the fda and industry: a recipe for collaborating in the new health economy

February 19, 2015

The FDA and industry: A recipe for collaborating in the New Health Economy

PwC

About this report

As part of its Improving America’s Health series, PwC has periodically surveyed industry executives on their relationship with the FDA.

In summer 2014, PwC’s Health Research Institute and Biocom polled 100 senior executives on issues such as regulations, the development of new therapies, and patient engagement.

1000 adult consumers were also surveyed about their views of the FDA, the pharmaceutical/life sciences industry and consumer access to therapies.

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Key findings

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Executives say that the FDA has become a more communicative and open partner

Executives understand that balancing innovation and risk comes with tradeoffs

Industry attitudes are shifting when it comes to determinations of value

Consumers want more engagement from both the FDA and industry4

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21st Century R&D vs. the Current Paradigm

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An evolving regulatory process

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Source: 2014 HRI Pharmaceutical and Life Sciences Executive Survey


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The FDA’s budget relies heavily on industry fees

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The FDA’s standard review times have decreased

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Priority reviews have also improved

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The FDA has become a better communicator…

Steps taken by the FDA to improve working relationships with companies

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Source: 2014 HRI Pharmaceutical and Life

Sciences Executive Survey

Better quality and more frequent communications with the FDA helps executives anticipate the agency’s expectations and satisfy regulatory requirements


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But executives still question the value of user fees

Areas that executives believe have improved the most as a result of increased PDUFA/MDUFA fees

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Just 32% thought the fees they paid have resulted in shorter review times and only 34% think the fees help accelerate innovation


Sciences Executive SurveyThe FDA and industry: A recipe for collaborating in the New Health Economy

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Industry wants the FDA to prioritize innovation

Areas that executives believe have improved the most as a result of increased PDUFA/MDUFA fees

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Only 34% of executives think user fees help accelerate innovation



User fees accelerate innovation


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Experts say…

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“Science is advancing so quickly, it’s difficult for any single organization to stay on top of everything. We need a system in the 21st

century that allows regulators to access the scientific expertise they need, both internally and externally, in a timely manner.”

- Stacy Holdsworth, senior advisor of US regulatory policy and strategy at Eli Lilly & Company


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Experts say…

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- Andrew von Eschenbach, former FDA commissioner

“You have to recognize that the user fee program is only addressing the review cycle and that’s not the issue. Review is only one piece. You need a much broader view. The issue is the process from the very beginning of development to the post market. And the FDA influences that entire process.”


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Experts say…

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- Fred Hassan, former CEO of Schering-Plough who now serves as a managing director at Warburg Pincus

“The FDA needs to be part of the innovation engine. I don’t think they currently see that as a primary role”


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Changes are already being implemented

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Executives are less familiar with new programs

Familiarity with accelerated approval programs




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Case Study: Breakthrough Therapies

Impact of new review models on the market

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Case study: Breakthrough therapies

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“I’m talking to the FDA every week about IMBRUVICA. My experience with the FDA, as part of that process, has been overwhelmingly positive. That doesn’t mean issues never arise, but I’m happy because my expectation of the FDA is that we can work through those issues and get them resolved, before they become larger issues.”


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“I’m talking to the FDA every week about IMBRUVICA. My experience with the FDA, as part of that process, has been overwhelmingly positive. That doesn’t mean issues never arise, but I’m happy because my expectation of the FDA is that we can work through those issues and get them resolved, before they become larger issues.”

- Urte Gayko, senior vice president of global regulatory affairs at Pharmacyclics on her experience managing Imbruvica, a breakthrough therapy drug


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“It does seem to be changing the way the agency is doing things. If this is a promising direction, we need to ask what else needs to change along with it. The breakthrough process is focused on drug review and approval but there are other things that need to get done at the same time such as review of good manufacturing processes.”

- Mark McClellan, former FDA commissioner and current senior fellow and director of the Health Care Innovation and Value Initiative at the Brookings Institution


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Rebalancing Risk vs. Innovation

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Consumers are willing to take on more risk

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Respondents overwhelmingly agreed that patients with life-threatening conditions should be able to access experimental therapies.



Sciences Consumer Survey

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Executives support consumers’ desire for access

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77% of executives agree that consumers willing to assume grater risks should have access to experimentaltherapies.




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But speedier access may require new thinking

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“We’re looking at outcomes data and how it should influence regulatory decisions.”

“Quickness to market is clearly a basic principle. With that comes an even greater need for post-market surveillance.”

- Stacy Holdsworth, senior advisor of US regulatory policy and strategy at Eli Lilly & Company

- Ray Woosley, president emeritus of the Critical Path Institute and founder of CredibleMeds – an organization focused on safe medication use.


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The Value Factor

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Experts say…

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“We don’t want to squelch innovation, but tell me what I'm getting for my healthcare costs. Show me that these new technologies are superior.”

- Scott Josephs, national medical officer at Cigna Corp. at an Advanced Medical Technology Association meeting on October 2014


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Executives attitudes are shifting on value

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Global experiences may shape industry views on value

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HRI’s survey suggests harmonization of regulatory requirements would make it easier, and faster, to bring a new drug to market worldwide.



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Cost is not considered

at all

Only if a patient

asks

To a lesser degree

To some degree

To a significant

degree

Cost is a top consideration for prescribers

This matters to consumers, who rank a physician’s recommendation as the most important factor that influences their treatment decisions, followed by their own out-of-pocket costs.

1% 2% 5% 38% 54%

Is cost a factor when deciding whether or not to prescribe a drug?

The FDA and industry: A recipe for collaborating in the New Health Economy Source: 2014 HRI Clinician Survey

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Cost-conscious consumers also seek value

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% of Americans deciding not to purchase medical services due to cost

Willingness to pay more for a personalized medical product



Sciences Consumer Survey

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Bringing in the Consumer

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Experts say…

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“Today’s consumers make decisions in conjunction with medical authority, not because of a medical authority.”

- Ido Hadari, CEO of Treato, in an interview with HRI


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Experts say…

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“Having a seat at the table is important, but how do you make sure the patient perspective can be turned into data points that can inform and influence the review process? We’re talking about quickly moving from a lofty goal of making patients part of the system to a very technical conversation about how to make it happen.”

- Cecilia Arradaza, Executive Director of the Milken Institute, which includes FasterCures -an organization focused on speeding medical innovation


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Experts say…

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“I’ve told industry that patients can contribute to the conversation. That the patient voice is important earlier in the process, such as how clinical trials are designed.”

- Diane Dorman, vice president of public policy for the National Organization for Rare Disorders


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Experts say…

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“No patient community has ever done this and it sets the stage for new partnerships with the FDA.”

- Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, explained how her group created the first patient centered guidance document on clinical trial design.


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Case Study: Priority review vouchers

Innovative methods to promote research in rare diseases

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“The question isn’t how to get a drug approved. The question is how you get a private company to make the drug in the first place.”

Case study: Priority review vouchers

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- Nancy Goodman, founder and executive director of Kids V Cancer, a patient organization that promotes pediatric cancer research.


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Future Considerations

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Future considerations

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Prioritize consumer input

Demonstrate value to regulators and purchasers

Seek new alliances

Pursue new opportunities to minimize risk and accelerate access

Enrich the dialogue


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Prioritize consumer input

Companies should seek out consumers’ views, diligently incorporate their input at every stage and share this information with the FDA.


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Demonstrate value to regulators and purchasers

Manufacturers will need to demonstrate value over existing therapies as both purchasers and consumers look for products that meet their needs.


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Seek new alliances

A greater reliance on public-private partnerships could provide a forum for the exchange of ideas. For instance, the Medical Device Innovation Consortium coordinates efforts of industry and regulators to develop new tools, methods and technologies.


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Pursue new opportunities to minimize risk and accelerate access

Pharmaceutical and life sciences companies should partner with the FDA to facilitate early access to experimental drugs for eligible patients with articulated risks. Continued investment in new tools and technology, such as biomarkers, may help manufacturers and regulators assess risk more accurately and quickly.


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Enrich the dialogue

Communicate more often and effectively with the FDA. Doing so will allow companies to build stronger relationships with agency officials, including the reviewers tracking their products. These interactions can help companies understand the agency’s thinking and compile stronger applications to avoid multiple review rounds.


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Questions?

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Contacts

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Michael MentesanaPrincipal, Pharmaceutical and Life Sciences(646) [email protected]

Chandresh HarjivanPrincipal, Pharmaceutical and Life Sciences(202) [email protected]

Bobby ClarkSenior Manager, Health Research Institute(202) [email protected]

mailto:[email protected]



Thank you

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