As is the tradition here at Health Law & Policy Matters, towards the end of the year we take stock of what transpired in our respective industries and highlight important legal, regulatory, and business developments. For those of us who monitor the Food and Drug Administration (FDA or the Agency) and counsel FDA-regulated entities, it has certainly been a whirlwind of a year.<\/p>\n\n\n\n
2017 began with no clear picture of who would be assuming leadership of the Agency, but also with a brand-new piece of critically important (and bipartisan!) legislation \u2013 the 21st<\/sup>\u00a0Century Cures Act (see our prior posts\u00a0here<\/a>) \u2013 which imposed new obligations and authorities on FDA that needed to be implemented, operationalized, and fully funded.\u00a0 In early May, Dr. Scott Gottlieb was sworn in as the 23rd<\/sup>\u00a0Commissioner of FDA and he moved quickly to shift policy priorities in almost every area that the Agency regulates, a goal that in some ways was made more efficient due to the concurrent timing of modernization mandates imposed by the Cures Act.\u00a0 This year\u2019s must-pass User Fee Act, the FDA Reauthorization Act or \u201cFDARA\u201d (see our prior posts\u00a0here<\/a>) engendered some hand-wringing and political drama over the summer, but was ultimately passed by Congress in August and signed into law.\u00a0 FDARA includes some important policy and programmatic changes for new prescription drugs\/biologics, generic drugs, and biosimilars, but it did not contain major wholesale reforms to the Agency\u2019s authorities due to the very recent passage of the Cures Act.<\/p>\n\n\n\n This is the first in a series of three installments that will review the actions FDA took in 2017, reflect on what they may mean for regulated industry, and provide a few predictions for 2018. This first installment, which will be broken up into two posts, focuses on therapeutic products; that is, drugs, biologics, human cells and tissue products, and gene therapies. Our subsequent posts will focus on medical devices and diagnostics, including whole-genome sequencing tests, and digital health and other software-related developments, respectively.<\/p>\n\n\n\n FDA\u2019s Drug Competition Action Plan = More Generics and Biosimilars to Market<\/em><\/strong><\/p>\n\n\n\n It will come as no surprise to our regular readers\u00a0\u2013 or to anyone who reads anything for that matter\u00a0\u2013\u00a0that high drug prices are a bipartisan \u201cthing\u201d right now and it is all hands on deck to fix the problem.\u00a0 From even before Day 1, given his prior scholarship on this issue, Commissioner Gottlieb was expected to shake things up at FDA and to use existing legal authorities more actively and creatively to foster greater competition; reduce unnecessary regulatory barriers to market; and generally help get generic and biosimilar markets working better for consumers \u2013\u00a0i.e.<\/em>, by lowering overall drug costs.\u00a0 Since we\u2019re only six months into implementation of Dr. Gottlieb\u2019s multi-pronged, self-named Drug Competition Action Plan, it remains to be seen whether he achieves that bottom-line impact on the markets.\u00a0 But without a doubt, Agency leadership has been taking bold and decisive actions towards that goal, several of which we blogged about earlier in the year (see\u00a0he<\/a>r<\/a>e<\/a>\u00a0and\u00a0here<\/a>).\u00a0 Many new guidance documents have been issued for Abbreviated New Drug Application (ANDA) applicants, including a\u00a0Draft Guidance on Formal Meetings Between FDA and ANDA Sponsors of Complex Products<\/a>\u00a0(think EpiPen) intended to expedite the development of such complex products.\u00a0 These efforts are very much welcomed by the generic drug industry, who negotiated significant changes to the Generic Drug User Fee Act as part of its first reauthorization cycle with FDARA.<\/p>\n\n\n\n FDA also hosted its own\u00a0public meeting<\/a>\u00a0in July to discuss whether its administration of the Hatch-Waxman Amendments, which created today\u2019s marketing pathway for generic drugs, is striking the right balance between innovation and access to lower-cost alternative treatments.[1]<\/sup><\/a>\u00a0Then in November, Commissioner Gottlieb participated in a Federal Trade Commission workshop on the issue of competition on prescription drug markets (see\u00a0here<\/a>).\u00a0 And as promised back in June when it published the first iteration of this list, FDA released on December 15th<\/sup>\u00a0an\u00a0update<\/a>\u00a0to its new \u201cList of Off-Patent, Off-Exclusivity Drugs without an Approved Generic.\u201d\u00a0 (We haven\u2019t compared this list to June list to see what happened to the numbers, but are definitely interested to see that analysis!)<\/p>\n\n\n\n Intriguingly, speaking at a conference in Washington, D.C. on December 5th<\/sup>, Dr. Gottlieb hinted at a significant policy announcement being planned for the beginning of 2018 on the topic of increasing competition in markets for biological products.\u00a0 FDA has continued to make progress in its implementation of the Biologics Price Competition and Innovation Act (BPCIA), which created the formal marketing pathway for biosimilar products, having approved 4 biosimilar applications this year (including the first biosimilar treatment for cancer, see\u00a0here<\/a>).\u00a0 The Agency also got the Biosimilar User Fee Act reauthorized as part of FDARA; issued a new draft guidance on\u00a0Statistical Similarity to Evaluate Analytical Similarity<\/a>; and has recently updated its online BPCIA public resources and provider training materials in a pretty nice way.\u00a0 But since the Obama Administration issued the long-awaited draft guidance on demonstrating\u00a0Interchangeability with a Reference Product<\/a>\u00a0back in mid-January, it seems fair to say that this year most of the BPCIA action came out of the\u00a0Supreme Court<\/a>\u00a0and other federal courts.\u00a0 So we will be watching for Dr. Gottlieb\u2019s promised policy announcement on biologics and biosimilars in early 2018.<\/p>\n\n\n\n Breakthrough Therapy and Other Expedited Development\/Approval Pathways on the Rise<\/em><\/strong><\/p>\n\n\n\n Another exciting trend in the therapeutics space this year was the pace and revolutionary nature of newly approved, first-in-class drug and biological therapies. As of November 30, 2017, the Agency had approved 40 new molecular entities (NMEs) this calendar year and 17 of those were for orphan (rare disease) indications. This number is very high for NME approvals and \u2013 given that December is not included in the total \u2013 2017 could be a historic year for this important FDA metric. The existing record for single-year NME approvals is from 1996 (53 total), which may have been somewhat of an outlier year; 2015 holds the second-place record with 45 such approvals and, after a lackluster 2016, it would be exciting if this year comes in above or on par with 2015.<\/p>\n\n\n\n In addition, 17 of those 40 NMEs had been designated and developed as Breakthrough Therapies \u2013 the highest annual approval count to date since the Breakthrough program was created by Congress as part of the 2012 user fee-reauthorization cycle. 28 of the NMEs had been reviewed under Priority Review, and 18 were designated for the Fast-Track Program, which also represents a record annual number for fast-track. It\u2019s also worth noting that the majority of these innovative products took advantage of more than one expedited program, with over 2\/3 of this year\u2019s drug and biologic (NDA and BLA) approvals using more than one.<\/p>\n\n\n\n [Note: These numbers are taken from a year-end presentation made by Patrick Frey from the FDA Office of New Drugs, available on the FDA website\u00a0here<\/a>.\u00a0 But it is worth adding that as of December 15th<\/sup>, there have been 42 NMEs approved according to\u00a0the public Agency count<\/a>.]<\/p>\n\n\n\n Besides the sheer number of new drug and first-in-class approvals, several incredible medical advances were approved by FDA and commercially launched this year.\u00a0 These include the first two approved CAR-T therapies, in which a patient\u2019s own immune cells are re-engineered using gene therapy tools to target the patient\u2019s individual cancer, one for children and one for adults (see\u00a0here<\/a>\u00a0and\u00a0here<\/a>).\u00a0 CAR-T represents a transformative treatment for cancer patients, and the two newly approved products also represent some of the first personalized cell therapies to get to market.\u00a0 Another ground-breaking approval from 2017 was the\u00a0first cancer treatment to target a specific biomarker<\/a>\u00a0rather than a particular organ where tumors originated, meaning it can be used to treat patients with many different forms of cancer as long as the underlying DNA defect causing the cancer meets the appropriate profile.\u00a0 Much of the success of these revolutionary treatments within the U.S. regulatory process, especially how quickly they were reviewed by FDA, can likely be attributed to the new Oncology Center of Excellence (OCE).\u00a0 OCE has proven so successful in its first year of operation that Commissioner Gottlieb has recently been reported to be mulling the possibility of extending the model to other areas, like neurological disease.<\/p>\n\n\n\n Other disease states also benefited from positive developments in 2017, such as the cystic fibrosis community, which saw the first new CF drug approved by FDA in almost 20 years.<\/p>\n\n\n\n The second part of our drug-specific installment will discuss the Regenerative Medicine Advanced Therapies (RMAT) designation, as well as additional observations regarding enforcement efforts in the drugs and biologics space.<\/p>\n\n\n\n Footnotes As is the tradition here at Health Law & Policy Matters, towards the end of the year we take stock of what transpired in our respective industries and highlight important legal, regulatory, and business developments. For those of us who monitor the Food and Drug Administration (FDA or the Agency) and counsel FDA-regulated entities, it … Read more<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_generate-full-width-content":"","footnotes":""},"_links":{"self":[{"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/pages\/4417"}],"collection":[{"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/comments?post=4417"}],"version-history":[{"count":1,"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/pages\/4417\/revisions"}],"predecessor-version":[{"id":4418,"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/pages\/4417\/revisions\/4418"}],"wp:attachment":[{"href":"https:\/\/www.healthlawpolicymatters.com\/wp-json\/wp\/v2\/media?parent=4417"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}
[1]<\/a>\u00a0For readers interested in viewing the public comments to this request for comments, you can go to the electronic docket\u00a0here<\/a>\u00a0and also the meeting page linked above for public presentations during the July 18, 2017 meeting.\u00a0 We expect ongoing policy and procedural changes by the Agency in 2018 as it continues to review public submissions to this meeting notice, which were not due until mid-November following a comment period extension.<\/p>\n","protected":false},"excerpt":{"rendered":"