PharmExec | Jill Wechsler
Great enthusiasm for personalized and patient-centered medicine is driving
a host of innovative regulatory and research proposals. The Food and Drug
Administration is seeking to promote informed self-treatment by switching some
current Rx drugs to OTC status with additional “conditions for safe use.”
Government support for Comparative Effectiveness Research (CER) offers new
opportunities for researchers, payers, and pharma companies to identify optimal
treatments for a broad range of medical conditions. The clamor for more
information on drug use and effects in such “real world” situations is shaping
clinical research and coverage decisions.
Promoting OTCs
As part of this overall effort to encourage Rx to OTC switches — thus
increasing patient access to treatments for chronic conditions such as high
blood pressure and cholesterol — the plan aims for strategies that can help
consumers select and use medicines appropriately without going to a doctor.
Under this “new paradigm” to improve public health, as described by agency
officials at a two-day public hearing in March, manufacturers would ensure
“safe use” of a newly turned- OTC medicine by utilizing information technology
to enhance and document appropriate treatment. Information kiosks in pharmacies
could direct consumers through self-diagnosis programs that check for risk
factors contraindicating treatment and monitor testing to ensure appropriate
care; pharmacists could supply personal counseling and additional oversight.
The expectation is that reducing the cost and inconvenience of visiting a
doctor to obtain a prescription will encourage broader self-treatment — and
promote adherence to recommended therapy after treatment begins. There’s strong
evidence that such Rx-to-OTC switches for common conditions, including vaginal
infections, allergies, and heartburn, have promoted effective care. At a
minimum, “rescue medicines,” such as asthma inhalers would be candidates for
enhanced pharmacy access.
Pharmacists are enthusiastic that this more flexible regulatory approach
would expand their role in improving public health — and generate added fees
for counseling and services. Vocal opposition comes from the American Medical
Association (AMA), which insists that physician involvement in patient
treatment is vital, especially for chronic diseases. Health plans and payers
stand to save money because most insurers and health plans do not cover nonprescription
medicines. This translates into higher out-of-pocket costs for individuals who
have drug benefits, a shift that the AMA predicts will undermine adherence.
Pharma companies could benefit from increased sales of nonprescription
products, but are fairly quiet about such prospects, particularly about how OTC
switches often aim to retain market share for a brand facing generic
competition. And drug distributors are uneasy about special programs that
require wholesalers to determine which pharmacies and customers may receive a
particular nonprescription product.
A policy that permits FDA to approve OTC products with added safe-use
requirements would require a lengthy FDA rulemaking process and most likely
authorizing legislation. It also would involve further development of label
comprehension studies and actual use trials able to document that the medicine
can be used safely and appropriately based on the approved “drugs facts box”
label, plus supporting information and services. IT and research firms are
ready with a range of strategies to support these efforts, including new ways
to evaluate and ensure compliance with self-diagnostic tools, to devise
collaborative programs that enhance care management, and to coordinate
follow-up and oversight in collaboration with physicians, payers and patients.
Despite regulatory, scientific, and political challenges, FDA leaders appear
optimistic that innovative studies and IT systems not only can enhance
medication use, but also identify drug interactions and improve
pharmacovigilance tracking.
CER challenges
Another way to help patients and providers make informed decisions — and to save money by curbing inappropriate care — is by evaluating effective treatment strategies and comparative study methods. The Patient Centered Outcomes Research Institute (PCORI) is poised to distribute some $120 million in the coming months — and nearly $400 million in 2013 — to launch a federal CER initiative, as authorized by the Affordable Care Act. Pharma companies already are providing more comparative and outcomes analysis to formulary committees, insurers, and payers to bolster claims of product value, and the new program should further document real-world effectiveness for certain medical treatments.
PCORI has spent the last year getting organized, hammering out definitions
for PCOR and CER, and seeking advice from all corners of the healthcare and
research community. Last month it finalized priorities for its research agenda
with an eye to awarding initial grants this fall. About $35 million will
support studies to identify medical products and practices likely to improve
patient outcomes for prevention, diagnosis, and treatment. Additional funds
will support efforts to improve healthcare systems and to set standards for CER
research methods, including less traditional approaches involving patient
registries and meta-analyses.
The growing demand for real-world CER data has generated considerable
discussion about the design of studies to demonstrate comparability and added
value of pharmaceuticals. At a PCORI stakeholder “dialogue” in February,
employers said it’s important to assess common chronic conditions that account
for much of healthcare spending, while patient advocates urged analysis of
treatment effects on individuals with less common critical diseases to avoid
one-sizefits- all coverage decisions that often reject new technology.
These developments are prompting sponsors to address CER earlier in the
drug development process, to engage stakeholders in clinical trial design
activities, and to explore innovative approaches for generating evidence, said
Eleanor Perfetto, senior director for reimbursement and regulatory affairs at
Pfizer, at an April CER summit sponsored by ExLPharma. Similarly, experts at
another CER conference organized by the Drug Information Association (DIA) and
the National Pharmaceutical Council (NPC) in March discussed the need to assess
co-morbidities, patient biology, and other characteristics likely to affect
response. Peter Neumann, professor of medicine at Tufts Medical Center, advised
sponsors to carefully choose comparators, outcomes, and subgroups to study.
A serious concern, though, is that too large and costly CER studies will
stymie innovation. Clinical trials with multiple subgroups, active comparators,
and long-term endpoints can make drug development more expensive, pointed out
NPC research director Jennifer Graff at the ExLPharma conference. Such
investment could pay off by supporting broader indications and higher prices
for medicines found effective with certain patients, along with a higher
probability of success for a clinical trial, faster FDA approval of a new
product, and providing a basis for additional indications in the future.
Yet, Naomi Aronson, executive director of the BlueCross BlueShield
Technology Evaluation Center, voiced skepticism that CER will greatly improve
the evidence base for health plan decision making. And even if a pharma company
can document that some patients do better on a new drug, if that therapy is too
expensive, she said, the health care system still won’t be able to afford it.
To reduce costs and risks, pharma companies are forming partnerships with
health plans and pharmacy benefit managers to assess treatment effects. A
Pfizer-Medco partnership, Perfetto noted, aims to utilize genomic and
phenotypic data in studies that match patients to treatments most likely to
provide benefit. Similar collaborations are evaluating a range of real-world
treatment strategies on patient subgroups and specific diseases.
These and other research initiatives highlight PCORI’s important role in
setting standards for CER analysis and in coordinating CER activities to avoid
redundancies. Dissemination of CER results also is critical to ensure that
clinicians incorporate research findings into practice. PCORI will support
communications activities by the Agency for Healthcare Research and Quality
(AHRQ), which is holding a symposium in June to further discuss how research
methods can shape the results of randomized trials and observational studies.
A hot issue for marketers is that FDA regulations constrain them from
commenting on CER findings that invariably touch on unapproved uses of a
medical product, even though the government, academics, payers, and insurers
are free to disseminate and discuss all aspects of outcomes and comparative
studies. At an NPC conference in February, Robert Temple, deputy director for
clinical science of FDA’s Center for Drug Evaluation and Research, emphasized
that industry is free to rebut clinical findings that they find objectionable,
so long as they present factual and objective information — and avoid promoting
off-label uses. Pharma companies want a more level playing field as CER plays
an increasingly important role in shaping coverage decisions, but insurers and
payers are leery that industry will use CER for promotional purposes. CER
studies will be under ever-sharper scrutiny to ensure they avoid bias and
encourage better care.
About the Author
Jill Wechsler is the
Washington Editor for Pharmaceutical Executive and writes about federal
government policies and programs that affect the pharmaceutical industry. Her
monthly Washington Report discusses legislative proposals, FDA initiatives and
actions by other government agencies, including Medicare pharmacy benefit
proposals, federal investigations related to pharmaceutical company marketing,
debate over DTC advertising, generic drug competition, over-the-counter
initiatives, among other topics. She has written for PE for more than ten years
and also covers Washington for other Advanstar publications, including
Pharmaceutical Technology, Applied Clinical Trials, BioPharm, Managed
Healthcare Executive and Formulary. Prior to that she reported on government
policies and a wide range of topics for business and consumer publications.