The following letter was sent to the President of the United States in response to talks between the U.S. and China related to trade. 

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Good Review Management Principles and Practices for New Drug Applications and Biologics License Applications, Guidance for Industry and Review Staff. BIO appreciates the effort to update and release the guidance to inform industry, sponsors, and FDA staff and reviewers about regulatory review practices. This will support a more efficient and transparent review of new therapies. BIO provides suggestions on how to strengthen the document and communications, and ensure all stakeholders have appropriate information and resources.  

This Special Report highlights the potential opportunities, entry approaches and local partner selection, and also include recommendations on market acceleration and internationalization.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings, which FDA says is intended to assist sponsors of drug and biological products for rare diseases in planning and conducting more efficient and productive pre-investigational new drug application (pre-IND) meetings. BIO applauds the FDA’s initiative to advance drug development and review for therapies to treat rare diseases. It’s estimated there are 7,000 identified rare diseases, but only 5% have an approved therapy. This guidance provides important information to drug developers to make the drug development process more consistent, transparent, and efficient. BIO provided general comments and line edits.

BIO submitted comments to the Food and Drug Administration (FDA) following the public meeting, Patient-Focused Drug Development (PFDD) Guidance: Methods To Identify What Is Important to Patients and Select, Develop, or Modify Fit-for-Purpose Clinical Outcome Assessments. BIO commends the FDA for working to ensure patient experiences are more systematically collected and used to inform the development and review of new therapies. BIO strongly believes that, in order to truly support patient-focused drug development, patient experience data should be considered for use throughout the drug development and review lifecycle. Appropriate fit-for-purpose tools for collecting patient experience data have the potential to inform protocol design, endpoint development, benefit-risk assessments, and labeling, among other aspects of drug development and assessment. To encourage stakeholders to collect fit-for-purpose patient experience data, we request the FDA more clearly indicate the breadth of regulatory decisions for which they will consider different types of patient experience data. BIO makes suggestions for how FDA can improve the guidance documents and FDA website, External Resources or Information Related to Patient Experiences. In addition, BIO requests FDA keep the scope of new PFDD guidance documents broadly applicable and flexible.

BIO submitted comments on the Food and Drug Administration’s (FDA) open docket, Patient-Focused Drug Development Guidance: Methods to Identify What Is Important to Patients and Select, Develop, or Modify Fit-for-Purpose Clinical Outcome Assessments. BIO commends the FDA for working to ensure patient experiences are more systematically collected and used in the development and review of new therapies. In particular, BIO appreciates the work to develop guidance documents and discussion guides prior to public meetings. To truly support patient-focused drug development, BIO believes patient experience data should be considered for use throughout the development and review lifecycle. Appropriate “fit-for-purpose” tools for collecting patient experience data have the potential to inform many aspects of drug development and assessment. To encourage stakeholders to collect fit-for-purpose patient experience data, BIO requests FDA publicly and clearly explain how and when they will use patient experience data in different drug development and regulatory decisions. In addition, BIO provides general comments and line edits as well as responses to questions posed by the FDA in the open docket.