Life Sciences Perspectives
WHAT WE ARE TRACKING
Expand the sections below to learn more about the trending topics in Q3 2023 and beyond.
- We are tracking updates from the US Food and Drug Administration (FDA or the Agency) on in-person face-to-face (FTF) formal meetings with FDA. Beginning June 12, 2023, the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) expanded in-person FTF industry meetings (with a hybrid component) to include requests for Type B end-of-phase 2 (EOP2), along with the previously announced Type A, biosimilar biological product development (BPD) Type 1, and Type X meeting requests. Many sponsors are eager to resume in-person advisory committee meetings, but FDA has not indicated when this might occur.
- FDA recently announced the availability of draft guidance, adopted from the International Council for Harmonisation’s recently updated E6(R3) draft guideline, with updated recommendations for good clinical practices aimed at modernizing the design and conduct of clinical trials. It includes using fit-for-purpose innovative digital health technologies (DHTs) and utilizing risk-based and proportionate approaches across the life cycle of a clinical trial to allocate resources and safety and integrity efforts accordingly. Comments on the draft guidance can be submitted to the docket through September 5, 2023.
- We expect to see continued FDA guidance and engagement with stakeholders on developing relevant clinical endpoints that aid in regulatory decision-making. For example, in June, FDA convened a virtual public workshop related to its Rare Disease Endpoint Advancement Pilot Program (rollout date of July 1, 2023), and is designed to enable sponsors to collaborate with FDA on the development of novel efficacy endpoints for rare diseases. Additionally, in April, FDA published draft guidance on patient-focused drug development and clinical outcome assessments. Public comments for the public workshop are due to the docket by July 23, 2023.
- FDA recently announced the availability of two draft guidances relevant to pediatric drug development and pediatric exclusivity: Pediatric Drug Development: Regulatory Considerations – Complying with PREA and Qualifying for Pediatric Exclusivity Under the BPCA and Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations. The two draft guidances are notable in signaling a policy change from the agency’s 2005 guidance — namely, that “FDA does not expect to issue [Written Requests] solely for studies or planned studies that are required under PREA.” Comments on both draft guidances are due by July 17, 2023.
- On June 26, 2023, the FDA issued draft guidance for industry on Psychedelic Drugs: Considerations for Clinical Investigations. The draft guidance contains FDA’s current thinking on the following topics with specific regard to psychedelic drug development: CMC, nonclinical/preclinical development, clinical design and trial execution, and abuse potential assessment. Interested stakeholders may submit comments on the draft guidance through August 25, 2023.
- Public comments to FDA’s recently published discussion paper “Using Artificial Intelligence and Machine Learning in the Development of Drug and Biological Products,” which is intended to initiate communication on artificial intelligence and machine learning (AI/ML) with stakeholders to promote mutual learning and discussion, are due to the docket by August 9, 2023. FDA is also planning a public workshop (date to be announced) to discuss how the community can work together to realize the potential of AI/ML for product development while being mindful of potential challenges.
- We continue to monitor for FDA’s implementation of its new clinical trial Diversity Action Plan authority for Phase 3 or pivotal study drug and biologic developers and device developers conducting clinical trials (whether under investigational device exemptions or not), pursuant to section 3601 of the Food and Drug Omnibus Reform Act (FDORA).
- We continue to watch for FDA’s implementation of a new provision of the Federal Food, Drug, and Cosmetic Act that allows FDA to designate a “platform technology” that has the potential to be incorporated in or utilized by more than one drug. Under section 2503 of FDORA, FDA may expedite the development and review of a subsequent application for a drug that incorporates or utilizes a designated platform technology and must issue draft guidance on the new designation program within one year of enactment of FDORA.
- We continue to watch for final FDA regulations that would harmonize clinical research regulations with the US Department of Health and Human Services (HHS) Common Rule.
- We continue to monitor a new bill (HR 1117) introduced in February in Congress, with a growing list of cosponsors, that would address inclusion of pregnant and lactating women in clinical research.
Deciding whether a company can market or sell a specific product.
- We are following reintroduced bipartisan legislation that would create a regulatory pathway for the provisional approval of drugs in certain cases. The Promising Pathway Act would require FDA to establish a “rolling, real-time, priority review pathway for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions.” Drugs approved under this proposed pathway would be granted time-limited approval and would be required to track usage through patient registries.
- We continue to follow substitute language reported in the Senate Committee on the Judiciary to the Preserve Access to Affordable Generics and Biosimilars Act (S. 142), introduced in January 2023 with 10 bipartisan cosponsors and reported to the Committee on the Judiciary with an amendment, to “prohibit brand name drug companies from compensating generic drug companies to delay the entry of a generic drug into the market, and to prohibit biological product manufacturers from compensating biosimilar and interchangeable companies to delay the entry of biosimilar biological products and interchangeable biological products.”
- We are monitoring any forthcoming publicly announced outcomes from sponsors who were selected as first participants in FDA’s Advancing Real-World Evidence (RWE) program, which provides those sponsors with four program meetings geared toward having a path forward with FDA for the use of RWE in indication expansion and approvals.
- We continue to monitor FDA’s continued focus on the use of DHT to advance health equity ahead of the Patient Engagement Advisory Committee meeting on September 6, 2023.
- We continue to monitor how FDA’s September 2022 final guidance on clinical decision support software is being applied by the Agency, with an eye toward potential software as a medical device (SaMD) definitional changes stemming from the reopening of the International Medical Device Regulators Forum SaMD working group.
- We are continuing to monitor FDA’s Total Product Life Cycle Pilot, designed to help reduce time from concept to commercialization for medical devices going through the FDA approval and clearance processes. We are also monitoring the Voluntary Alternative Pathway, a potential new regulatory pathway for novel products, including digital health products, referenced by Center for Devices and Radiological Health (CDRH) Director Jeff Shuren at the Food and Drug Law Institute’s annual conference in May 2023.
- We are continuing to monitor the implementation of new cybersecurity authorities for software-containing or internet-connected devices, enabling FDA to require sponsors to develop cybersecurity plans for post-market implementation, as mandated in section 3305 of FDORA. FDA issued its guidance on its refuse to accept policy for cyber devices and related systems earlier this year.
- We are following stakeholder input to FDA’s recently issued draft guidance on predetermined change control plans (PCCPs) for AI/ML-enabled devices.
- We are analyzing the final FDA guidance on the content of premarket submissions for device software functions.
In Vitro Diagnostic/Laboratory Testing
- We are monitoring the Verifying Accurate Leading-edge IVCT Development Act of 2023, which was introduced in the US House of Representatives by Reps. Larry Bucshon (R-IN) and Diana DeGette (D-CO) in March 2023.
- We are awaiting a proposed rule to amend FDA’s regulations “to make explicit that laboratory developed tests (LDTs) are devices under the Federal Food, Drug, and Cosmetic Act,” which FDA included in the Spring 2023 Unified Agenda.
- We are monitoring FDA’s Oncology Center of Excellence’s recently launched new voluntary pilot program, the Oncology Dx Pilot, which focuses on the use of certain LDTs with oncology products to identify cancer biomarkers. Through this program, FDA will request performance information for the tests used to enroll patients in a sponsor’s clinical trials that support drug approval. Based on an assessment of that information, FDA will post to its website the minimum performance characteristics recommended for similar tests that may be used to select patients for treatment with the approved drug. Laboratories may use this information to guide development of their LDTs to identify specific biomarkers used for selecting cancer treatment in order to help facilitate better and more consistent performance of LDTs and overall better drug selection and improved care for patients with cancer.
How companies manufacture, promote, and monitor their approved products.
- The requirements of the Drug Supply Chain and Security Act (DSCSA) have been phased in since November 2014, but full interoperable electronic track and trace systems must be in place for certain manufacturers, repackagers, wholesale distributors, dispensers, and third-party logistics providers of prescription drugs by November 27, 2023. However, the Healthcare Distribution Alliance (HDA) on June 2, 2023, sent a letter to FDA citing the lack of manufacturer readiness and seeking a phased approach employing enforcement discretion “limited to certain DSCSA requirements and trading partners, with full implementation phased in over a period of 2 years.” On June 20, 2023, the HDA reiterated its opposition to a “blanket delay” of implementation while continuing to press for a more nuanced “phased-in” approach. It is unclear whether and how FDA will respond to this effort from the HDA.
- FDA recently issued a long-awaited proposed rule to amend its human prescription drug labeling regulations to require a new type of Medication Guide — "Patient Medication Information” — for prescription drug products used, dispensed, or administered on an outpatient basis. This new proposed Medication Guide would be an FDA-approved, one-page document that highlights essential information that patients need to know about the prescription drug product. Comments to the proposed rule can be submitted to the docket through November 27, 2023.
- FDA recently convened a hybrid workshop, “Advancing the Utilization and Supporting the Implementation of Innovative Manufacturing Approaches,” focused on regulatory strategies to support the utilization of manufacturing technologies for drugs and biological products.
- We are awaiting a final rule by which FDA will align its quality system regulation with ISO 13485.
- Section 3611 of FDORA codifies FDA’s authority over medical device inspections to allow the Agency to collect “records or other information” from facilities “in advance of, or in lieu of” inspections of manufacturing establishments. We will be monitoring for FDA’s guidance on this topic.
- FDA expects device labelers to complete their Global Unique Device Identification Database (GUDID) updates to include Global Medical Device Nomenclature (GMDN) Term Codes by the end of July 2023 to assure that accurate information is released publicly in AccessGUDID and openFDA on August 14, 2023. We also are monitoring reports that FDA is currently issuing letters to device manufacturers related to informational errors in GUDID.
- On June 27, 2023, FDA published final guidance on Presenting Quantitative Efficacy and Risk Information in Direct-to-Consumer Promotional Labeling and Advertisements, updating FDA’s 2018 draft guidance on this topic. This new guidance updates industry on the current thinking of FDA’s Office of Prescription Drug Promotion (OPDP) on presenting quantitative DTC efficacy and risk information in a consumer-friendly way. Highlights of the guidance include a focus on: presentation of control group information, use of absolute frequencies (e.g., 5 out of 100 patients) and percentages (e.g., 5%), the need for contextual information when presenting relative frequencies, formatting considerations for presenting quantitative information, and the use of visual aids to improve consumer comprehension. On the heels of this guidance, we are monitoring whether OPDP will issue any untitled or warning letters to industry for DTC labeling or advertising to drive home any of its recommendations in this recent guidance. The only untitled letter issued thus far by OPDP in 2023 pre-dates publication of this final guidance, but we note that its focus on quantitative efficacy information presented on a consumer website illustrates the application of FDA’s thinking in its recent guidance.
- We continue to follow how the Inflation Reduction Act of 2022 (IRA) will affect reimbursement for pharmaceutical and biologic products (price caps, required discount program for all drugs, mandatory rebates if a drug’s price increases at a rate that outpaces inflation, a redesign of the Medicare Part D benefit, and an increase in biosimilar pricing). In February 2023, the Centers for Medicare & Medicaid Services (CMS) released proposed guidance for the calculation of inflation rebates under Medicare Part B and Medicare Part D, and in March 2023, CMS released proposed guidance for the drug price negotiation program. CMS released its final initial drug price negotiation program guidance in June 2023. We anticipate that CMS will continue to release proposed and final guidance over the coming months. Notably, applications for the small biotech exception were due at the beginning of July 2023, and applications for the initial delay request for biosimilar manufacturers were due in May 2023. In addition, the selection of the first 10 drugs subject to the negotiation program on September 1, 2023. Also, in June 2023, four lawsuits were filed by Merck, BMS, the US Chamber of Commerce, and PhRMA, alleging that the IRA’s drug price negotiation program and in particular the excise tax punishment imposed by the IRA are unconstitutional, citing violations of the First Amendment’s prohibition on compelled speech; the Fifth Amendment’s prohibition on government taking of property without just compensation; the Fifth Amendment’s prohibition on the government depriving private parties of property without due process; the Eighth Amendment’s ban on excessive fines and penalties; and an assertion that the IRA violates the Constitution’s separation of powers. Each suit requests declaratory and injunctive relief, including a delay in the implementation of the IRA’s drug price negotiation provisions.
- We are monitoring HHS’s proposed rule that would modify the current alternative dispute resolution process under the 340B drug discount program, which has been the subject of much scrutiny and disagreement among pharmaceutical manufacturers (who are obligated to cap prices significantly for patients of qualified covered 340B public health entities) and the public health entities whose patients benefit from reduced 340B pricing. The proposed rule was open for comments until January 30, 2023, and would simplify and expedite the process by which covered entities and drug manufacturers resolve disputes about overcharging, duplicate discounts, or drug diversion.
- Congress is considering numerous proposed pharmacy benefit manager (PBM) reforms in both the House and the Senate. Legislative proposals include but are not limited to: eliminating rebates; divorcing service fees from the price of a drug, discount, or rebate; prohibiting spread pricing; limiting administrative fees; requiring PBMs to report formulary placement rationale; promoting transparency; and many others.
- CMS issued a proposed rule, “Misclassification of Drugs, Program Administration and Program Integrity Updates Under the Medicaid Drug Rebate Program (MDRP)” (Proposed Rule). Among other topics, the Proposed Rule focuses on enhancing MDRP administration by incorporating policies that would facilitate more accurate and consistent drug information reporting, data collection, and operations. One of the most significant proposals is to revise the definition of “best price” to provide that manufacturers must aggregate cumulative discounts, rebates, or other arrangements to determine the final price realized by the manufacturer for a particular unit of a drug. The CMS fact sheet is available here. Comments on the Proposed Rule are due to the docket by July 25, 2023.
- CMS has finally released its new proposed coverage procedures that would permit earlier coverage of medical technology that has received FDA breakthrough designation. The new program, the Transitional Coverage of Emerging Technology pathway, will leverage existing national coverage determination and coverage with evidence development processes to help ensure earlier coverage for advanced technology and to address the so-called medtech valley of death (i.e., the period after FDA market authorization and before Medicare coverage). Comments on the proposal are due to the docket by August 28, 2023.
Companies’ interactions with their customers and other stakeholders.
- We continue to see an increase in Department of Justice enforcement of the False Claims Act and Anti-Kickback Statute in laboratory, diagnostic testing, and telehealth spaces.
- In its third advisory opinion of 2023, OIG approved a lab testing company’s proposal to provide a prepaid $75 gift card to individuals to encourage them to return a sample collection test kit in connection with a colorectal screening test. OIG concludes that it does not raise an issue under the beneficiary inducement statute as the card would satisfy the “preventive care exception,” including because the gift card would not be a “cash equivalent.” OIG concludes that it would implicate the Anti-Kickback Statute but that there is a low risk of increased costs to federal health care programs, and the incentive would promote patient compliance with a recommended screening test, along with the relevant controls put into place by the requester. Accordingly, OIG would decline to impose sanctions on the arrangement.
- OIG also released an FAQ that distinguishes among cash, cash equivalents, and in-kind gift cards for purposes of the fraud and abuse laws. According to OIG, “cash” refers to monetary payments in the form of currency, and “cash equivalents” include items convertible to cash (such as a check) or items that can be used like cash, such as a general-purpose prepaid card such as a Visa or Mastercard gift card, or gift cards offered by large retailers or online vendors that sell a wide variety of items. OIG considers some gift cards to be “in-kind,” for example if they can be redeemed only for certain categories of services or items (e.g., a meal delivery service or gasoline).
- OIG has indicated that it will undertake an annual analysis of Medicare payments for clinical diagnostic laboratory tests in 2022, with a report date expected in FY 2024.
- OIG announced a plan to update the format of and public notice protocol for its voluntary, nonbinding compliance program guidance documents (CPGs). This plan demonstrates how OIG is taking steps to more rapidly address emerging risks. OIG stated that it will no longer publish updated and new CPGs in the Federal Register.
Companies’ interactions with sensitive patient data.
- We continue to see increasing touchpoints for companies accessing sensitive patient information through integrated data platforms that could more significantly implicate the Health Insurance Portability and Accountability Act (HIPAA), General Data Protection Regulation (GDPR), and US state privacy and data security laws like the California Consumer Privacy Act of 2018 (CCPA).
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