above,was developed to ensure the validity and reliability of PRO endpoints, and the FDA even has its special SEALD (Study Endpoints and Label Development) Team to oversee the appropriate use of PRO endpoints in trials — Talk about Signed, SEALD, Delivered!

Isn’t PRO the Same as Quality of Life?

Contrary to popular belief, PRO is not the same as quality of life (QOL). There are many types of PROs, including QOL (more specifically, health-related QOL), symptom severity, sexual functioning, psychological distress, pain, and treatment satisfaction. Some measure multiple aspects, others single aspects of health. PROs may apply across different diseases (generic) or specifically to a single disease or set of disorders.

Measuring PROs

PROs are subjective and therefore need to be measured with validated “instruments” (rating scales, questionnaires). Read the FDAs 2009 guidance and you will realize quickly how complex this world of PROs really is! Thousands of PRO instruments are listed in the On-Line Guide to Quality-of-Life Assessment (OLGA) and nearly 700 in the Patient Reported Outcomes and Quality of Life Instrument (PROQOLID) database. The Short Form-36 (SF-36) and Euroqol-5D (EQ-5D) are widely used generic instruments. The Adult Asthma Quality of Life Questionnaire (AQLQ) and Migraine Specific Quality of Life (MSQOL) instrument are examples of disease-specific instruments.

BOTTOM LINE: ‘Mucho’ attention needs to be paid to PROs . . . many assume they have no “real” value, but now you know they can greatly affect product labels and product differentiation, both of which affect YOU (your HEALTH and your WALLET) directly!

Stay tuned for future issues on health economics, outcomes research and compar­ative effectiveness!!

Toto's Tips

  • Invest some time in researching PROs. PROs are being used increasingly in clinical trials, as well as other studies. Some knowledge will help you to stay ahead of the game.
  • Do not use the term PRO interchangeably with QOL or HRQOL. PRO is an umbrella term that encompasses many types of subjective self-reports from the patient.
  • Review the FDA’s 2009 guidance on PROs so you understand the concept and require­ments.
  • Remeber PROs are subjective and therefore need to be measured with validated “instruments,” as described in the referenced guidance.