PharmApprove Panel at BIO Addresses Success in Drug Development Beyond Regulatory Approval

Lawrenceville, NJ (May 20, 2015) – PharmApprove – the leading strategic, regulatory and scientific communication consultancy to the pharmaceutical and biotechnology industries – will be presenting an interactive discussion on market access during the 2015 BIO International Convention in Philadelphia, PA. PharmApprove’s session, titled “Approval ≠ Commercial Success: Start Thinking Like a Payer,” will take place on June 16th at 10:15 am.

Dr. Laurie Smaldone, PharmApprove President and CSO, will moderate the group, which includes Steffen Thirstrup, PhD, Director of NDA Group’s Regulatory Advisory Board and former Head of Division at the Danish Health and Medicines Authority (former Danish Medicines Agency) and Coos de Graaf, PharmApprove Vice President, European Operations and former EVP and Head of Global Development at Solvay Pharmaceuticals.

According to Dr. Smaldone: “With this panel, we want to ensure that companies don’t waste their development dollars on products that will ultimately have no standing in the marketplace. Regulatory approval is a huge success benchmark, but it’s just as important to make sure that approved medications are positioned for optimal reimbursement. That’s why we want to help biotech companies anticipate what payers are going to want to know, and make sure those considerations are included in the development plan from day one.”

Prior to joining PharmApprove, Dr. Smaldone was Senior Vice President of Global Regulatory Science at Bristol-Myers Squibb, and also served as CEO of an early-stage inflammation company. She received her MD at Yale University, where she completed her residency in Internal Medicine and fellowship in Medical Oncology. Panelists Thirstrup, who is also an adjunct professor in pharmacotherapy, and de Graaf also have extensive experience with both clinical development and payer negotiations.

“The question of how to anticipate payer concerns is applicable to large and small biotech companies, all over the world” said de Graaf. “Any company’s survival is contingent on being commercially successful. And if you’re not inviting all of the necessary voices to the table early, you might wind up overlooking considerations that not only support approval but valuation until it’s too late.”

PharmApprove’s panel will begin with an overview, before moving into an interactive discussion involving several real-life case studies that showcase some of the pitfalls that can occur when payer concerns aren’t addressed early enough. Audience participation is encouraged throughout. Participants will leave with a clear understanding of the differences between designing programs for regulatory approval and for commercial success – and how to achieve both.

About PharmApprove: PharmApprove is the leading strategic, regulatory, and scientific communications consultancy to the pharmaceutical and biotech industries. The firm offers both strategic and tactical support to companies facing high-profile, high stakes events and engagements anywhere along the road to approval and commercialization, helping them win health authority approvals, deliver compelling regulatory communications, and make persuasive pharmacoeconomic arguments to payers and HTAs. Learn more at

Join Us June 14th – Preparing for a US FDA Advisory Committee Meeting

PharmApprove Communications Coach Lisa Peluso to present at DIA’s Annual Meeting

“Preparing for a US FDA Advisory Committee Meeting”

June 14, 2015 1:00PM – 4:30PM

DIA Annual Meeting
Washington, DC
Walter E. Washington Convention Center

Learn more

The Seven Deadly Sins of Regulatory Communications

 By Dr. Laurie Smaldone 

For many companies, effective regulatory communications can be a cause for worry. It’s necessary for approval but, like the weather, seems largely out of your control.

Maybe you realize only too late that the agency wanted a different set of endpoints. Or maybe you see in hindsight that you needed different subgroups.

Bottom line, you end up making mistakes that lead to setbacks and delays that could’ve been avoided with a more open and proactive approach. We think of these as the Seven Deadly Sins of regulatory communications. What are they?

  • Talking too much/listening too little. You can’t hear what the FDA is saying if you’re delivering a monologue. Sometimes, companies feel they need to throw the kitchen sink at the agency and overwhelm them with data as a precaution. A better approach is to take a deep breath and really try to hear the agency’s advice.
  • Not understanding the whyA consequence of the first. If you listen too little and don’t put yourself in their shoes, you won’t understand the reasons behind the FDA’s advice.
  • Failure to establish a credible, transparent, collaborative relationship. The FDA is not your enemy. They share your goal of helping patients. Work with them.
  • Not being prepared for the unexpected. Positive or negative, regulatory communications is a game with curveballs. Through careful planning and active communication with the FDA, you give yourself a chance to make the adjustments that could lead to approval down the line.
  • Failure to explicitly ask the tough questions. Address them head on. Ask the hard questions on your own—early, openly, and honestly.
  • Arguing every issue. Instead, negotiate and pick your battles, knowing you can’t win ‘em all. Recognize what matters and focus on that.
  • Allowing differences in interpretation to persist. On your own team or in dialogue with FDA, you need to clarify ambiguities in the data or in agency guidance.

I’ll be talking more about these issues—and offering tips on how to avoid mistakes in regulatory communications—at BIO 2014 in San Diego on June 25. I’ll be joined by Dr. Eric Brass, Professor of Medicine, David Geffen School of Medicine at UCLA and Director, Harbor-UCLA Center for Clinical Pharmacology; Dr. Marianne Mann, Consultant and former Deputy Director, FDA Division of Pulmonary and Allergy Drug Products; and Dr. David Essayan, Executive Vice President, ONCORD, Inc. and former Supervisory Medical Officer, FDA.

We hope our session “Are You Really Hearing What the FDA is Saying? Lessons Learned in Regulatory Communications” will help people avoid some the major pitfalls of communicating with FDA. After all, regulatory communications excellence is central to success!

PharmApprove Client MannKind Achieves Positive Votes from Key FDA Advisory Committee

Princeton, NJ (April 3, 2014) — PharmApprove, the leading strategic, regulatory, and scientific communications consultancy to the pharmaceutical and biotech industries, congratulates client MannKind Corporation on its successful U.S. Food and Drug Administration (FDA) Endocrine and Metabolic Advisory Committee meeting this week.

The committee voted 13 to 1 on April 1 to recommend that Mannkind’s AFREZZA® (insulin human [rDNA origin]) Inhalation Powder be granted marketing approval by the FDA to improve glycemic control in adults with type 1 diabetes and voted 14 to 0 to recommend that AFREZZA be granted marketing approval by the FDA to improve glycemic control in adults with type 2 diabetes. If approved, AFREZZA would be the first ultra rapid-acting mealtime insulin therapy available in the United States.

“PharmApprove is proud to have partnered with and supported the AFREZZA team as they presented a clear and compelling case for approval of this new insulin therapy,” said Laurie Smaldone, MD, President and Chief Scientific Officer of PharmApprove. “Our passion is working with R&D teams to prepare for key milestone regulatory events and hearings. We couldn’t be more pleased with the positive committee vote for AFREZZA.”

The FDA is not bound by the Advisory Committee’s recommendation but will consider its guidance in reviewing the New Drug Application (NDA) that was submitted for AFREZZA. The Prescription Drug User Fee Act (PDUFA) date for the FDA to complete its review of AFREZZA is April 15, 2014.

Regulatory Communications Made Accessible

By Dr. Laurie Smaldone

Every phase of drug development has its challenges and thrills. Most exciting for me is the moment when a company begins the conversation with regulators. Why? That conversation is a vital step on the journey toward commercialization, a moment when researchers must persuade an audience outside their company that a drug or device enhances public health. It’s a unique challenge. Scientific teams, long accustomed to research, must quickly turn their attention to communication to demonstrate how their data satisfies regulatory guidelines.

While communicating with regulators is certainly routine and required, in my experience, it is one of the more misunderstood aspects of product development and often a missed opportunity. For many, the regulatory approval process remains mysterious, a black box on the way to commercialization.

PharmApprove aims to make the world of regulatory communications a little more accessible through monthly blog posts that address its key challenges. One of those challenges, for example, is learning to see evidence through a regulator’s eyes, a mental gearshift vital to success. To help address such challenges, PharmApprove’s experts will offer advice that improves scientific communications skills, clarifies current regulatory issues, and provide tips about presenting scientific data persuasively.

Over the years, PharmApprove has developed expertise and tools to prepare teams for US Advisory Committee and CHMP hearings. We have since expanded our services to help scientific teams achieve success all along the development process and navigate the complexities at important regulatory milestones. These complexities range from deciding how to weigh conflicting advice from regulators to pressure testing phase 3 plans. At every stage our passion is to help scientists develop the confidence, clarity, and skills for successful regulatory interactions.

Together, PharmApprove professionals have hundreds of years of industry experience. Individually, they represent a variety of skills and specialties that range from statistical acumen to clinical disease area knowledge to coaching and scientific communications expertise. In my own career, for example, I’ve led the commercialization of drugs in a variety of disease areas from cancer to HIV/AIDS to metabolic disorders.

PharmApprove is an acknowledged leader in strategic, regulatory, and scientific communications, and in future posts, my colleagues and I will share our expertise and experience with you. Our hope is that these insights will help colleagues to stand in front of regulators with greater confidence. After all, we have the people, processes, and passion to help you win.

Dr. Laurie Smaldone is President and CSO of PharmApprove. She has 25 years of executive leadership experience in drug development and regulatory strategy across all major therapeutic areas and product technologies. She has guided global teams through over 100 US FDA and European EMA proceedings, including preparations for key FDA meetings, Advisory Committee hearings, and EU Scientific Advice and Oral Arguments. Dr. Smaldone is trained in medical oncology and received her MD degree at Yale University.

PharmApprove Leaders To Present on FDA Advisory Committee Preparedness at Washington, DC Conference

Washington, D.C. (PRWEB) February 05, 2014

Two leaders from PharmApprove – the leading strategic, regulatory and scientific communication consultancy to the pharmaceutical and biotechnology industries – will present this week at the 5th Annual “Effective Preparation for FDA Advisory Committee Meetings” conference organized by CBI, on Thursday, February 6th. Pete Taft, founder and CEO, and Dr. Laurie Smaldone, President, will lead an Interactive Mock FDA Advisory Committee Meeting for conference-goers.

Taft, a communications consultant and former journalist, founded PharmApprove in 1999, in order to help pharmaceutical clients successfully face regulatory committees convened by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Smaldone, PharmApprove President since 2011, has 25 years of leadership experience in drug development, regulatory strategy, and regulatory approvals across all major therapeutic areas and product technologies.

Taft and Smaldone will offer insights on how participants can prepare for an Advisory Committee, and showcase best practices for facing tough questions. Later at the conference, Taft will moderate a discussion on “Improving and Optimizing Vendor Selection.”

CBI, an Advanstar company, is a life sciences industry leader in providing conference platforms that facilitate innovation, collaboration and elevation across the pharmaceutical, biotechnology, medical device and healthcare industries.

About PharmApprove:

PharmApprove is the leading strategic, regulatory, and scientific communications consultancy to the pharmaceutical and biotech industries. The firm offers both strategic and tactical support to companies facing high-profile, high stakes events and engagements anywhere along the road to approval and commercialization, helping them win health authority approvals, deliver compelling regulatory communications, and make persuasive pharmacoeconomic arguments to payers and HTAs. Learn more at

PharmApprove congratulates Chelsea Therapeutics

PharmApprove congratulates Chelsea Therapeutics on their successful Cardiovascular and Renal Drugs Advisory Committee meeting on January 14, 2014. The committee voted  16 -1 to recommend  approval for Northera™ (droxidopa) for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with primary autonomic failure (Parkinson’s disease, multiple system atrophy and pure autonomic failure), dopamine beta hydroxylase deficiency and non–diabetic autonomic neuropathy.  Northera was previously granted Orphan Drug Designation for this rare disorder that affects about 300,000 patients in the US and EU. 

PharmApprove, Leading Regulatory and Scientific Communications Firm, Expands in Europe with Coos De Graaf Named New Head of European Operations

PharmApprove expands their services and geographic reach with an expansion in Europe and the hiring of a new Principal.

Lawrenceville, NJ  (December 17, 2013) PharmApprove, a division of Taft and Partners, announced today that it has expanded its geographic reach by opening a European Office. Coos De Graaf has joined the firm as Principal, Commercial Operations in Europe, signaling a new push towards expanded services and geographic markets. De Graaf has more than 30 years of experience in the pharmaceutical industry, including numerous roles in senior management.

PharmApprove is a leading strategic, regulatory and scientific communication consultancy to the pharmaceutical and biotechnology industries.

Mr. De Graaf will lead PharmApprove’s efforts to help companies successfully navigate the product approval and value demonstration process throughout Europe. With his leadership, PharmApprove will be able to offer expanded services to clients as they prepare to bring new therapeutic agents and devices before regulatory and health technology assessment agencies.

“PharmApprove has established client experience in Europe, but adding a leader of Coos De Graaf’s caliber positions us extremely well to continue growing to serve our clients globally,” said Laurie Smaldone, M.D., President of PharmApprove. “In addition we are excited to announce expanded services building upon our success in supporting product approvals to include market access communication support.”

Mr. De Graaf led various executive functions at Solvay Pharmaceuticals including global compliance and clinical and commercial development. Other past accomplishments include developing the European presence for the Campbell Alliance .

“I am excited to join PharmApprove, which has a long track record of success working with pharmaceutical and biotechnology companies,” said De Graaf. “There are tremendous opportunities to extend our expertise in Europe, both in the development and post-approval stages.”

Historically, PharmApprove has helped drug developers effectively communicate to regulators and advisory panels in the US and Europe. With the company’s new expansion, it has not only strengthened its European capabilities but fully built out three main pillars of focus:

  •     Advisory Committee Preparation
  •     Regulatory Communication
  •     Market Access Communications Support

PharmApprove has relaunched its web site – — with full background on its services and Principals.

About PharmApprove:

PharmApprove is the leading strategic, regulatory, and scientific communications consultancy to the pharmaceutical and biotech industries in the US, Canada and Europe. The firm offers both strategic and tactical support to companies facing high-profile, high stakes events and engagements anywhere along the road to approval and commercialization, helping them win health authority approvals, deliver compelling regulatory communications, and make persuasive pharmacoeconomic arguments to payers and HTAs.

PharmApprove President Laurie Smaldone Honored At EWNJ’s “Salute To The Policy Makers”

New Brunswick, NJ (April 24, 2012)  – Executive Women of New Jersey (”EWNJ”) will honor Dr. Laurie Smaldone at its Salute to the Policy Makers 2012 Awards Dinner on May 17, 2012, at the Hyatt Regency New Brunswick in New Brunswick, NJ. Currently in its 32nd year, EWNJ holds the biennial fund-raising event to recognize women of professional distinction in New Jersey.

“Since its founding in 1980, EWNJ has championed the advancement of women to the most senior levels of international corporations, non profits, and public service firms. At this year’s ‘Salute to the Policy Makers’ event, we will celebrate the professional accomplishments of 36 exceptional New Jersey executives,” said EWNJ President-Elect and 2012 Salute Dinner Chair Keitha M. Lackey, who also serves as a Director for Horizon Blue Cross Blue Shield of New Jersey. “It is my honor and pleasure to serve as Chair for this year’s gala, which serves a dual purpose: Honoring an outstanding group of women and benefiting an excellent cause.”

Dr. Smaldone’s career began with Internal Medicine and Medical Oncology training at Yale University, which led her to a position at Bristol-Myers Squibb in clinical cancer research, advancing development programs through to registration. She then held a range of positions of increasing responsibility and scope in multiple disease areas, including Executive Director, HIV/AIDS; Vice President, Infectious Diseases; Senior Vice President, Global Regulatory Science; and Corporate Vice President, Business Risk Management. Dr. Smaldone was most recently CEO of a clinical phase biotechnology company, prior to joining PharmApprove as President in 2011.

Proceeds from the biennial event will fund EWNJ’s scholarship program, which has provided over $1.1 million in scholarships to more than 350 deserving New Jersey women enrolled in advanced degree programs at New Jersey institutions since instituting the program in 1986, as well as other programs that advance women.

For more information on corporate sponsorship or tickets to the Salute to the Policy Makers awards dinner, contact EWNJ at 609-249-7982 or visit EWNJ on the web at

Facing the FDA? This Princeton Firm Has Your Back

by Scott Morgan | Reprinted from the November 9, 2011, issue of U.S. 1 Newspaper 

You have a groundbreaking drug that has just completed the development phase. Now you have to prove it to the federal government.

What you will have to get past are thousands of pages of data written in scientist language and a panel of experts representing the Food & Drug Administration.

The members of this panel do not want to hear jargon. They want clear, concise responses that will prove you know what you’re talking about. And, like a presidential debate or the Miss America pageant, any stumbles in your response can have disastrous effects on your chances of coming out on top.

Enter PharmApprove, a pharmaceutical communications company that specializes in getting drug companies through the last — and usually most nerve-wracking — step of the regulatory approval process. The company is a division of Taft & Partners, a communications firm based at 1 Palmer Square. PharmApprove is run by Pete Taft, the CEO of both companies.

The son of an executive with Rohm & Haas, Taft went to Dickinson College, Class of 1973, and worked as a sports writer in Santa Monica and a reporter in Vermont, with a stint teaching English in between. While freelancing for glossy magazines in New York he met his future wife, Mara Connolly, who was working at big ad agencies. Taft switched careers and opened Taft & Partners in 1999.

PharmApprove was launched in 2006 and in September hired Laurie Smaldone as its new president. Smaldone comes to PharmApprove from Bristol-Myers Squibb, where she spent 20 years in clinical development.

“When a drug goes through the regulatory process,” Smaldone says, “it’s a 10-year ordeal. At the end an organization has to translate thousands and thousands of pages of data into short statements. And be prepared for some really tough questions.”

The trouble companies run into during the pre-approval phase, Smaldone says, is that presenters — often scientists — tend to fall in love with their data. Rather than answering in English, they spend a lot of time regurgitating highly technical information.

Or a lot of presenters fall into what Smaldone calls the “slide Olympics,” a tendency to put all the information on slides and use them to answer every question asked.

But slides, she said, should be supportive, not used as responses. An FDA panel wants to hear from a presenter, not be inundated with slides.

What separates FDA panel presentations from the myriad presentations drug companies make during the investment and development phases is that FDA panels are non-scientific public hearings. “Everything is confidential until it’s not,” Smaldone says. And when you face the FDA, all your research, information, and secrets are open to an audience of interested citizens, journalists, potential investors, and your competition.

PharmApprove spends 20 to 24 weeks preparing presenters for what Smaldone calls “a very formidible event.” Everything from helping a presentation team organize its thoughts and breaking down jargon to coaching presenters on how to answer and how to stay poised.

Nerves, Smaldone says, can get the better of otherwise prepared people when they realize the weight the FDA presentation carries. After several years of discovery, development, testing, and investment, the entire operation can come to an immediate halt if the FDA doesn’t think you have good enough answers.

The last thing a company wants is a credibility problem when it steps up in front of a panel. Part of PharmApprove’s coaching is getting presenters to anticipate the questions a federal advisory panel might ask — say about side effects. “No drug has no side effects,” Smaldone says. “It’s a matter of weighing benefit versus risk.” PharmApprove helps companies understand how to present risk data so that it shows the benefit a drug will have for a particular group (stroke patients, for example) without creating undo risk.

And are there different data sets for different sub-groups? Does the drug affect men and women differently? Or older people versus younger?

A risk management plan needs to account for questions like these, and if a presenter trips over an answer, or the information is considered too slight, a promising drug can die on the panel floor. “We help rehearse teams,” Smaldone says. “But ultimately the data is the data.”

Smaldone views the FDA process as a production not unlike theater — and she should know, having been a dancer in high school and college. But when she got to Yale she found that medicine was her calling. She earned a bachelor’s in biology and philosophy in 1975 and stayed at Yale, earning her M.D. in 1979.

Smaldone was a practicing oncologist and Yale faculty member for a few years. Her original plan was to learn about clinical trials from the industry side, rather than from the medical side. But she quickly became hooked by cutting edge research happening in the pharma industry. She left Yale to take her job at Bristol-Myers Squibb.

After BMS Smaldone briefly served as president of a now-defunct pharma company named PhytoMedics, then moved into consulting with PharmApprove, already familiar with some of the most common trip-ups presenters deal with. PharmApprove offers a list of tips for anyone facing an advisory panel.

Make the panel the number one priority. Preparation for an advisory panel can take up as much as 80 percent of your team’s time. Don’t try to cram for a presentation.

Also, don’t confuse the agency with the panel. The agency will be there, but it’s not the one you need to impress. Consider not what the agency wants but what the panel needs to hear.

Build teams on performance ability. Developing a drug takes a lot of work from a lot of people over a long time. But not everyone in the development process is suited to give presentations.

Follow the axiom: “The best scientists don’t necessarily make the best presenters.” Assemble your team around who can deliver the message best, not around who put in the most hours.

Work from scripts. Don’t read from the script, but know what the script says. If it is your road map, you will not lose your way during your presentation.

You will win or lose in the Q&A. Making a succinct, informative pitch is one thing. But the panel is going to ask questions, and they’re not going to be easy ones. Learning to anticipate questions and having ready answers can spell the difference between approval and flop.

Embrace the critics early. Outsiders can offer you valuable insight by asking tough questions. Embrace this, and get the feedback early while you still have time to work on the problems.

KISS works. Keep your presentation simple. One-point-per-slide kind of simple.

Panelists are busy people uninterested in complicated technical jargon. PharmApprove recommends taking Napoleon’s advice to his troops — “Be clear, be clear, be clear.”

Rehearse, rehearse, rehearse. When you’re done doing all that, rehearse again. Know the material inside-out and upside-down. After all, Smaldone reminds, “You only get one chance.”