Has Digital Finally Come of Age for Pharma?

Pharma can’t really afford not to go digital. With ever-increasing healthcare practitioner (HCP) and patient usage of digital technology, the ‘norm’ for marketing and communications must completely change. Digital should take the lead role, argues John Hackney.

The proliferation of mobile device and computer usage by HCPs and patients, and therefore the need for pharma to leverage digital channels, is growing exponentially. Manhattan Research recently found that 26% of European doctors own an iPad, and they spend more than a quarter of their professional online time on that device. The same research also found that iPad-owning doctors use desktop and laptop computers for 55% of their professional Internet use, and smartphones 18% of the time. By the time you read this, the data will have increased even more!

Pharma brands and companies have adopted some types of technology and digital communications, largely in a tactical way. For the most part, pharma hasn’t kept up with its users’ needs. Digital communications are still under-used for HCP campaigns, patient disease awareness and patient compliance. Digital is still secondary, or ‘in addition to,’ rather than ‘instead of,’ offline marketing.

The Digital Reality

Customer needs, digital communications, social networks, media channels, targeting, and opportunities for pharma brands have developed sufficiently in recent times so that a brand can be launched, built, repositioned, and sustained almost entirely using digital channels. Non-digital communications would have a minor supporting sales collateral role.

Before I receive an onslaught of comments along the lines of “yes but regulations means our hands are tied when it comes to digital,” of course there will always be regulations challenges. Approvals take time, and you need to plan accordingly, but they’re not insurmountable. Bring in your legal and medical teams from the outset of any communications programme development, so they become active stakeholders. Get everyone to agree the ‘rules of engagement.’ Plus you can use the time it takes for approvals to your benefit. Your media planner and buyer has time to become a master in creating great relationships and deals with digital media owners in the run up to your campaign going live.

Can pharma really afford not to do go digital? Reaching HCPs and patients online can be around 10% of the cost of doing it non-digitally, from a cost per thousand impressions perspective. Plus high quality open source content management system (CMS) platforms and other software/production techniques mean you don’t have to spend lots of cash building stuff. Instead you can spend money where it counts — reaching and engaging with your audience.

Not only can you move virtually all of your marketing communications online, you’ve also got the potential to do most of your sales support online. Closed loop marketing (CLM) continues to make leaps and bounds from a technological perspective. Your sales force becomes more effective when in front of customers and, more importantly, when they aren’t. It allows customers to engage with the company or brand when they want, accessing what they feel they need. Self (remote) detailing, co-browsing, web-ex, dynamic (intelligent) CMS — all mean that the relationship between customer and company can be rich, fulfilling and one‑to‑one, even on a remote basis.

Digital is virtually limitless in how it can and will be used. For example, we’re developing apps for patient tracking, dosage converters, and other tasks to support and encourage patient compliance. Creating virtual circles between patient disease awareness and HCP education through digital channels is also a growing trend. You tell patients what to look out for, they go to their doctor, you make sure that the doctor is armed with all the information that they need to answer patient questions, and to provide better diagnosis and patient care. Still unconvinced? Here are a few examples of recent campaigns run almost entirely online.

Blockbuster Loss of Exclusivity Campaign

A category-leading blockbuster brand nearing loss of exclusivity (LOE) needed to maintain prescribing behaviour amongst frontline GPs and specialists. Switching from an almost exclusively offline approach, the lion’s share of marketing activity was put behind a multimarket digital marketing programme aimed at relevant HCPs. The aims? To ensure they were fully aware of the benefits of this brand versus future generics on an ongoing basis, and to keep existing prescribers post LOE.

The campaign included a website ‘destination’ with pertinent ‘hooks’ and hard-hitting promotional messages to encourage site exploration and return visits; more detailed content for those that required greater substantiation; highly targeted and adaptable regional and local drive-to-site banner ad campaigns featuring interactive surveys and games, and the use of quick response (QR) codes. The digital media buying strategy meant messages were constantly put in front of the right HCPs for a sustained period of time. Those that wanted to know more were directed to an engaging, content-rich website, giving them the power to access as much information as they felt they needed.

This £1.5-million campaign, including media buying, resulted in a phenomenal 0.5% click through rate via more than 120 banners in nine languages across 11 European countries — that’s around 16 million online impressions during 6 months. The average targeted HCP was exposed to the messaging around 340 times throughout the campaign. The website also had an incredibly low 60% bounce rate.

Now take a moment to consider the creative and media buying cost implications to have such a targeted, yet widespread reach. Can you imagine the cost of 120+ ads in nine languages across 11 countries if they were done as print ads? And how many HCPs would have really paid attention to print ads? Not to mention the fact there’s really no way to measure print ads’ response rate and then revise your campaign messaging and media ‘on the fly’ — a critical aspect of the digital campaign.

Patient Education Campaign Goes Digital

A major pharma company is completely replacing its pan-European television and press patient education advertising campaign with an online marketing and communications programme. The programme is aimed at consumers and is entirely educational — designed to get people who have regularly tried and failed to quit smoking to go to their GPs for help.

Going digital means they can have a proper, highly targeted customer relationship management (CRM) programme for the first time. For example, targeting people via Facebook who are coming up to their thirtieth or fortieth birthdays, people who have just got married, who are changing jobs, or in a new relationship. All of these are considered to be ‘smoking cessation’ triggers.

Then relevant messages are put in front of these people, driving them to a dynamic CRM website, which users ‘opt in’ to. By tracking signed up users, they can regularly send personalized emails based on what people do or don’t do on the site. Ultimately, this is about taking people on a whole journey, from initial awareness through to setting a doctor’s appointment.

This digital programme enables the pharma company to set key performance indicators and benchmarks, and then track and manage the performance of the campaign and messaging in real time to ensure that the objectives are being met. They can weed out messages that don’t work well, or refine site content and the user experience to optimize the programme.

Now consider an offline campaign. This would need to play out in its entirety before you’re able to gauge how well it’s performed. Not only this, but you’re severely limited in the type of tracking that can be done.

Fishing Where the Fish Are

This isn’t about a prescription drug, but it’s an interesting healthcare brand story. SASMAR’s Conceive Plus is a vaginal fertility lubricant designed to assist the path to getting pregnant naturally.

The brand is currently undergoing a complete relaunch to raise awareness amongst a broader target audience in the UK, and it is all being done digitally. SASMAR knows that its prospective customers are highly active in the digital world, visiting online sources of information to improve their knowledge. So logic dictates a focus on digital.

The campaign includes a new content-rich website, with drive-to-site and viral media campaigns aimed at women and couples who are trying to conceive, via search, display, and online PR. Forming the cornerstone of an important social media presence, a Facebook page sends out relevant news and content, and engages in two-way conversation with users. The result is an integrated and enhanced online presence that connects with target customers, guides them through the decision-making process, creates awareness, comprehension, and desire, stimulates and facilitates brand purchase, and engenders brand advocates to spread the message and virally generate incremental sales. SASMAR is able to target all women who potentially want to try to improve their chances of getting pregnant because they’re both reachable and identifiable in the digital world.

There are very few things that pharma companies and brands cannot achieve online in a sales, marketing, or marketing communications sense. Clearly you have to work within the regulations, use digital channels appropriately and understand how to protect yourself from adverse event reporting. But from CLM through to patient compliance and education, there’s virtually no part of a marketing communications campaign that can’t be run on a digital basis these days.

Inspiring Passionate Innovation in Pharma R&D

Transforming the efficiency of their research and development teams is crucial for big pharmaceutical companies these days and they are not alone. Businesses across the board, from banks to supermarkets to telcos, are looking at how their creative engines can deliver more for less. Matt Kingdon and Jon Platt from ?What If! suggest how Big Pharma can learn from their example.

Pharmaceutical R&D teams are facing urgent challenges. Their big brands are tumbling over the ‘patent cliff,’ and the increasing burden of costs on governments and individuals to pay for the

Too often the chosen remedy is a contradictory mix of cost cutting and exhortations to up the output of successful new drugs. This may be painful to hear, but most R&D teams we’ve worked with are comprised of people who are risk averse and analytical, not entrepreneurs. It’s unsurprising to find low morale lurking in these organizations.health care of an ageing generation is hobbling the launch of new drugs. Forced to stretch their budgets across the demands of older populations, health services and regulators are demanding greater differentiation in new products. It’s no longer enough for a new drug to be marginally more effective. It must offer entirely different benefits or radically better results to win approval and command a price premium, and very few can meet the standard.

The crisis has promoted radical new ways of developing drugs, such as GSK’s Discovery Performance Units, which kept innovation in-house but inspired employees to think like entrepreneurs by regularly making them seek funding. But radical solutions take time to take hold. The good news is that many complex, regulated businesses outside of healthcare have increased the efficiency of their innovation engines, and many have implemented hundreds of subtle, inexpensive changes that aggregate to a wholesale change in culture. If pharma R&D teams take a look at these practices and experiment with how they work,

The single most important driver of innovation is passion. It’s a desire to do a great job, to make your customer happy, or to right a wrong. A small team joined with a similar passion is awesome to behold. Sadly, many of us don’t get enough of that at work. Here are three strategies for developing passion in pharma R&D teams.they too can become more agile and productive, even with fewer people and resources.

The Whites of Their Eyes

In the 90s, many packaged goods companies got the insight bug. What had been called market research, a paper-heavy desk exercise, was replaced with hands-on executives who swapped PowerPoint for notepads, got out of the office, and saw how and why their consumers did what they did. They were shopping, eating, and even clubbing with their customers. This adventurous approach to seeing the whites of customers’ eyes was not restricted to marketing. Scientists and technicians left the lab for the living room, too. We have found this approach to be highly effective.

Here’s an example. A major, global pharmaceutical client of ours had received bad news. Its antidepressant drug had failed its stage-two clinical trials, a development with potentially fatal implications for share price. But what did ‘fail’ mean? Digging into the target product profile (TPP) revealed ambitious performance goals across key dimensions of efficacy, side effects, and tolerability. To be successful, this had to be a wonder drug. The scientists had been stuck in the lab too long. They were struggling to connect with the emotions that patients and their caregivers were experiencing.

We travelled across the US to meet people suffering from a wide range of depression. We met a musician whose guitars were covered with dust, a man who hadn’t been outdoors for years, and a painter who was hearing voices as we spoke to her. We met people who were young, old, who had care, and who had no one to turn to. This experience was moving, even for scientists who had studied depression for years.

What the scientists took away from their trip was how crippling the side effects of antidepressants could be. They had read about this, but not witnessed it. One teenage sufferer had told them that full remission wasn’t worth it if she gained weight, stopped sleeping and sweated all day. The TPP demanded efficacy above all else. Patients wanted a more balanced approach.

The scientists went back to the lab and amended the TPP. They rebalanced what they were looking for: a drug with a slightly lower performance measure and less intrusive side effects. And guess what? They already had the molecules to deliver against that TPP. The road trip paid off. The scientists didn’t have to start from scratch, slashing development time by several years—a turn of events predicted to save the company more than $1billion.

Stop Being Sensible and Serious

Most people in business apply an abundance of analysis, objectivity, and skepticism to new ideas. We call these ‘reductive’ behaviours. Because their cultures are based on the scientific discipline of clinical trials, pharma companies are thronged with reductive thinking ninjas. In innovation, reductive thinking is vital when applying commercial judgments to emerging solutions. But without ‘expansive’ thinking, those new solutions will be strangled at birth.

Expansive thinking is what a child does when you give him or her a gift in a box, and they spend the next week ignoring the gift and turning the box into a car or a house. It’s thinking that seeks multiple possibilities rather than one right answer. It’s based on playfulness and positivity: an agreement to build on each others’ ideas rather than knock them down. Since we were all children once, we can rediscover how to think expansively with a bit of practice.

The trick to expansive and reductive thinking (and, therein, the secret to effective collaboration) is to make sure that, when solving a problem, your team knows it’s either thinking expansively and generating solutions or that it’s time to think reductively and move into choosing mode. You’re avoiding situations where these behaviours get mixed up. We’ve all been in meetings where somebody has suggested an idea, and five helpful souls have told him what’s wrong with it in the same moment. And we’ve all been in meetings where you’re trying to make a choice, but others want to open up the discussion to new ideas.

A term called ‘signalling’ is used in our innovation training. It means acquiring the habit of letting those around you know if you need expansive or reductive thinking at that point. Consider a typical exchange:

Scientist A: ‘Hey, why don’t we create a website and release our challenges and data for the world to work on – an open innovation programme.’

Scientist B (raises eyes to heaven): ‘Hmm, we tried that, and it didn’t work.’

This isn’t a healthy dialogue. Now let’s ‘signal’:

Scientist A: ‘Hey, I’ve got an idea, but it’s only half-formed. Can you help me build it? Why don’t we create a website and release our challenges and data for the world to work on – an open innovation programme.’

Scientist B (realising an expansive response has been requested): ‘OK, let’s explore. We could do that, but we’ve not had a lot of luck. Your idea makes me think we should hold a conference and invite a small number of leading researchers to collaborate. Now there’s an idea I hadn’t thought of!’

Introducing planned expansive and reductive thinking to pharma R&D teams, managed with clear signaling, transforms a team’s ability to collaborate and innovate.

Science Fiction, Not Science Fact

One of the biggest challenges facing pharma R&D leaders is knowing where to place strategic bets that won’t pay off for 10 to 20 years. Smart organizations are learning that approaches they used to rely on for future-scenario development don’t work anymore. They have to think like science fiction writers, not rational analysts, and develop scenarios to place bets that are leaps ahead of what the competition is seeing.

To ensure the bets they place are smart, we developed an approach called ‘foresight.’ It marries future certainties, such as demographic change, with collaborative imagining events to produce scenarios with a 10-20-year horizon.

Recently we used this approach for a major fuels and lubricants manufacturer. We sought to broaden the question beyond current paradigms, engaging experts and academics outside the category. In this case, the usual question might have been, ‘What’s the future of lubricating oils?’ But asking that question ensures that answers remain inside the current business model. So we asked, ‘What’s the future of mobility?’ We gathered anyone who had anything to say on the future of mobility. Our R&D scientists heard from travel writers, airline stewards, chauffeurs, people with restricted mobility, road planners, and even someone who claimed to have invented a teleportation device. Immersing themselves in a future world of possibilities in which their product or brand may play a role stretched the team’s thinking. The company is now allocating multi-million dollar investments in future mobility worlds that were not obvious at the outset.

We’ve mirrored this approach with other pharma R&D teams. In a recent exercise looking at compliance in respiratory medication, we flipped the challenge from ‘how to comply’ to ‘how to create desire.’ We introduced scientists to fashion and perfume houses, and they realised that the formulation development of, say, inhalers was less important than the aesthetics. This prompted a shift in investment away from formulation research and toward the development of cool-looking devices, which had shorter lead times and fewer regulatory hurdles.

Seeing the whites of patients’ eyes, signaling intent, and swapping sci-fact for sci-fi are simple, low cost, and even fun tools for injecting passion – and impact – into innovation.

World’s Pharmaceutical Development Manufacturing Base Moving to India

The India Brand Equity Foundation (IBEF) has compiled its findings and released an overview of the current generics market in India. They predict that the Indian pharma market is now at the precipice of the next stage in its development, having seen manufacturing innovation and development technologies rise- thanks to the explosion in generics production.

During the last 3 years, exports of pharmaceuticals (largely generics) have grown at over 21.5% (CAGR) and now accounts for over $13bn in annual sales. Highlighting India’s dominance, nearly 40% of Abbreviated New Drug Applications (ANDA) received by the FDA in 2012 were from India, with a further 87 confirmed and another 25 already received between January and June 2013.

This huge growth in generics production has seen the country become a hotbed of manufacturing innovation – India has over 3000 DMFs registered with USFDA – which coupled with increased investments in R&D means India is now ready to challenge traditional big pharma and start producing more patented products. A natural evolution of the success of the generics market has been the rise in supergenerics across India where much R&D spend is currently being invested (e.g. Lincoln Pharma’s patent for NDDS).

With the world’s pharmaceutical development manufacturing base moving to India – there are 546 USFDA approved company sites (second only to the US), 23 companies holding 1100 authorizations with UK’s MHRA, and 166 companies with CEPs (Certificates of Suitability) from EDQM – coupled with the rise in supergenerics, the country’s next natural step is to use its world leading development expertise in the creation of new chemical entities.

Recognising this opportunity, the Government of India is putting in place supportive initiatives with the goal of cementing the country’s position as the ‘pharmacy of the world’ and creating a global innovation hub. With generics predicted to rise to 35% of global pharmaceutical market value by 2016 (some $400bn+), and with an annual growth rate of 27% amongst Indian generics exports (comparing very favourably with the global average of 10%) the Government and Pharmexcil are forecasting much of this revenue will be reinvested across the country in new research- leading to a steady pipeline of future drug targets.

In support of this, the Indian Government has committed to three schemes including a major multi-billion dollar initiative with 50% public funding through a public-private partnership (PPP) model to harness India’s innovation capability. In addition, the Government has made tax-breaks available to the pharmaceutical sector and a weighted tax deduction of 150% for any R&D expenditure incurred. Additionally, they have also introduced 19 dedicated Special Economic Zones to help stimulate pharma sector investment across the country.

Steps have also been taken to streamline procedures covering development of new drug molecules and clinical research- including two schemes ‘New Millennium Indian Technology Leadership Initiative’ and the ‘Drugs and Pharmaceuticals Research Programme’, which has been specially targeted at drugs and pharmaceutical research.

Already this year, India’s Dr. Reddy’s, Lupin Labs, Sun Pharma, Ranbaxy and Cipla have invested over $500million in R&D, which is allowing increased innovation in manufacturing processes  and will ultimately help to lower the cost of medicines production.

“India, termed as the Pharmacy of the World, has a basket of wide spectrum of generics that are second to none in terms of quality. The industry is on the track to expand its reach to newer markets, which makes it equally critical for the Indian pharmaceutical industry to keep its focus on quality, affordability and accessibility of medical solutions for the global pharma market. The country’s success in generics manufacturing is helping to keep our industry at the forefront of innovation and over the next few years we are lending our support to the R&D effort across the country so that we are leading in generics production and even developing new drugs out of India” said Mr Rajeev Kher, Additional Secretary, Department of Commerce, Ministry of Commerce and Industry, Government of India.

“Being a world leader in generics, India already has a huge presence in the highly regulated markets in terms of pharma exports. Almost two thirds of Indian generic exports are to the highly regulated markets (e.g. the US and Europe), which speaks volumes about the quality of Indian medicines. The Government of India is supporting Brand India Pharma campaign to reiterate that the Indian pharma market offers credible, affordable and sustainable healthcare solutions” said Dr P.V. Appaji, Director General, Pharmexcil.

About IBEF: India Brand Equity Foundation (IBEF) is a Trust established by the Department of Commerce, Ministry of Commerce and Industry, Government of India. IBEF’s primary objective is to promote and create international awareness of the Made in India label in markets overseas and to facilitate dissemination of knowledge of Indian products and services. Towards this objective, IBEF works closely with stakeholders across government and industry.

HIV/AIDS and Biotechnology

When the HIV/AIDS epidemic became widely known in the early 1980s I lived in San Francisco. I lost many dear friends during those years. That made working on “HIV/AIDS and Biotechnology” very personal to me.

Thirty years later and despite an all-too-common public perception that this terrible disease has been solved, the research to find a vaccine is actually more important than ever. The sad truth is that the cost in both human lives and economic treasure remains astronomical.

Producing this short film for BIO about the role of biotechnology in the on-going fight to save lives and dollars brought me face-to-face with three awesome individuals: a researcher, a patient, and an advocate. The researcher is Dr. David Asmuth, one of the world’s leading HIV experts. The patient, Brian Brown, received VACC-4X, a biotech vaccine produced by Bionor Pharma, in a clinical trial and went a full 18 months without anti-retroviral treatments. The advocate is Stephen Bailous, executive vice president of the National Association of People with AIDS (NAPWA) — he’s also an HIV survivor.

Together, these three men send a compelling message to all of us: If we are to conquer HIV/AIDS the necessity to fund sustained biotechnology research has never been more vital and more hopeful.

I wish to thank our entire team, especially our director Emily Deckelman, for their commitment to producing this important video.

Drugs Need Friends Too

At the BIO CEO & Investor Conference, industry leaders will address how new technologies fit into the biotech business model.

Drugs approved with companion diagnostic tests represent the next wave of personalized medicine and have the potential to significantly improve patient outcomes.

Companion diagnostics are changing the face of drug discovery and development and clinicians and investors alike are coming together for partnerships focused on the development of therapies for specific patient populations.

As biomarkers are discovered, using a companion test to predict if a specific drug or therapy will work in individual patients, and determining the appropriate dosage, can improve health care safety and drug efficacy.

The Personalized Medicine Coalition, a nonprofit advocacy group, reports that there were 72 prominent examples of personalized medicine drugs, treatments, and diagnostics products available last year.

Last year, there were two significant drug approvals in companion diagnostics:

• Pfizer’s Xalkori: Approved to treat patients with late-stage lung cancer who have an abnormal ALK gene in August.  Comes with a companion diagnostic test to screen potential patients for the ALK mutation.
• Daiichi Sankyo (Acquired when the company bought U.S. based biotech Plexxikon last year ) and Roche’s Zelboraf:  Approved in August for BRAF V600E-mutation positive melanoma.  Also comes with a companion diagnostic test to screen patients for the mutation.

Regulatory challenges associated with an unclear oversight process have slowed timelines and thwarted progress. Obviously, the FDA must address the complexity of personalized medicine as it is not a one-size-fits-all approach to drug discovery and development.

Delivering new and improved treatments and cures to patients should be the primary focus of companion diagnostic R&D, without question. Partnerships hold the key to advancing drug development and delivering on the potential of companion diagnostics.

At next month’s BIO CEO & Investor Conference, industry leaders will address how these new technologies fit into the biotech business model and how companies can best leverage companion diagnostics to enrich products and expand therapeutic opportunities.

The session, Drugs Need Friends Too – Companion Diagnostics and Drug Development, will be moderated byPatrick J. Mahaffy, President and CEO of Clovis Oncology.  Boulder, CO-based Clovis Oncology is a biopharmaceutical company focused on companion diagnostics that direct their product candidates to the patients most likely to potentially benefit.  The company has three product candidates in the pipeline.

Japan Action on GE Papaya

The U.S. Department of Agriculture has announced that the Government of Japan approved Hawaii’s Rainbow papaya for commercial shipment to Japan. The Rainbow papaya is genetically engineered to be resistant to the papaya ringspot virus. This announcement marks the beginning of a new chapter for Hawaiian papaya growers.

“The market opening in Japan is great news for Hawaii’s papaya producers and even better news for American agricultural exports,” said Michael Scuse, Acting Under Secretary for Farm and Foreign Agricultural Services.

In the 1990s, an outbreak of the papaya ringspot virus decimated Hawaii’s papaya crop.  Scientists from Cornell University, the University of Hawaii, The Upjohn Company and USDA’s Agricultural Research Service used biotechnology to develop the Rainbow papaya, which is resistant to the virus.  After receiving full clearance from the U.S. government, the Rainbow papaya was commercialized in 1998.  Now, the majority of Hawaii’s papaya crop is resistant to ringspot virus through genetic engineering.

“The story of the genetically engineered Rainbow papaya is a prime example of how biotechnology can help farmers and consumers by literally saving the Hawaiian papaya industry from extinction,” says BIO’s President and CEO Jim Greenwood.  “This announcement ensures that Hawaii’s papaya producers will continue to help grow our nation’s agricultural sector by expanding exports, creating jobs, and strengthening our nation’s eceonomy.”

Japan was once the major market for Hawaiian papayas, with annual sales reaching $15 million in 1996. These sales dropped to $1 million by 2010 while U.S. exporters awaited Japan’s approval of Rainbow papaya.  With Japan’s approval for import of Rainbow papaya, U.S. papaya producers are set to regain access to this important market, supporting jobs through increased exports.

Brazilian Innovation: A Patent Success

The story of Acheflan highlights the role of patents in homegrown innovation in developing countries.  Professor Michael Ryan of Georgetown reviewed several case studies (including Acheflan) in Brazil that highlight the differences in biomedical innovation both pre- and post-intellectual property reforms.

In the early 1980’s, Ache Laboratorios Farmaceuticos (a Brazilian generics manufacturer) became aware of a plant that grew near coastal cities that local fishermen would mash into an oil rub to serve as an anti-inflammatory and anti-scarring medicine.  Ache wanted to develop the plant into a product they could bring to the Brazilian and worldwide markets.  Ache realized that to develop the product they would need to isolate the active ingredient and then take it through toxicology studies, animal testing, and human clinical trials to demonstrate the safety and efficacy of the product.  However, Brazilian patent law at the time prohibited patenting of pharmaceutical patents.  While Brazilian patent law allowed for process patents, Ache quickly realized that competitors could easily reverse engineer the product and make their own version.  Ache did not pursue the project.

When Brazil reformed its patent law in 1996 to allow patents on pharmaceutical products, Ache resumed work on the shelved project.  Now they could finally invest in the project with a promise of a return guaranteed by a patent.  Not having the capacity to conduct research, Ache established research partnerships with Brazilian professors.  From 1998-2004, some 100 university agronomists, biochemists, pharmacologists, and medical doctors were involved in taking the product through isolation and toxicology studies.

The company launched the product, Acheflan, in 2005 and the years of hard work paid off.  Acheflan is the first medicine innovated and introduced in the marketplace by Brazilians and within a year the product received a 30 percent share of the anti-inflammatory market.  By the end of 2007, its market share exceeded 40 percent (beating out competitors among established global pharmaceutical companies).  The company is in the process of launching the product worldwide.

While Brazilian patent laws still require much improvement to lay the foundation for its future as a developed country, this case study is one of many suggesting that even incremental positive changes to the IP environment can catalyze commercialization of Brazilian innovation

Let’s Get PDUFA V Approved, Fast

At this time of the year, it can be helpful to reflect on the past to guide us in the future. I was recently reading an editorial about the National Health Council (NHC) printed in January 1957 – 37 years after the organization’s creation. It spoke about the need for action with “an unprecedented degree of cooperation among health agencies and the people they serve.” Well, that time is now and the issue is reauthorization of the Prescription Drug User Fee Act (PDFUA).

I want to thank the FDA and the pharmaceutical industry for again hearing the concerns of patients and for addressing our issues in a meaningful way. Today, we have a forward-thinking reauthorization agreement – PDUFA V.About a year and a half ago, at a Food and Drug Administration (FDA) meeting with patient advocacy organizations, we talked about how it was patients, two decades earlier, who chained themselves to the gates outside the FDA and the National Institutes of Health demanding access to treatments that were still in clinical trials and under review. Patients were told that they couldn’t have access to those treatments because researchers didn’t know if the compounds were safe or effective. But the patient community pushed back; many were going to be dead in a year. Their actions resulted in the creation of the early access program and a new environment that led to the first PDUFA.

Since the authorization of the first PDUFA, we have seen tremendous progress in the development of medicines for people with chronic diseases and disabilities. The time it took for the FDA to approve new drugs got shorter. However, there is now an emerging frustration among many people with chronic conditions who feel they still are not getting access to treatments as quickly as they would like. For many, such as patients with degenerative diseases like Alzheimer’s, clinical development moves slower than the progression of the disease.

For the past year and a half, the patient advocacy community has worked with the FDA and industry to get its three key priorities included in the proposed reauthorization agreement.

First, we need a qualitative, objective, framework for assessing the benefit-risk of new drugs. Benefit-risk is an important part of the FDA review process, but there is no consistently used framework or agreement among all stakeholders. How do we account for known and unknown risk or variation among subpopulations? Are benefits and risks appropriately placed in the therapeutic context?

No country in the world has articulated such a process, and there is increasing anxiety among patients who are denied access to new drugs based on a benefit/risk assessment they simply don’t understand.

The FDA and industry have agreed to include in PDUFA V the creation of a qualitative framework that would assess the level of certainty and variability of the benefits and risks, and that would provide for flexible scoring in the context of the therapeutic indication.

This benefit-risk framework will work for both patients and consumers. Both are stakeholders in this issue, but depending on where you fall on the health care needs spectrum, your perspective of benefit-risk can shift dramatically. For healthy consumers, the tolerance for risk, variability, and uncertainty for a new medicine to treat a condition such as hay fever is virtually zero. But if you are diagnosed with ALS (Lou Gehrig’s disease) and you have only two years to live, your tolerance for a risky new drug is dramatically different.

Second, we need greater use of patient-reported outcomes and biomarkers. Patients want more of a say in the drug review process, and they want the approval process to go faster than the progression of their disease. The patient community saw the reauthorization of PDUFA as an opportunity to develop metrics and tools to make the review process more effective, more efficient, and allow for the delivery of safe and effective medicines to people who need them.

Under PDUFA V, resources for patient-reported outcomes (PROs) will be increased, resulting in the patient perspective being incorporated earlier in the review process, dramatically reshaping drug research. It was psoriasis patients who taught us that it is not necessarily the size of their lesions that matters, but where the lesions are located on their body, such as on their faces or joints. This realization greatly altered the focus of psoriasis drug research.

PDUFA V would also increase resources to help the FDA speed up the approval of biomarkers in clinical trials. The use of these surrogate endpoints or clinical markers is greatly needed for people with chronic conditions. Polycystic kidney disease (PKD) is a genetic disorder of the kidneys. With PKD, fluid-filled cysts develop in the kidneys, which then can increase in both size and weight, sometimes weighing many pounds each. Currently, the recognized end point for PKD is kidney failure, which can occur as late as 40 years after diagnosis. No company would undertake a 40-year clinical trial, so qualifying a biomarker for a PKD drug is imperative. It is conceivable that if a new compound prevents a PKD patient’s kidney from getting larger, then something positive is happening.

Third, for the millions of people with rare diseases, we need new resources and greater flexibility in the regulatory review process. As proposed by FDA and industry, PDUFA V will utilize new regulatory science to speed the development of and access to new medicines for people who desperately need them.

The opportunity to engage in the PDUFA reauthorization process has been tremendously beneficial for the patient advocacy community, and I hope for the FDA and industry.

I say to those reading this column and to members of Congress, let’s get PDUFA V approved. Let’s get it done fast.

15 Keys to Success from Fierce Biotech’s Top 15 Women in Biotech

This week, Fierce Biotech recognized 15 women who are leading the way in biotech. Each of them represents the drive and determination that it takes to succeed in the industry, but what do they recommend beyond these inherent qualities? We pulled together the list below based on their profiles so that others can learn from their experience (yes, that made the list). In no particular order:

1. Take risks

2. Have an entrepreneurial spirit

3. Encourage teamwork

4. Be creative

5. Mentor others

6. Balance personal and professional life

7. Shatter stereotypes

8. Cultivate your business acumen

9. Be technically savvy

10. Seek knowledge and information

11. Stay curious

12. Exemplify leadership

13. Cultivate your career

14. Plant seeds

15. Buck the trends