Keeping pace with the ever-changing life sciences industry, Morrison Foerster stays on top of the trends and political, legal, and economic drivers to support striking the balance between innovative discovery and purposeful business planning to assure our clients continued success. MoFo Life Sciences shares timely legal and business insights and in-depth analyses on trends and the complex technologies at the heart of the global pharmaceutical, biotechnology, diagnostics, medical devices, digital health, and AgTech fields.Learn more about our <a href="https://www.mofo.com/capabilities/life-sciences-healthcare" target="_self">Life Sciences practice</a>.<h2 dir="ltr">ABOUT MORRISON FOERSTER</h2>Morrison Foerster is a leading global law firm that transforms complexity into advantage for its clients. Our clients include some of the largest financial institutions, banks, consulting and accounting firms, and Fortune 100, technology, and life sciences companies. Highlighting the firm’s commitment to client service, leadership in market-changing deals and impact litigation, and values-based culture, Morrison Foerster has been repeatedly recognized as one of the top 10 firms on The American Lawyer’s A-List. Year after year, the firm receives significant recognition from Chambers and The Legal 500 across their various guides, including Global, USA, Asia‑Pacific, Europe, UK, Latin America, and FinTech Legal. Our lawyers passionately care about delivering legal excellence while living our values. Morrison Foerster has a long-standing commitment to creating a culture that respects and celebrates differences, while providing an inclusive environment. The firm has achieved Mansfield Certification Plus since 2018 as a result of successfully reaching at least 30 percent women, communities of color, and LGBTQ+ lawyer representation in a notable number of current leadership roles and committees. The firm also has a long history of commitment to the community and society through providing pro bono legal services, including litigating for civil rights and civil liberties, improving public education and fostering the wellbeing of children, advocating for veterans, promoting international human rights, enforcing the right to asylum, and safeguarding the environment.

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Dr. Meghan Poon counsels clients on patent preparation and prosecution, portfolio strategy, and IP due diligence in the areas of biotechnology and biologics. She has significant experience in diverse technologies including antibodies, cell therapies, gene therapies, diagnostics, and gene-edited plants. She represents clients ranging from large international pharmaceutical companies to midsized companies, startups, and universities.Meghan has filed numerous Patent Term Extension applications to secure additional term for her clients’ patents, which cover FDA-approved products. In addition, Meghan has extensive experience addressing complex obviousness-double patenting and subject matter eligibility issues impacting her clients’ patent portfolios. Meghan also represents clients in both investor-side and target-side due diligence, in deals from seed funding to initial public offerings.Prior to law school, Meghan attended graduate school at the University of California, Los Angeles, where she received a Ph.D. in biological chemistry based on her work on protein-protein and protein-DNA interactions at a molecular level. Meghan is the author of several scientific publications and has presented her research at international conferences.Meghan received her J.D. from the University of Southern California Law School and spent a year as a visiting student at Santa Clara University, where she focused on intellectual property.

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Dr. Meghan Poon counsels clients on patent preparation and prosecution, portfolio strategy, and IP due diligence in the areas of biotechnology and biologics. She has significant experience in diverse technologies including antibodies, cell therapies, gene therapies, diagnostics, and gene-edited plants.

Meghan Poon

Wolfgang advises clients on contentious and non-contentious matters involving their most valuable intellectual property assets in life sciences, digital, and high-tech industries.His subjects of focus include research and development collaborations, complex licensing agreements, and other technology transactions, as well as know-how protection strategies, and employee invention law. He also has substantial experience in litigating patents, trademarks, and other intellectual property assets, often in a multijurisdictional context. In addition, Wolfgang and his team frequently clear trademarks and ad campaigns and prosecute trademark applications.Wolfgang is co-editor of our <a href="https://lifesciences.mofo.com/" dir="ltr" target="_blank">MoFo Life Sciences Blog</a>.Wolfgang is a partner of Morrison Foerster based in our German office in Berlin.<h4 dir="ltr">Representative Transactional Matters</h4><ul dir="ltr"><li>U.S.-based BioTech fund on various research collaborations and other transactions with German start-ups or research institutions.</li><li>Negotiate multimillion-euro strategic partnership between two AgTech leaders concerning the development and commercialization of pest-resistant transgenic traits.</li><li>Craft global multi-million collaboration regarding AI-powered robotic MaaS.</li><li>UK-based digital health company on product roll-out of RPM applications in Germany; coordinate international expansion.</li><li>Advise numerous German start-ups on their U.S. expansion.</li><li>Draft diverse global patent and know-how license agreements in the areas of AgTech and life sciences—including gene licensing and licensing of genome editing technologies (e.g., CRISPR)—and medical devices; draft correlating supply and distribution agreements.</li><li>Structure IP rights in connection with joint ventures and other M&A transactions, including the carve-out of a patent-heavy semiconductor business comprising nine jurisdictions into a German–U.S. joint venture.</li><li dir="ltr">Advise on government-funded, multi-partner R&D projects and/or R&D projects with academic institutions, as well as on employee invention law.</li></ul><h4 dir="ltr">Representative Contentious IP Matters</h4><ul dir="ltr"><li>Lead counsel in an international dispute between software companies on rights and obligations following termination of a multi-million licensing and distribution arrangement; coordinate local counsel.</li><li>Defend Japanese chip manufacturer in high-profile SEP-litigation; advise on global settlement arrangement.</li><li>Represent leading internet retailer in patent infringement and concurrent invalidity proceedings in the context of a multijurisdictional dispute concerning touch-screen technology.</li><li>Represent diverse pharmaceutical and life sciences companies in national and cross-border patent infringement proceedings and other technology-related disputes, including preliminary injunctions as well as pre‑trial discovery and inspection proceedings, concerning, inter alia, drug-coating technologies, toner cartridges, medicinal gases, transgenic plants, and vaccines.</li><li dir="ltr">Conduct trademark, copyright, and false advertising litigation, including, most notably, a high-profile trademark litigation for a German bank, and a nine-country cross-border trademark litigation involving a famous pharmaceutical brand.</li></ul>

Dr. Wolfgang Schönig berät Mandanten zum Schutz des geistigen Eigentums sowohl in streitigen als auch nichtstreitigen Angelegenheiten, mit einem besonderen Fokus auf die IP-rechtliche Beratung von Unternehmen aus den Bereichen Life Sciences, Digitalwirtschaft und High-Tech.Zu seinen Beratungsschwerpunkten gehören Forschungs- und Entwicklungskooperationen, komplexe Lizenzvertragsgestaltungen und andereTechnologie-Transaktionen, sowie Know-how-Schutz-Strategien und das Arbeitnehmererfinderrecht. Er verfügt zudem über umfangreiche Prozesserfahrung zur Durchsetzung von Patenten, Marken- und anderen IP-Rechten, häufig auch in einem grenzüberschreitenden Kontext. Darüber hinaus bewertet Dr. Wolfgang Schönig zusammen mit seinem Team regelmäßig die Rechtskonformität von Werbekampagnen, prüft die Verfügbarkeit von Marken und entwickelt entsprechende Anmeldestrategien.Dr. Wolfgang Schönig ist Co-Editor des Life Sciences Blogs von Morrison Foerster.Er hat seinen Doktortitel an der Heinrich-Heine-Universität Düsseldorf und seinen LL.M. an der Fordham School of Law in New York erworben, jeweils mit Schwerpunkt im gewerblichen Rechtsschutz. Seinen MBA-Abschluss erhielt er von der European University in Barcelona.Dr. Wolfgang Schönig ist Partner des deutschen Büros von Morrison Foerster in Berlin.<h4 dir="ltr">Repräsentative Transaktionen</h4><ul><li dir="ltr">BioTech-Fonds mit Sitz in den USA bei verschiedenen Forschungskooperationen und anderen Transaktionen mit deutschen Start-ups oder Forschungseinrichtungen</li><li dir="ltr">Verhandlung einer hochvolumigen strategischen Partnerschaft zwischen zwei Technologieführern im Bereich Crop Science betreffend der Entwicklung und Kommerzialisierung von bestimmten Resistenzen</li><li dir="ltr">Gestaltung einer globalen Multi-Millionen-Zusammenarbeit für KI-gestützte Roboter</li><li dir="ltr">Ein in Großbritannien ansässiges Unternehmen für digitale Gesundheit bei der Markteinführung von RPM-Anwendungen in Deutschland; Koordination der internationalen Expansion</li><li dir="ltr">Beratung zahlreicher deutscher Start-ups bei deren US-Expansion</li><li dir="ltr">Strukturierung und Verhandlung diverser Patent- und Know-How-Lizenzen in den Bereichen Ag Tech und Life Sciences – einschließlich Fragen zu genome editing wie CRISPR-Medizingeräte, sowie Gestaltung von Softwarelizenzverträgen und Erstellung korrespondierender Liefer- und Vertriebsvereinbarungen</li><li dir="ltr">Strukturierung von IP-Rechten im Zusammenhang mit Joint Ventures und anderen M&A-Transaktionen, darunter die Koordination eines neun Länder umfassenden IP Carve-Outs im Rahmen eines Halbleiter-JVs</li><li dir="ltr">Beratung zu staatlich geförderten Multi-Partner F&E-Projekten und/oder F&E-Projekten mit akademischen Einrichtungen sowie zum Arbeitnehmererfindungsrecht</li></ul><h4 dir="ltr">Repräsentative IP-Streitigkeiten</h4><ul><li dir="ltr">Lead Counsel in einem internationalen Rechtsstreit zwischen Softwareunternehmen über Rechte und Pflichten nach Beendigung einer millionenschweren Lizenz- und Vertriebsvereinbarung; Koordinierung der lokalen Anwälte</li><li dir="ltr">Verteidigung eines japanischen Chipherstellers in einem prominenten Streitfall bezüglich Standardessentieller Patente (SEP); Beratung zu einer globalen Vergleichsregelung</li><li dir="ltr">Verteidigung einer führenden Internetplattform gegen Ansprüche eines Patenttrolls wegen vermeintlicher Verletzung von Patent bezüglich Touchscreen-Technologie sowie korrespondierende US-Streitigkeiten</li><li dir="ltr">Vertretung von Pharma- und Life Science-Unternehmen in nationalen und grenzüberschreitenden Patentverletzungsverfahren und anderen technologiebezogenen Streitigkeiten, einschließlich einstweiliger Verfügungsverfahren und vorprozessualer Beweissicherungs- und Besichtigungsverfahren, unter anderem in Bezug auf Arzneimittelbeschichtungstechnologien, Tonerkartuschen, medizinische Gase, transgene Pflanzen und Impfstoffe</li></ul>

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Wolfgang Schönig is a partner of Morrison Foerster (International) LLP based in our German office in Berlin. His practice specialty is advising on IP aspects relative to transactions in, among others, the life sciences, AgTech and pharmaceuticals industry, as well as legal representation in IP disputes. Wolfgang is specialized in research and development collaborations, complex licensing agreements, technology acquisitions, know-how protection strategies, and employee invention law.

Wolfgang Schönig focuses his practice on IP aspects relative to transactions in the digital, telecoms, high tech, life sciences, and pharmaceuticals industry, as well as legal representation in IP disputes.

Wolfgang Schönig advises clients on IP aspects relating to transactions in, among others, the life sciences, AgTech and pharmaceuticals industry, as well as legal representation in IP disputes. His additional focus areas include research and development collaborations, complex licensing agreements, technology acquisitions, know-how protection strategies, and employee invention law.

Wolfgang Schönig

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The Consolidated Appropriations Act for 2023 was signed into law on December 29, 2022 and includes the Food and Drug Omnibus Reform Act (FDORA).  FDORA authorizes a variety of new and important amendments to the Food, Drug, and Cosmetic Act (FDCA) governing drugs, medical devices, and clinical trials, as well as cosmetics and other personal care products.  FDORA enacts many of the amendments to the FDCA that were previously drafted and negotiated as part of Congress’s reauthorization of the U.S. Food and Drug Administration’s (FDA) user fee programs.  Some key provisions of FDORA are summarized below.Generally Applicable Reforms<ul><li>Clinical Trial Diversity – Several FDORA provisions are aimed at addressing the need for racial, ethnic, gender, and social diversity in human clinical research.  Previously, while FDA had encouraged and sometimes requested that sponsors enroll more diverse patient populations in late-stage clinical trials of FDA-regulated products, it did not have the authority to mandate such measures.  FDORA requires sponsors to submit “diversity action plans” for phase 3 clinical trials or other pivotal studies for drugs and devices, unless otherwise waived or exempted.  The action plans must include the sponsors’ goals for enrollment, the underlying rationale, and an explanation of how the sponsor plans to achieve them.  FDA is tasked with issuing new guidance on diversity action plans and hosting at least one public workshop to solicit input from industry stakeholders on increasing diversity in enrollment and encouraging clinical study participation that reflects the prevalence of the disease among demographic subgroups.  The public workshop will take place sometime in 2023.</li><li>Real-World Data and Real-World Evidence (RWD/E) – FDORA requires FDA to issue guidance on considerations for the use of RWD/E to support regulatory decision-making for drugs and devices.  FDA must also conduct public workshops related to development of endpoints for rare diseases, which may include, “as appropriate, the use of [RWD/E] to support the validation of efficacy endpoints . . . for rare diseases.”</li><li>Pre-approval Communications with Payors – FDORA provides that both drug and device manufacturers are permitted to share certain information with payors, formulary committees, and similar entities regarding investigational products and investigational uses of approved products.  This amendment codifies FDA’s existing policy on pre-approval communications with payors that was detailed in its<a href="https://www.fda.gov/media/133620/download" target="_blank"> Guidance for Industry on Drug and Device Manufacturer Communications with Payors, Formulary Committees, and Similar Entities</a> (June 2018).</li></ul>Drug Reforms<ul><li>Accelerated Approval – FDA requested various updates to the language in the FDCA authorizing the agency to grant accelerated marketing approval to certain products for serious or life-threatening disease or conditions.  FDORA amends FDA’s accelerated approval process for drugs and biologics by:</li><ul><li>Allowing FDA to require the sponsor to have confirmatory clinical trials underway before accelerated approval is awarded.</li><li>Requiring sponsors of products granted accelerated approval to submit progress reports for post-approval studies to FDA every six months.  Information from the progress reports must be publicly available on the FDA website.</li><li>Giving FDA the option to use expedited procedures to withdraw approval if the confirmatory trail fails to verify the product’s clinical benefit.</li><li>Requiring FDA to post the rationale for any decision not to require a post-approval study in connection with an accelerated approval.</li><li>Ordering the establishment of an intra-agency Accelerated Approval Council with senior official members from various FDA centers and divisions, including the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), to “ensure the consistent and appropriate use of accelerated approval” across the agency by engaging directly with product review teams which may include providing training and developing guidance.  The Council must meet at least three times a year and publish an annual report detailing its activities.</li></ul></ul><ul><li>Bioresearch Monitoring (BIMO) Inspections – FDORA explicitly permits FDA inspection of facilities involved in the preparation, conduct, or analysis of clinical and non-clinical studies submitted to FDA, in addition to other persons holding study records or otherwise involved in the study process, such as contract research organizations and institutional review boards.  FDA must issue draft guidance on this additional inspection authority within 18 months of FDORA enactment.</li><li>Classification of Certain Products as Drugs – A provision in FDORA clarifies that any contrast agent, radioactive drug, or over-the-counter monograph drug is classified as a drug and not a device.  This provision was in direct response to Genus Medical Technologies, LLC v. FDA, 994 F.3d 631 (D.C. Cir. 2021), in which the court found that FDA lacked discretion to regulate a product meeting the statutory definition of a device as a drug.  Nonetheless, there may still be examples of other products that have been traditionally regulated as drugs but should be regulated as devices that are not included in these products that are deemed to be drugs.</li></ul>Medical Device Reforms<ul><li>Cybersecurity of Devices – FDORA imposes new premarket submission requirements for devices that meet the definition of a “cyber device” which must now include cybersecurity information, including a software bill of materials and plan to address cybersecurity vulnerabilities.  “Cyber devices” are defined as those that include software (including SAMD), have the ability to connect to the internet, and contain any such technological characteristics that could be vulnerable to cybersecurity threats.  FDA has the authority to identify devices or categories of devices that are exempt from the requirements.  FDORA also amends the list of prohibited acts in the FDCA to include failure to comply with these cybersecurity requirements.</li><li>Predetermined Change Control Plans – FDORA amends the FDCA to provide that predetermined change control plans may be approved in premarket applications and supplements or cleared under premarket notifications (510(k)).  If a predetermined change control plan is approved or cleared, then a change to a device that is consistent with such approved or cleared plan does not require submission of a supplemental PMA or new 510(k).</li><li>Device Bans for Specific Uses – FDORA expands FDA’s authority to ban medical devices under section 516 of the FDCA to allow FDA to ban specific intended uses.  This provision is enacted in response to the decision in Judge Rotenberg Educ. Ctr., Inc.v. FDA, 3 F.4th 390 (D.C. Cir. 2021), which held that FDA could not ban individual intended uses of an otherwise legally marketed device.</li></ul>Cosmetic and Consumer Product ReformsAs detailed in a previous <a href="https://lifesciences.mofo.com/topics/congress-expands-fda-authority-over-cosmetics-regulation?" target="_blank">blog post</a>, FDORA included the Modernization of Cosmetics Regulation Act (MoCRA), which strengthens FDA’s authority over the regulation of cosmetic products.  MoCRA introduces a comprehensive structure that implements regulatory requirements for responsible parties involved in the manufacture of cosmetic products, including facility registration, product and ingredient listing, serious adverse event reporting, compliance with product labeling requirements, and maintenance of records substantiating product safety.  MoCRA also tasks FDA with issuing regulations to establish good manufacturing practices for cosmetics and standardized testing methods for detecting asbestos in talc-containing cosmetic products.  In the prior blog post, the MoFo FDA + Healthcare Regulatory team discusses the specifics of the cosmetics regulation reforms and FDA’s expanded authority.In addition to MoCRA, FDORA also included the INFORM (Integrity, Notification, and Fairness in Online Retail Marketplaces) Consumer Act in an effort to deter online sale of counterfeit goods.  Among other things, the INFORM Consumers Act requires online retail marketplaces that include third-party sellers of consumer products to verify the identity of “high-volume third-party sellers” by authenticating the seller’s government ID, tax ID, bank account information, and contact information.Notable OmissionsWhile many of the policy riders left out of the user fee reauthorization legislation were included in FDORA, it is important to note that Congress did not reach agreement for some other key regulatory reform measures in time for inclusion as part of the omnibus bill.VALID Act – The Verifying Accurate Leading-edge IVCT Development (VALID) Act would have rewritten FDA regulation of in vitro diagnostics (IVDs), redefining them as in vitro clinical tests (IVCTs), a new category of medical device products that includes both laboratory-developed tests (LDTs) and in vitro diagnostics.  The VALID Act would have conferred on FDA explicit statutory authority over LDTs for the first time and subjected this product category to requirements for registration and listing, premarket review and approval, adverse event reporting, and related processes for FDA oversight.Product Listing for Dietary Supplements – The original Senate version of the user fee reauthorization bill also contained provisions establishing product listing requirements for dietary supplements that would require dietary supplement manufacturers, distributors, and packers to list product information with FDA.  The provisions would require FDA to provide unique listing numbers for each listed supplement and conduct facility inspections for high-risk facilities, suppliers, and dietary supplement types.

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Brigid is a food, drug, and medical device lawyer who counsels life sciences companies on a wide range of U.S. Food & Drug Administration pre- and post-market regulatory issues.

Brigid DeCoursey Bondoc

Brigid is a food, drug, and medical device lawyer who counsels life sciences companies on a wide range of U.S. Food & Drug Administration pre- and post-market regulatory issues.

Brigid Bondoc

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FDORA Passage Brings Significant Changes to FDA’s Drug, Device, and Cosmetic Authorities

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The Consolidated Appropriations Act for 2023 was signed into law on December 29, 2022 and includes the Food and Drug Omnibus Reform Act (FDORA).  FDORA authorizes a variety of new and important amendments to the Food, Drug, and Cosmetic Act (FDCA) governing drugs, medical devices, and clinical trials, as well as cosmetics and other personal care products.  FDORA enacts many of the amendments to the FDCA that were previously drafted and negotiated as part of Congress’s reauthorization of the U.S. Food and Drug Administration’s (FDA) user fee programs.  Some key provisions of FDORA are summarized below.

FDORA Passage Brings Significant Changes to FDA’s Drug, Device, and Cosmetic Authorities

On September 30, 2021, the U.S. Food and Drug Administration (FDA) <a target="_blank" href="https://www.federalregister.gov/documents/2021/09/30/2021-21324/interpreting-sameness-of-gene-therapy-products-under-the-orphan-drug-regulations-guidance-for">announced</a> final guidance titled “<a target="_blank" href="https://www.fda.gov/media/134731/download">Interpreting Sameness of Gene Therapy Products under the Orphan Drug Regulations</a>.”  The guidance finalizes the January 2020 draft guidance and provides FDA’s current perspective on certain criteria that help determine sameness of human gene therapy products for orphan drug designation and exclusivity purposes.  In the Federal Register notice announcing the final guidance, FDA noted that it received comments on the draft guidance that “generally supported the approach described in the guidance.”  FDA considered these comments and requests for additional clarification when finalizing the guidance, “adding clarification and examples, as feasible.”<h4>Background</h4>The Orphan Drug Act (ODA) seeks to incentivize the development of drugs for rare diseases, defined in the ODA as those affecting fewer than 200,000 people in the United States.  The incentives include a 25% tax credit on applicable research and development expenditures, waived user fees when submitting applications to FDA, and the potential for a seven-year period of orphan drug exclusivity for the approved indication.  Manufacturers must request and be granted designation before they are eligible for any incentives. When FDA grants marketing approval for a designated orphan drug for a use or indication within the designation disease or condition, FDA will determine if the drug is eligible for orphan drug exclusivity.  Orphan drug exclusivity is product and indication specific, meaning FDA cannot approve a drug containing the same active moiety for the same intended use or indication during the period of marketing exclusivity. FDA uses different criteria in determining sameness for macromolecule and small molecule drugs [1]. The FDA defines “same drug” for macromolecule drugs as “a drug that contains the same principal molecular structural features (but not necessarily all of the same structural features) and is intended for the same use as a previously approved drug, except that, if the subsequent drug can be shown to be clinically superior, it will not be considered to be the same drug." [2]If a sponsor requests orphan drug designation for a drug that is the same as a drug already approved for the same use or indication, the sponsor must provide a plausible hypothesis that its drug is clinically superior to the already approved drug.  Designation as clinically superior is based on greater efficacy, safety, or a major contribution to patient care.  In order to receive the seven-year market exclusivity, a sponsor must demonstrate that its product is actually clinically superior.<h4>Interpreting Sameness of Gene Therapy Products</h4>Human gene therapy products may qualify for orphan drug designation if they are intended for the treatment of a rare disease or condition and the sponsor sufficiently establishes a basis for expecting the drug to be effective in treating the rare disease.  The existing regulations do not describe how the “same drug” definition applies specifically to gene therapy products for orphan drug designation and exclusivity.  Mirroring the January 2020 draft guidance, FDA’s final guidance provides some insight into the current interpretation of how the “sameness” criteria applies to gene therapies.Assuming that two gene therapy products are intended for the same use or indication, FDA will consider the “principal molecular structural features” of the gene therapy products when determining “sameness.”  In the final guidance, FDA states its intention to generally “consider certain key features such as transgenes and vectors used in gene therapy products to be ‘principal molecular structural features’ under this regulation.”  However, FDA does not intend to classify two gene therapy products as different based solely on minor differences in the transgenes and/or vectors, and will determine whether differences are minor differences on a case-by-case basis.For two gene therapy products intended for the same use or indication, if the products express different transgenes, FDA generally intends to consider them to be different drugs because they will not contain the same principal molecular structural features.  This would be the case regardless of whether the two gene therapy products at issue have or use the same vector.  FDA also intends to consider vectors from a different viral group to be different for purposes of determining “sameness.”  Additionally, FDA clarified in the final guidance that it will consider two gene therapy products from the same viral group to be different “when the differences between the vectors impact factors such as tropism, immune response avoidance, or potential insertional mutagenesis.”  FDA intends to determine whether variants of a vector from the same viral group are the same or different on a case-by-case basis.In a case where two gene therapy products express the same transgene and have/use the same vector, FDA may also consider additional features of the final product when determining “sameness,” such as regulatory elements (e.g., promoters or enhancers).  In these instances, FDA generally intends to determine “sameness” of gene therapy products on a case-by-case basis.[1] 21 CFR 316.3(b)(14).[2] 21 CFR 316.3(b)(14)(ii).

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FDA Finalizes Guidance on Interpretation of Orphan Drug “Sameness” for Gene Therapy Products

The EU General Court’s Ruling on Pay for Delay

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On September 30, 2021, the U.S. Food and Drug Administration (FDA) announced final guidance titled “Interpreting Sameness of Gene Therapy Products under the Orp

FDA Finalizes Guidance on Interpretation of Orphan Drug “Sameness” for Gene Therapy Products

Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In addition, a number of policies implemented in response to COVID-19 are expected to have a positive impact on rare disease drug development going forward. Yet, the lasting nature of these policies, post-pandemic, remains uncertain, creating an opportunity for rare disease drug sponsors to be proactive in engaging with regulatory authorities and the patient community to advocate the retention of these positive policy developments.A quick review of the <a href="https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm" target="_blank">FDA’s Orphan Drug Designation Database</a> for designations with a 2020 date finds that 476 Orphan Drug Designations (ODDs) were issued by FDA’s Office of Orphan Drug Product Development. This compares to 340 ODDs issued by FDA during 2019. This indicates that both product development and FDA resources remained focused on rare disease products, even during the exceptional strains 2020 brought to both groups. On August 28, 2020, FDA proceeded with the planned public meeting on “<a href="https://www.fda.gov/drugs/news-events-human-drugs/public-meeting-cder-standard-core-sets-clinical-outcome-assessments-and-endpoints-grant-program-0" target="_blank">CDER Standard Core Sets Clinical Outcome Assessments and Endpoints Grant Program</a>,” an important milestone in the COA Endpoints Pilot Grant Program where grantees were able to share their development plans for the COA standard core COAs sets and receive feedback from stakeholders. The COA Endpoints Pilot Grant Program launched late in 2019 and is part of FDA’s Patient Focused Drug Development efforts. Three grants were made to enable COA standard core set development in (1) migraine, (2) acute pain in infants and young children, and (3) physical function across a range of chronic conditions. The goal of the COA standard core sets is to establish a minimum list of impacts that matter most to patients, are likely to demonstrate change, and that should be assessed during a clinical trial.Although not purposefully directed at rare disease clinical trials, FDA’s “<a href="https://www.regulations.gov/document/FDA-2020-D-1106-0002" target="_blank">Guidance on Conduct of Clinical Trials of Medical Products during COVID-19 Public Health Emergency Guidance for Industry, Investigators and Institutional Review Boards</a>” released on March 18, 2020, has in fact significantly benefited ongoing and newly initiated rare disease trials. FDA often updated the guidance throughout 2020, in April, May, July, September, and in December, each time addressing new questions and providing further clarity on trials for enrollment, remote visits, remote monitoring, and protocol modifications. Clinical trials for rare disease indications are particularly important to the rare disease patient community since there are approximately 7,000 identified rare diseases and a mere 10% have an FDA-approved treatment or therapy, meaning many patients can only access unapproved treatments through investigational studies. The policy changes made in response to the pandemic may pave the way in the future for decentralized, virtual clinical trials that are expected to significantly benefit rare disease studies where patient enrollment, engagement, and retention are exceptionally challenging. These policies allowed for integrating home health visits and telemedicine, direct-to-patient drug and supply delivery, and collection of data by portable or wearable devices. A November 12, 2020, Tuffs Center for the Study of Drug Development report found that “55% of active ongoing clinical trials have transitioned to remote and virtual execution models since early spring,” indicating that the transition to decentralized trial design is prevalent and may become the norm in coming years.FDA implemented administrative simplifications, such as permitting applicants and sponsors to submit information to the Office of Orphan Drug Product Development through email submissions rather than the recently launched online portal for ODD requests. Congress also assisted in maintaining existing rare disease programs in 2020, authorizing an extension of the <a href="https://www.fda.gov/media/90014/download" target="_blank">Rare Pediatric Disease Priority Review Voucher</a> program in December 2020, that was, as we disused in an <a href="https://lifesciences.mofo.com/topics/Bill-Proposes-Continuation-of-FDA-Rare-Pediatric-Disease-Priority-Review-Vouchers.html" target="_blank">earlier post</a>, at risk of expiring in the midst of the pandemic.FDA’s rare disease focus in 2020 extended to minimizing the impact of COVID-19 circumstances for rare disease patients. For example, FDA quickly realized the increase in demand for hydroxychloroquine as a potential treatment for COVID-19 and established a priority review process for all generic hydroxychloroquine drug applications so that lupus patients could continue to access the drug for its FDA-approved use. FDA also worked with manufacturers of rare disease drugs as part of FDA’s supply chain management efforts in response to COVID-19.Rare disease patients were uniquely impacted during the COVID-19 pandemic, with 74% of the <a href="https://rarediseases.org/wp-content/uploads/2020/05/NRD-2061-RareInsights-CV19-Report_v2-2.pdf" target="_blank">NORD survey</a> respondents reporting a medical appointment had been canceled due to the pandemic, and 29% of respondents reporting loss of a job, with 11% of those job losses also meaning a complete loss of health insurance. We are encouraged that sponsors continue drug development programs, as evidenced by the large number of ODDs in 2020, and that FDA continues to devote resources and attention to programs focused on rare disease drug development. We will monitor these areas in 2021 and remain hopeful that the clinical trial policies put into effect during the pandemic prove to be viable and accessible tools for future rare disease clinical trials.On the patent exclusivity and investment fronts, orphan diseases have increasingly been on the forefront, with focus on drugs and partnering for these indications. This reflects a major shift and a breakaway from the traditional, narrower view of the value and importance of these programs. “Five or 10 years ago the dogma was that these indications would not necessarily warrant full investment, either for clinical development or patent exclusivity,” according to Morrison & Foerster’s partner <a href="https://www.mofo.com/people/catherine-polizzi.html" target="_blank">Dr. Catherine Polizzi</a>, recognized patent law leader with over 25 years in life sciences patent practice. “I thought this was a short-sided view, and that there was tremendous opportunity in rare diseases, not to mention the important attention needed for these patients. Those who previously ignored this have very much changed course.”

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Intellectual versatility combined with incisive, pragmatic counseling and business acumen is the hallmark of Catherine’s practice.
Catherine helps emerging and established companies through the challe

Catherine M. Polizzi Ph.D.

Catherine Polizzi

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Rare Disease: Sustained Progress in Development Regardless of the Pandemic

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Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare dis

Rare Disease: Sustained Progress in Development Regardless of the Pandemic