The Biotechnology Industry Organization believes that fully realizing the promise of biotechnology requires a comprehensive national strategy that fine-tunes some policies and overhauls others.

BIO’s set of policy proposals address two vital needs: 1) the need to re-engineer the biotech economic model, and 2) the need to re-invent the idea-to-market pathway for biotech cures and other products.

Below is that plan:

Creating an FDA that Turns Hope into Cures

The American population is growing older — life expectancy is up by a decade since 1965 and 72 million Baby Boomers are about to enter Medicare. It has never been more critical to support an industry that is working to cure diseases and will impact all Americans by saving lives and dollars. It is imperative that the U.S. Food and Drug Administration (FDA) recognizes its national role in advancing innovation by reviewing innovative products in a timely manner and promoting a consistent and science-based decision making process that is reflective of patient needs.. By facilitating the creation of a 21st century FDA and more effective clinical research and development processes, the proposals below help establish a clear and effective pathway for turning hope into cures.

Elevating FDA and Empowering Operational Excellence

Include Innovation in FDA’s Mission Statement

FDA must have both the capacity and commitment to incorporate the latest scientific advances into its decision-making so that processes can keep pace with the tremendous potential of companies’ cutting-edge science. Congress can help encourage medical breakthroughs by updating FDA’s mission to incorporate modern scientific tools, standards, and approaches.

Establish a Fixed Term of Office for the Commissioner of Food and Drugs

Encouraging consistent and stable leadership at FDA — with protection from the political influence that typically occurs during a Presidential Administration transition — would better equip the agency to fulfill its mission as a science-based regulator to promote and protect the public health. The law should be amended to provide that the President appoint the Commissioner to a six-year term of office. Once confirmed, the Commissioner would be removable by the President only for pre-specified reasons — neglect of duty, malfeasance in office, or an inability to execute the FDA’s mission.

Grant FDA Status as an Independent Agency

The FDA regulates nearly a quarter of the consumer goods supplied to the American public. As such, the agency should have the same authorities to make budget, management, and operational decisions as afforded other independent agencies such as the Environmental Protection Agency. This would empower the agency to work more effectively with the President and Congress to carry out its mission to promote and protect the public health, and would also enhance the agency’s ability to obtain quality and consistent leadership.

Establish an External Management Review Board for FDA

The FDA is a large, complex organization. Amending the law to establish a Management Review Board (consisting of experienced external advisors) that conducts periodic reviews of FDA’s management and organizational structure and provides fresh, visionary, and independent thinking and recommendations on how to improve FDA’s ability to fulfill its mission could help the agency address its chronic operational challenges.

Advancing Regulatory Science & Innovation

Release FDA Funding to Support Regulatory Science Public-Private Partnerships

Congress established an independent, nonprofit foundation to support public-private partnerships for the purpose of advancing FDA’s mission through, for example, the formation of collaborations to advance the use of biomarkers, surrogate markers, and new trial designs to improve and speed clinical development. However, Congressional appropriations bills have subsequently restricted FDA’s ability to transfer federal funding to the foundation. These funding restrictions should be lifted so that the foundation can fulfill its intended purpose and promise

Create an FDA “Experimental Space” to Pilot Promising New Scientific and Regulatory Approaches The FDA has developed several initiatives to advance regulatory science. However, FDA’s ability to incorporate modern science into its regulatory processes has been limited because there is no entity within the agency with unified responsibility for systematically analyzing the findings and recommendations from these initiatives, and with clear authority to pilot promising scientific and regulatory approaches. An FDA “Experimental Space,” led by a new Chief Innovation Officer, should be established with the responsibility and authority to ensure that promising new approaches are integrated into agency operations at all levels.

Enhance FDA’s Access to External Scientific and Medical Expertise

Scientific and medical knowledge, techniques, and technology are advancing at a more rapid pace today than at any other time; however, FDA’s capacity to access information about these advances has not kept pace despite the widespread perceptions of the agency as the global standard bearer for science-based regulatory review. It is essential that FDA’s access to scientific and medical advice be enhanced by improving the operations of FDA Advisory Committees, establishing Chief Medical Policy Officers in the immediate offices of the Center Directors, and providing FDA staff with additional avenues for accessing external scientific and medical expertise.

Enabling Modernized Patient-Centric Clinical Development

Increase Access to Innovative Therapies through Progressive Approval

Patients, particularly those with illnesses for which no adequate therapy exists, want access to promising new therapies earlier in the drug development process. Expanding and improving the accelerated approval pathway into a progressive approval mechanism would provide patients timely access to needed therapies, while helping ensure smaller biotech companies are able to maintain operations through extensive phase III clinical testing. Only innovative products for unmet medical needs, significant advances to standard of care, targeted therapies, or those that have been approved by the European Medicines Agency or other mature regulatory agencies would qualify for progressive approval.

Of the 54 orphan drugs approved between
1998 and 2007,
58% were discovered and
developed by biotech companies.

Nature Reviews/Drug Discovery, November 2010

Empower FDA to Utilize a Weight-of-Evidence Approach to Establish Effectiveness

FDA is statutorily required to approve applications for new drugs when they have been demonstrated to be safe and there is “substantial evidence” that the new drug is effective. FDA typically requires two “adequate and well controlled” studies under this standard.

A weight-of-evidence approach to data analysis, however, allows the decision-maker to look at all data and information, whatever its value, and give each appropriate consideration.

Between 1999 and 2005, the average length
of clinical trials
grew by 70%. Currently, the
average time from discovery of a drug
to getting it to patients is
10 to 15 years.

Source: Tufts Center for the Study of Drug Development

Leverage Electronic Health Records to Facilitate Clinical Research

Using health information technology (IT) such as electronic health records in clinical research will improve and speed up the drug development process while decreasing costs. However, there are significant barriers preventing widespread use of health IT in clinical research, including slow adoption by providers and lack of standards. To help remove those barriers, Congress should create a Clinical Informatics Coordinator in the Office of the Commissioner of Food and Drugs charged with developing processes to validate and encourage the use of health IT in clinical research and establishing pilot projects to use health IT in clinical research.

Require FDA to Disclose to Companies Reasons for Non-Approval

Current law implies that new drug and biologic applications must either be approved or denied. In practice, however, there is a third response in which FDA neither approves nor officially denies the application (which would require FDA to give the company specific procedural rights such as a hearing); rather, FDA finds the application to be incomplete in some way and therefore ineligible for approval. When FDA makes such a finding, it should communicate to the company in clear terms why risk was determined to outweigh benefits and why tools such as Risk Mitigation and Evaluation Strategies are insufficient (in addition to indicating what must be done to address any deficiencies). This will help ensure a consistent and transparent risk-benefit evaluation and provide the company with better information on what, if any, additional studies are required to achieve approval.

 

See also:

BIO: Unleashing the Promise of Biotechnology (pt.1)
BIO: Unleashing the Promise of Biotechnology (pt.2)
BIO: Unleashing the Promise of Biotechnology (pt.3)

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