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...FDA Commissioner Scott Gottlieb, MD, announced that the Center for Drug Evaluation and Research would be taking steps to modernize the organization and functions of the Office of New Drugs in order to address scientific and medical advances within the industry. The goal is to make the review process more integrated across science and regulatory expertise. Janet Woodcock, director of CDER, plans on elevating the role of FDA scientists and medical officers and providing these officers with more tools and support “to advance the clinical and regulatory principles that the FDA uses to evaluate new drugs for safety and efficacy.”...Other changes will include the development of guidance documents, giving review staff more time with sponsors, and getting sponsors involved earlier in the development process. Engaging disease specialists, academic researchers, regulatory partners at other agencies, and patient groups is also a goal of CDER.

For more than 15 years NICE has punched above its weight internationally but as it comes under attack for its methodology, will its international clout suffer?...The bad headlines are back; on 20 May, the National Institute for Health and Care Excellence...came under fire from the media for its decision to bar Roche's Perjeta (pertuzumab) for breast cancer from the NHS...While patient groups and charities are worried about the effect on cancer patients in England, who may be denied treatment, there may also be a knock-on effect in other countries, which still look to NICE for guidance over their own reimbursement decisions...NICE International...advises countries from Ghana to Kazakhstan on HTA (health technology assessment) methodology and implementation...Yet, its international spread has come hand-in-hand with the growing disillusionment about NICE's decisions...An IMS Institute study in 2013 compared the reimbursement of cancer drugs in five countries that used cost per QALY (NICE's quality-adjusted life years) methodologies, including the UK, with five using broader methodologies, including Germany and US. It found that the cost per QALY countries reimbursed fewer cancer drugs, had slower access to those they did adopt, and generally performed poorer in terms of cancer outcomes. Moreover, it was not clear that they saved much money.

It may have been too good to last. After an upswing in pharma's global reputation in 2015, its standing among patient groups has swung the other way—again. Only 38% of patient groups said the industry had either an "excellent" or "good" reputation, down from almost 45% last year...after last year’s peak, pharma scores dropped across all the measurements in PatientView's annual survey, including innovation, transparency, pricing, ethical marketing and high-quality, useful products...Almost all of the other healthcare segments...from biotech to insurers, also dropped in their esteem. The only exception was not-for-profit insurers.

• Blame President Donald Trump...with his very public criticisms of pharma "getting away with murder" and his vows to bring down drug prices.
• Then there's the ongoing pricing debate…
• ...another part of the reason for the overall declines may be that, as patient groups become a more important part of the healthcare conversation and infrastructure, they feel more confident speaking their minds...

Perched on an exam table at the doctor’s office watching the clinician type details about their medical problems into their file, what patient hasn’t wondered exactly what the doctor is writing? As many as 50 million patients may have a chance to find out in the next few years, following the announcement this week of $10 million in new grants to expand the OpenNotes project, which works with medical providers to expand patient access to clinician notes...OpenNotes started in 2010 as a research project to examine what would happen if patients had easy access to their doctor’s visit notes, which may include a summary of their conversation, the symptoms patients describe and their doctor’s findings from a physical exam. Although patients have a legal right to their medical records, getting those documents is often difficult and expensive...Our goal is basically to make fully transparent medical records the standard of care across the country...The project works with providers, patient groups and electronic health record vendors, among others, to encourage the use of OpenNotes, and evaluates the impact of OpenNotes on health outcomes and costs...In addition to improving communication between patients and their providers, some research suggests OpenNotes may improve patients’ ability to stick with their medication regimens...

...World Health Organization wants to develop a fair pricing model for pharmaceuticals...the agency plans to convene governments, patient groups, and drug makers to fashion the notion into something realistic. The trick is to find the right balance between access to affordable medicines and enticing companies to develop new and improved medicines, while also ensuring lower-cost generics remain available. We spoke with Suzanne Hill, the secretary to the WHO Expert Committee on the Selection and Use of Essential Medicines, about how the agency hopes to get this done…

Pharmalot: This seems rather lofty or at least complicated. How is this supposed to get done?

Hill: We want to get evidence together that looks at the price drivers — how manufacturers set prices and what we know and don’t know about that. And we need to understand the questions surrounding R&D costs. We also want to understand the strategies that countries, payers, and health systems are using to manage prices and which ones may be effective and which aren’t. We also want to understand the return on investment that manufacturers need to ensure that supplies of medicines are continued...