As Prescribed

FDA recently issued a new draft guidance titled Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies, which proposes omitting comparative efficacy studies (CES) where appropriate “based on the totality of the evidence” to instead rely only on analytical, PK/PD, and immunogenicity data to inform biosimilarity determinations. As the agency explains, it has gained substantial experience with comparative analytical assessments (CAA) and now wants to update its framework for determining when a CES may not be necessary to support biosimilarity.

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has announced its intention to introduce sweeping reforms for accelerating rare disease therapy development and approval, with the aim to position the UK as a global leader in the field. The new framework will put in place flexible licensing for rare disease therapies with adaptive trial design and a new “investigational marketing authorization.” It will also ensure a patient-centered approach with an emphasis on transparency, flexible benefit-risk thresholds, and use of real-world evidence.

The US Food and Drug Administration has announced a renewed focus on a long-considered issue—whether FDA should release complete response letters (CRLs) issued to drug or biologic applicants. Specifically, on July 10, 2025 FDA compiled and released more than 200 CRLs issued between 2020 and 2024 for approved new drug application (NDA) and biologics license application (BLA) products. Improving on past agency practice, these CRLs are now located within a single database, providing the public with easy access to a trove of information regarding FDA’s thinking when making approval decisions.

The US Food and Drug Administration (FDA) recently announced a strategic roadmap to phase out animal testing requirements for certain drug products, starting with monoclonal antibodies. This shift marks a significant change in regulatory policy, reflecting advancements in science and technology that offer alternative testing methods.

Aiming to expedite drug development, enhance the body of clinical evidence supporting new and existing therapies, and improve participation and diversity in clinical trial populations, the US Food and Drug Administration (FDA) recently issued three guidance documents that impacted the clinical trial landscape and established a new hub for clinical trial innovation.

The US Food and Drug Administration (FDA) issued draft guidance, providing recommendations to sponsors who are considering submitting a non-interventional study (i.e., an observational study) to FDA to support the demonstration of substantial evidence of effectiveness and/or evidence of safety of a drug or biologic, as another chapter in its evolution toward the use of real-world evidence (RWE).

For our third and final Biotech Week Boston post, partner Stephen Altieri—one of the many within our 250-member-strong comprehensive cross-practice life sciences team with an advanced scientific degree—discusses efficient patent protection for emerging biotech companies with patent agent Emily Coury.