Recent trends in the regulatory landscape and healthcare could significantly impact the pharmaceutical industry.
Expansion of Therapeutic Modalities: The modality landscape is broadening beyond traditional small molecules and proteins to include peptides, antibody-drug conjugates, nucleotide-based therapies (especially antisense oligonucleotides, siRNAs, and mRNAs), radionuclides (theranostics) and cell and gene therapies. This diversification brings new preclinical and drug development requirements and additional regulatory complexities. For example, the complexities of bringing a radionuclide to market are amplified when regulatory agencies must cope with the fact that most radioactive therapeutics need to be used within one week – and often the same day – that they are manufactured.[1] These requirements necessitate regulation at the level of the radionuclide generator itself (68Ga and 99mTc) or as radionuclide precursor formulations (177Lu, 111In, 64Cu, 90Y) due to the sophisticated infrastructure involved[2].
Increased Focus on Digital Technology: Regulatory changes in 2024 aim to clarify technology and data collection rules. This includes updates to the International Council for Harmonisation’s Good Clinical Practice E6(R3) draft guideline, defining best practices related to technology, software, and remote elements. The FDA now provides more complete guidance on collecting remote data using digital health technologies.
Regulating Laboratory-Developed Tests (LDTs) as Medical Devices: The FDA formally decided to regulate LDTs as medical devices in 2024, despite pushback from some companies. The formal guidance was issued 06 May 2024[3] and takes effect 05 July 2024. The new regulation aims to ensure the reliability of these tests, requiring that they are cleared, approved, and authorized by the FDA. This is being implemented to attenuate the FDA’s concerns of the safety and effectiveness of these treatments, whose results naturally influence medical decisions. This change may lead to challenges in market entry for new tests and could impact how a pharmaceutical company plans for the use of companion diagnostics.
Implementing the NIH Data Management and Sharing Policy: With the new policy[4], organizations must adopt a Data Management and Sharing (DMS) framework for research linked to various initiatives. This policy aims to enhance data sharing and transparency in research, necessitating robust data management systems for compliance.
Continued Push for Diverse and Patient-Centric Trials: Regulators emphasize the importance of including more diverse patient populations in clinical trials and incorporating patient input throughout the drug development process. There’s a move towards more patient involvement in regulatory review and decision-making, reflecting a broader trend of patient-centricity in healthcare.
Use of March-in Rights: The Biden Administration has outlined plans to use March-in rights to seize patents of government-funded drugs if pharmaceutical firms price them too high. This controversial measure could lead to substantial debate and litigation, potentially affecting the U.S. research and development sector.
These changes reflect a broader trend towards more inclusive, patient-focused, and technologically advanced drug development and regulation approaches. Companies in the pharmaceutical sector will need to adapt to these evolving regulatory landscapes to remain compliant and competitive.
Resources
n.a. (2023, May 19). INTERNATIONAL COUNCIL FOR HARMONISATION OF TECHNICAL REQUIREMENTS FOR PHARMACEUTICALS FOR HUMAN USE, ICH HARMONISED GUIDELINE GOOD CLINICAL PRACTICE (GCP) E6(R3). International Council for Harmonisation. https://database.ich.org/sites/default/files/ICH_E6(R3)_DraftGuideline_2023_0519.pdf
Cohen, J. (2023, December 7). Biden Administration Says It Plans To Use March-In-Rights For Drugs To Lower Prices. https://www.forbes.com/sites/joshuacohen/2023/12/07/biden-administration-says-it-plans-to-use-march-in-rights-for-drugs-to-lower-prices/?sh=22eadc8f389a
[1] Production and regulatory issues for theranostics – ScienceDirect
[2] Emerging Radionuclides in a Regulatory Framework for Medicinal Products – How Do They Fit? – PMC (nih.gov)
[3] Federal Register :: Medical Devices; Laboratory Developed Tests
[4] Data Management & Sharing Policy Overview | Data Sharing (nih.gov)
