News from Tuesday, October 19, 2021
Articles
The Future Of Value Assessment In A Post-Pandemic United States
(10/19, Donna Cryer, Health Affairs Blog) comments “..If we learn the right lessons from the COVID pandemic, the future of value assessment will be one that highly values people with—and without—serious, chronic conditions or disabilities as essential members of society. Value assessment will operate with the speed, flexibility and wisdom necessary to maximize that value. Achieving this future will require innovation, compromise, courage—and inclusion. To move forward, the people most affected by health care decisions—namely, patients of all colors and backgrounds—must be at the center of the process.” Full
The Key to Addressing Rising Health Care Costs Is Patient-Centered Evidence
(10/19, Nakela Cook, Morning Consult) comments “...What makes PCORI unique is its focus on the patient at the individual level. There is no one answer for everyone confronting a particular condition. What works for one person may not be the appropriate option for another. We must see patients as individuals with particular circumstances, needs and preferences — not as conditions to be treated. PCORI works closely with patients to understand their needs so that the work we fund is truly relevant to those it is intended to benefit.” Full
Failed Cancer Immunotherapies Cost Medicare Hundreds of Millions. How Should FDA Revamp Accelerated Approvals?
(10/19, Angus Liu, Fierce Pharma) reports “...Across six cancer indications for four medicines through the FDA’s accelerated approval program which have since been pulled, Medicare had spent $224 million between 2017 and 2019, a new study published in JAMA Internal Medicine shows. Describing the expenditure as ‘waste,’ the team of researchers from Harvard University and London School of Economics and Political Science suggests the FDA should ‘enforce timely completion of confirmatory trials and accelerated withdrawal’ if a drug fails to confirm its benefit.” Full
MedPAC's Part B Options Include ‘Value-Based' Payment During Accelerated Approval
(10/18, Michael McCaughan, Pink Sheet) reports “...The US Medicare Payment Advisory Commission's initial list of ideas to overhaul payment for drugs in the physician-administered Part B setting includes applying cost-effectiveness benchmarks for new drugs and biologics approved via the Accelerated Approval pathway... Among the menu of items brought forward by MedPAC staff, the proposal to use ‘value-based pricing’ for Accelerated Approval drugs has perhaps the most limited overall scope – but still bears watching as an idea that could move quickly through Congress in the context of potential efforts to reform the FDA pathway.” Paid Subscription Required
UK: A Test of NICE’s Resolve: Clinical Guidelines for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS)
(10/19, Leela Barham, PharmaPhorum) comments “...The incentives to follow NICE clinical guidelines are a little unclear. Unlike Technology Appraisals there’s no funding directive but this is a case where the recommendations are to do less, not more. Yet at the same time, the argument goes that the new guideline will be followed and that’s the issue for some clinicians: that GET and CBT when it does add clinical value won’t be offered to patients. The issue is therefore circular: NICE won’t publish because they don’t believe it will be implemented, some clinicians don’t want it to be published because they believe it will be implemented. But perhaps it also signals a degree of panic within NICE. The decision to pause is perhaps understandable, but to do so the day before the final guidance was due to come out suggests leaving that tricky decision too late.” Full
Press Releases
Analysis Group's Real-World Data Study Supports FDA's Recent Exkivity™ (Mobocertinib) Approval
(10/19, Analysis Group Press Release) “...‘The FDA's acceptance of RWD and real-world evidence (RWE) in approvals is changing how pharma thinks about R&D,’ said Analysis Group Managing Principal Mei Sheng Duh, M.P.H., Sc.D. ‘Takeda is at the fore of this understanding, and retained us to collaborate on the plan of the RWD study after its end of Phase 1 Type B meeting with the FDA to ensure the RWD study would align with the single-arm trial in the regulatory submission. The RWD provides contemporaneous natural history information and a benchmark of current treatment. The totality of evidence supports the clinical trial outcome for regulatory consideration.’” Full
Journals
Estimated Medicare Spending on Cancer Drug Indications With a Confirmed Lack of Clinical Benefit After US Food and Drug Administration Accelerated Approval
Mahnum Shahzad, BA, et al.
October 18, 2021, JAMA Internal Medicine