As we begin the final quarter of 2022 and the leaves here on the east coast begin to turn and fall, it seems the clock may be running out on FDA and the Center for Drug Evaluation and Research (CDER) to meet its goal of …
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James E. Valentine
- Is Confirmatory Evidence Having a Moment?October 11th, 2022
- A Historic Day in Drug Development: FDA Approves Amylyx’s Drug to Treat ALS, Demonstrating FDA’s Application of Appropriate Flexibility in Rare DiseasesSeptember 30th, 2022
On September 29, 2022, FDA approved Amylyx’s NDA for its drug, Relyvrio (sodium phenylbutyrate/taurursodiol), for treatment of patients with amyotrophic lateral sclerosis (ALS) (see FDA announcement here). This approval decision charts a path for the exercise of appropriate flexibility in regulatory decisions for other rare …
- Let’s Listen to Patients: FDA Releases Final Guidance on Patient Engagement in the Design and Conduct of Medical Device Clinical StudiesMarch 1st, 2022
Our readers probably know that we value and advocate for patient engagement as an essential component in the development of medical products (see our firm’s commitment here). The COVID-19 pandemic cannot keep us from actively facilitating Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings with the Food …
- The FDA PDUFA VII Goals Letter (FY 2023-2027): A Review of Our Top 10 CommitmentsSeptember 21st, 2021
Few FDA publications are as eagerly anticipated in the drug development world as the twice-a-decade PDUFA Reauthorization Performance Goals and Procedures (hereinafter the “goals letter”). When FDA published the fiscal years (FY) 2023-2027 goals letter at the end of August, making known their commitments and …
- Critics Suggest FDA Approving Aduhelm Will Erode the “Public Trust”: What About Patients’ Trust?June 24th, 2021
For the last 13 years, this blogger has been at the center of what has now been dubbed “patient-focused drug development.” For 6 years, I served as a patient liaison within FDA in what was then called the Office of Special Health Issues. This was …
- FDA’s Accelerated Approval of Biogen’s Aduhelm for Alzheimer’s: A Sign of Applying the Emergency Use Standard Beyond COVID?June 8th, 2021
Yesterday’s FDA approval of Biogen’s Alzheimer’s drug, Aduhelm (aducanumab-avwa), is historic and is of a magnitude that it may be harbinger for future Agency actions, especially in neuropsychiatric conditions. This has been a decision we’ve been closely following. So much so, in November, right after …
- FDA Finalizes Guidance on Obtaining Agency Feedback on Combination Products; Highlights Best Practices for Meeting and Communicating with FDADecember 14th, 2020
On December 4, 2020, FDA finalized the guidance document, Requesting FDA Feedback on Combination Products (Guidance), which was issued to fulfill the requirement under Section 3038 of the Cures Act. As a reminder, Section 3038 contained several provisions to help facilitate FDA engagement and appropriate …
- FDA Credits Recent Drug Approval to Patient Community Engagement; We Applaud the Agency for the Recognition and Its Legacy of PFDDDecember 3rd, 2020
On November 23, 2020, the FDA announced that it had approved Alnylam Pharmaceuticals’ Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria 1 (PH1), a rare metabolic disorder that causes recurrent kidney stones and loss of kidney function. In the Agency’s press release, it credited …
- Shifting Patient Engagement in the Era of COVID-19: HPM Facilitates the First Four Virtual Externally-Led PFDD Meetings, Moderating the Fifth TodayAugust 28th, 2020
We all have had to adapt to a world with COVID-19 concerns. One of the challenges has been how to continue having Externally-Led Patient-Focused Drug Development (PFDD) meetings in these times of social distancing, travel restrictions, mask requirements, and other efforts to “flatten the curve”. On …
- FDA Works Around the Clock, Provides More Detailed Guidance on the Conduct of Clinical Trials Amidst the COVID-19 PandemicApril 1st, 2020
In the White House coronavirus updates, President Trump has commended FDA for working “around the clock” to expedite the review of new medical products to help with the testing and treatment of COVID-19. This is not puffery, as we at HP&M have experienced lightning fast …
- Conducting a Clinical Trial Amidst the COVID-19 Pandemic? FDA Issues Guidance to Help Sponsors with Decision-making to Overcome ChallengesMarch 18th, 2020
The COVID-19 pandemic has had a significant impact on day-to-day life for all of us in an attempt to “flatten the curve” to slow transmission of the coronavirus (SARS-CoV-2). However, for those of us involved in the development of medical products, quarantines, site closures, travel …
- Novel Clinical Trial Designs for Gene Therapies: An Exploration of Challenges by the National Academies’ Forum on Regenerative MedicineNovember 22nd, 2019
On November 13, 2019, the National Academies of Sciences, Engineering, and Medicine (NASEM) Forum on Regenerative Medicine hosted a workshop on Exploring Novel Clinical Trial Designs for Gene-Based Therapies. The Forum was tasked with exploring the design complexities and ethical issues associated with gene- and …
- FDA’s Latest PFDD Guidance Puts What Is Important to Patients at the Center of Drug Development. How? By Asking Them.October 11th, 2019
On October 1, 2019, FDA issued its second Patient-Focused Drug Development (PFDD) draft guidance – the next in a series of four methodological guidance documents, which discuss the collection of patient experience data to support decision-making related to drug development and approval (see some of …
- Everything Old is New Again: FDA Revises its 2014 Rare Pediatric Disease Priority Review Voucher Guidance for IndustryAugust 2nd, 2019
Developing drugs for kids with rare diseases is important work. If you would like to hear from one of these kids, check out this video: “My Philosophy for a Happy Life,” by the late Sam Berns. Offering priority review vouchers to sponsors that develop new drugs …
- HP&M Shares Experience with 16 Externally-Led Patient-Focused Drug Development Meetings; Summarizes Commonalities in Rare Disease Patient Perspectives in Comment to FDAMay 31st, 2019
On April 29, 2019, the Food and Drug Administration (FDA) convened a public meeting entitled “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities.” This meeting was attended by people with rare diseases, their caregivers and family members, patient advocates, industry representatives and …