Generic Industry’s Future May Include A Role In The Gene Therapy Space
Executive Summary
Trade association’s CEO says a new strategic planning initiative will consider among other things how gene therapy could fit into the generic industry business model.
Personalized medicine does not immediately fit into the generic industry model of high-volume and low prices, given its goal of providing tailored treatments for a single patient. Still, Association for Accessible Medicines president and CEO Dan Leonard wants the industry to consider its options.
During his 27 May State of AAM address as part of the generics trade association’s annual meeting, Leonard announced the start of a strategic planning initiative to discuss the industry’s role in gene therapy, personalized medicine, and the rest of the pharmaceutical landscape going forward.
“Now that we can see on the horizon an era in which hyper-personalized medicines like gene therapies will become commonplace, what role will our industry play in shaping that era?” he asked. “What does that even mean in a future where therapies are tailored to each individual patient? How do we go about generating competition and serving our purpose in that market?”
Leonard took over as head of AAM in August 2020 after several years in charge of the National Pharmaceutical Council. (Also see "New AAM CEO: Traditional Lobbying On Lowering Health Care Costs Can Mix With COVID-19 Talks" - Pink Sheet, 6 Nov, 2020.)
Dan Leonard
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In addition to setting the industry direction, the strategic plan also will identify policies and issues that must be raised and dealt with in the near-term, such as patient access to the medicines.
“What problems and policies do we need to think about and work on today to ensure that access to these therapies is possible, not just for the elite, but for everyone who needs them?” Leonard said.
Indeed, access to gene therapy may be among the most pressing issues facing the sector. The products can be a one-time curative therapy, but also could carry a $1m price tag. Payers have been preparing for a boom in cell and gene therapy approvals, as well as the associated costs. (Also see "Payers Must Brace For Coming Wave Of Cell And Gene Therapies " - Scrip, 14 Oct, 2019.)
The US Food and Drug Administration’s Center for Biologics Evaluation and Research is expected to receive additional funds in the upcoming prescription drug user fee reauthorization to hire more staff and upgrade IT systems to handle the oncoming wave of applications. (Also see "PDUFA VII Negotiations Completed, Commitment Letter Ratification Ongoing" - Pink Sheet, 22 Mar, 2021.)
The industry strategy also could chart a strategy to deal with the potential patent issues that may emerge. The industry continues to battle with brand companies over biologics patents. (Also see "US Patent Reform Legislation Threatened By Biden Support For Vaccine IP Waiver, Republicans Say" - Pink Sheet, 18 May, 2021.)
Complex Products Savings Waiting To Be Unlocked, Leonard Says
Leonard also highlighted a more immediate opportunity for the industry, the complex product sector, where he said there is enormous potential still to be unlocked.
Generics sponsors have struggled to gain approval for many complex products amid bioequivalence and device questions, in addition to the FDA assessment process, which Leonard said is delaying lower-cost generics from market entry.
Reforms have come through the generic drug user fee program, including the pre-ANDA meeting process. Sponsors have called for additional transparency and predictability in the assessment system and are pushing for more changes in GDUFA III to speed approvals. (Also see "US FDA’s Drug Shortage Program On ‘Site Engagement’ To Be Nixed In GDUFA III" - Pink Sheet, 12 May, 2021.)
However, FDA officials have said the current process already is primed to make the complex generic process more collaborative. (Also see "Complex Generics: US FDA, Sponsors View Roadblocks Very Differently" - Pink Sheet, 9 Feb, 2021.)
Familiar Problems Still Lingering
Leonard also raised several problems that industry veterans are used to hearing about at trade group meetings. Generic sponsors continue to struggle with efforts to limit 180-day exclusivity of small-molecule products and wholesaler consolidation, as well as slow biosimilar uptake.
The issues have plagued the generics industry for several years. The effort to change 180-day exclusivity rules to eliminate system gaming emerged during the Trump Administration. The House Energy and Commerce Committee moved the bill to the full House of Representatives in 2019, where it stalled. (Also see "AAM Urges Congress To Re-evaluate Blocking Act" - Generics Bulletin, 13 Jan, 2020.)
Industry consolidation also is a well-known problem. (Also see "At US Generic Association Annual Meeting, Worries About Sustainability" - Pink Sheet, 12 Feb, 2018.)
Biosimilar uptake continues to be slow-going, although optimism is increasing with the launches of multiple biosimilars of AbbVie Inc.’s blockbuster biologic Humira (adalimumab) expected in 2023. (Also see "AbbVie’s Stalling Tactics On Humira Pay Off – In More Ways Than One" - Pink Sheet, 20 May, 2021.)
Supplement approval improvements and other changes have been proposed for the upcoming biosimilar user fee reauthorization to help the industry grow in the US. (Also see "New Supplement Timelines, Categories Proposed For BsUFA III" - Pink Sheet, 27 Apr, 2021.)