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FDA/CMS Speaker Q&A

We asked our speakers to share their expertise on recent developments in federal drug regulations, industry changes, and the resulting impact on their work.

Dr. Sarfaraz Niazi, Adjunct Professor, Department of Biopharmaceutical Sciences at UIC College of Pharmacy


1) What will it take for biosimilars to succeed in the US market?

Broader participation of developers to expand the choices, a better understanding of the FDA guidance and several key changes in the process and regulations of approval.

2) What obstacles are the most challenging and what, in your expert opinion, are immediate solutions or workarounds?

Current cost of $100-200 Million is untenable; more creative approaches to strategic partnering with CROs and CMOs and creative approaches to establish bio similarity are needed.

3) How does and which federal regulation policies impact your work, and what positive changes do you hope to see in the near future?

Establishing bio similarity means assuring no clinically meaningful difference that should obviate any additional studies to support interchangeability; the tier-1 analytical similarity testing requires complete revision to support what is clinically meaningful.

Dr. Lindsay Rosenwald, Chairman, President and CEO of Fortress Biotech


1) Why are drugs so costly, and what are the driving factors that impact cost, directly and indirectly?

Critics often focus on the price of a single drug and neglect to consider risk and reward as a whole. The financial support of stockholders, venture capitalists, angel investors and others, is vital to the development of innovative treatments. However, because only a handful of the drugs that go into the clinic gain FDA approval, drug companies must entice investment support by facilitating ways for shareholders to not only recoup the cost of approved drugs, but also of the therapies that have been unsuccessful. In order for drug companies to continue to evaluate promising drug targets that may have major impacts on human health, it is important that, when looking at cost issues, we factor in not only the cost of developing a single drug, but also development costs for those that failed. 

 2) What would you say to critics who say, “Big pharma is in the business of selling medication, not finding cures”?

I don’t believe that’s accurate. In the past, biopharma companies have been focused on developing treatments for chronic conditions such as hypertension, hypercholesterolemia, etc. Hepatitis C, which was untreatable, is now completely curable through biopharma industry-developed medicines. These days, biopharma companies are not only focused on treating chronic diseases, but also developing cures through applications like gene therapy and gene editing. Companies are incentivized to work towards cures because if they don’t, they’ve missed an opportunity that competitors will surely take.

 3) How does, and which, federal regulation policies impact your work? What positive changes do you hope to see in the near future?

The FDA and its policies have the greatest impact on our work, as the agency implements development requirements to ensure that therapies are safe and effective. The FDA has implemented valuable incentive programs like Breakthrough Therapy designation and Priority Review, and is tuned into the need for faster approval of quality drugs. 

 In the future, the use of artificial intelligence may positively impact drug development by easing the burden of clinical trials without sacrificing efficacy or safety. As technology innovates to require fewer patients to undergo trials, biopharma companies will conserve resources, which can then be invested back into R&D. This may expedite drug development while directly influencing how treatments are priced. 

Dr. Jonathan Goldsmith, Associate Director Rare Disease Program at Center for Drug Evaluation and Research (CDER) Food and Drug Administration (FDA)


1) What are the latest trends and developments in rare diseases/research?

Rare Disease approvals continue to be ~40% of Novel New Drug approvals at the Office of New Drugs/CDER/FDA.

2) What benefits has PRVs served for the last decade? What have they failed to do? 

Rare Pediatric Disease Priority Review Vouchers were enacted in 2012. Guidance on the topic was issued in draft form on November 17, 2014. The first voucher was awarded on January 7, 2014. In March, 2016, the GAO found that it was too early to gauge effectiveness of this voucher program.

3) How does and which federal regulation policies impact your work, and what positive changes do you hope to see in the near future?

Sections of PDUFA VI (I.4.) indicate that “FDA will build on the success of the Rare Disease Program in CDER and CBER by continuing to advance and facilitate the development and timely approval of drugs and biologics for rare diseases, including rare diseases in children.” This will include integration of the RDP into review teams for rare disease drug development programs and continuation of provision of training to all CDER and CBER review staff. “RDP staff will continue to engage in outreach to industry, patient groups, and other stakeholders to provide training on FDA’s RDP…and staff will also facilitate interactions between stakeholders and FDA review divisions to increase awareness of FDA regulatory programs and engagement of patients in FDA’s regulatory decision-making.”

Tom Watson, Executive Director at TW Consulting Group


1) How has partnering with pharma and biotech organizations aided in developing strategies for Pre-approval Access and global programs for patients to access treatment they otherwise wouldn’t be able to access?

Pharma and Biotech companies are traditionally set up to research, develop and market treatments that address areas of unmet medical need. The strategy and activities needed to design, set-up and run successful pre-approval access programs do not always naturally align with these objectives. Therefore, companies gain great value in partnering with individuals and organizations that have extensive first-hand experience within the pre-approval access space.

2) What hurdles exist for companies interested in satisfying the immediate needs of patients whilst balancing developmental objectives and how are you aiding to solve the problem?

Many of the hurdles that exist for companies to balance these needs can be overcome with enough planning time and focus, underpinned with a clear strategy. I engage with companies at an early stage and work through each of the topics that need to be addressed in order to design a successful global program. Of course, many companies are playing catch up in this area (and are already receiving requests for their investigational treatments) and there are more immediate risk mitigation strategies that can be employed to help in this situation. But I would encourage all companies to have put together a well thought through pre-approval strategy the moment promising data emerges, assuming the treatment is addressing an area of high unmet medical need e.g. oncology, rare disease.

3) How does and which federal regulation policies impact your work, and what positive changes do you hope to see in the near future?

The FDA expanded access regulations along with other pre-approval access routes around the world provide a framework for the work I undertake on behalf of Pharma and Biotech Companies. These regulations are continuously evolving and being updated (Italy Compassionate Use regulation was amended last week). In almost all cases, the changes and updates are providing a clearer and more defined route for access encouraging companies to allow for patients to access their investigational treatments if appropriate and under the right conditions.

The U.S. is the epicentre of focus in the space due in part to the political debate surrounding earlier access to medicines and the Right to Try movement. This debate, alongside the changes within the 21st Century Cures Act, has a major impact on my work in many forms (which I look forward to discussing at the FDA/CMS summit).

I hope to see further enhancements of regulations within the FDA to encourage Pharma and Biotech companies to provide access to desperately ill patients where the benefit/risk ratio stacks up. I would like to see mandatory early stage clinical development discussions with the FDA and companies on pre-approval access strategies and I already see a move towards this.

Interested in learning more?

Join us this December to hear more from these experts, and their colleagues, about what industry shifts and regulation policies we can expect in the near future.