“We have to change the way we develop drugs,” Woodcock stated. The current method is very expensive and doesn’t provide important information on why people respond differently to drug interventions. Sponsors are moving to personalize dose by patient genotype and examine why different drugs are effective for different genotypes of a virus, such as Hepatitis C. But it is “not feasible,” she said, to do a separate trial to determine who should receive a drug, at what dose and with what likely adverse effects for every patient group.

Instead, Woodcock proposed to “turn the clinical trial paradigm on its head” and for research organizations or coalitions to set up ongoing, standing trials to examine patient responses to multiple issues raised by test therapies and biomarkers for certain diseases or conditions. Woodcock cited the I-Spy study launched by the National Institutes of Health to screen new compounds for breast cancer, and similar efforts by disease groups and professional societies to establish networks able to make rapid evaluations of genetic subsets and biomarkers.

Such approaches will support rapid approval of highly effective “breakthrough” therapies, as authorized by the FDA Safety and Innovation Act (FDASIA) to streamline approval of serious and life-threatening treatments. FDA continues to receive and grant requests for breakthrough designations and expects to see more highly targeted and effective drugs come to market, Woodcock noted. These approaches, moreover, are likely to modify FDA’s usual requirement for two randomized, controlled clinical trials to support the approval of new treatments.

Reliable diagnostics are the foundation of these approaches and are desperately needed to make sense of the large volume of information emerging from genomic testing,  Woodcock added. FDA has published guidance on how to develop and gain approval of companion diagnostics to ensure the validity of tests used to make critical treatment decisions. Still in development, though, is long-awaited guidance on drug-diagnostic co-development, a rapidly evolving field that remains hard to define for industry and  FDA alike.

Focus on value

Woodcock also raised concerns that the high cost of many new targeted therapies and related diagnostics may contribute to rising health care expenditures and draw opposition from payers. Unusual for an FDA official, she noted increased resistance to reimbursement for “high tech” therapies and diagnostics that offer only limited gains in efficacy. The new field of targeted therapy needs to focus on “adding value,” she commented; manufacturers should strive to develop combinations of treatments that actually control or cure disease, as opposed to short-term improvements in efficacy.

The emergence of more breakthrough drugs has produced a number of “game changers” that are more effective in treating serious diseases, she pointed out, but noted the need to do more. The future of personalized medicine is to deliver “new treatments at reasonable cost that have significant impact in treating or preventing disease,” she stated. For cancer, the goal is to find out how to alter the course of disease, and not stop at incremental improvements, she observed, adding that a cure for Hepatitis C won’t raise any complaints about cost.

Personalized therapies have to be more effective, less toxic – and more cost-effective – than today’s interventions, Woodcock concluded, for this field to really become a major factor in healthcare.

In addition to taking a close look at pharma’s internal processes, manufacturing facilities and corporate policies this year, the US Justice Department (DOJ) – in the name of “protecting consumers where they are most vulnerable,” according to Frimpong – is also placing a “focus on people,” since processes and policies (at least publicly-circulated processes and policies) don’t typically break the law; people do. “We know there are enormous cost pressures, but you can’t sacrifice safety under these pressures,” said Frimpong.

To that end, Frimpong recommended that attendees ask themselves five questions about the group of people they work with, to help identify potential problems before they result in an investigation or worse. In summary, Frimpong’s questions are:

1. Do we have the right people? Are they experts in their specific task areas? “People are not fungible,” said Frimpong. Given the complexity involved in certain manufacturing practices, for example, it’s crucial that the right person is working in the right role.

2. Do our people have the right incentives enabling them to see problems, report problems, and fix problems? Frimpong said strong internal communications are key to facilitating diligent compliance programs.

3. Are our people satisfied and engaged with the company and their jobs? Frimpong said the departure of key people can sometimes lead to lapses in compliance, citing SB Pharmaco, a GSK subsidiary, and the manufacturing deficiencies at the company’s (now-closed) plant in Cidra, Puerto Rico.

4. Are people and policies working in harmony? Companies should set realistic goals, and refrain from crafting compliance programs and procedures that can’t be met. Unrealistic standards are doomed to fail, said Frimpong.

5. Do we, as chief compliance officers, know what our people are actually doing? Given the size and scope of many large, multinational pharmas, it’s important that leadership make a concerted effort to communicate with and assess the performance of specific individuals whenever possible.

Frimpong underscored the problem – and gave examples – of companies who’ve put patients in harm’s way in service of the bottom line, saying, “when companies put profits over patients, everyone loses.”

Extra: For a report from Jill Wechsler on comments about the recent Caronia decision made by Tom Abrams, director of the Office of Prescription Drug Promotion, at the CBI Congress, click here.

What is strictly legal in the context of sales and marketing practices, working with government officials in global markets, and running and reporting on clinical programs, may not always be the last word on how a company should act in these areas. In other words, companies often need to go beyond adherence to the letter of the law, and instead consider the broader implications of specific conduct, before a decision is made, or a policy developed.

As the role and function of compliance teams grows within pharma companies, different approaches to the law, and what it should look like in practice, can differ, which sometimes pits internal lawyers against one another. “Compliance wants to truly ferret out any wrongdoing that’s going on – that’s not to say legal doesn’t – but compliance wants to expose it,” says Tim Ayers, VP, chief compliance officer, at Dendreon, a biotech focused on cancer therapies. “Legal wants to wrap everything under privilege, not to hide things, but to deal with things from a very legal, mitigating risk perspective, whereas compliance wants to come in and say, ‘Listen, this is what we’ve done, let’s fix it.’”

Companies operating under a corporate integrity agreement (CIA) by necessity have elevated compliance officers to the c-suite, but in other companies that haven’t had to deal with Office of Inspector General (OIG) or Department of Justice (DOJ) investigations – and there aren’t many of these left – compliance teams are sometimes subordinate to legal departments, even though the members of both groups hold law degrees. It’s in the application of the law, where the hairs get split. “Legal likes to ask very narrowly tailored questions, specific to the legal issue,” says Ayers. “And it might be perfectly sound legal advice, but it might not make sense from a compliance perspective, at forty-thousand feet.”

So what is the right organizational structure for compliance teams, and the chief compliance officer? “I used to be of the opinion that it all depends on the company’s evolution and lifecycle,” says Ayers, who was formerly associate general counsel and executive director of compliance at Seattle Genetics, and who has also served as general counsel at Allos Therapeutics, Salix Pharmaceuticals, and as a regional attorney at Pfizer. “But now I’m becoming increasingly of the opinion that there is an inherent tension between legal and compliance, and if [compliance] is subordinate to legal, it’s inevitable that an issue will rise up which literally speaks to this conflict of interest.”

For more on the issue of compliance, and how companies are bringing compliance personnel into the upper echelons of senior management, look for PharmExec’s feature on the subject in the January issue. Additionally, Ayers and many other pharma compliance executives and government regulators will be on hand for CBI’s 10^th Annual Pharmaceutical Compliance Congress, to be held at the Ritz-Carlton in Washington DC on January 29–30.

Since the last Ipsos survey in 2008, pharma’s reputation has improved significantly in the US, more so than in any of the other 24 countries surveyed. Other countries, however, namely China, Spain, Brazil, Russia, Germany and India, have a lower opinion of the pharma industry today than four years ago.

In 2012, citizens in the survey countries reported, on average, a more favorable impression of pharma than an unfavorable one – 40% and 18%, respectively. On a cross industry basis, Pharma’s “net favorable” score was 21, putting it in fifth place behind personal computers (37) at the top of the list, restaurants (31), retail (28) and telecommunications (24). As one might expect, oil and gas (2), credit card companies (-3) and the tobacco (-28) industry carried the worst reputations globally this year.

Asked whether the pharmaceutical industry is appropriately regulated, survey participants in France, Russia and Germany – countries on the negative side of the global consensus – felt that industry needs more regulation. France sentiment was especially strong; 59% of the respondents said pharma is too lightly regulated.

Other findings from the report include:

• Emerging Markets have a more satisfactory opinion of the industry than the mature market countries.
• The US hold the most balanced view of the need for regulation- 36% said more is needed, 46% remain content with current regulations, and 18% said there should be less.
• Attitudes toward industry and top companies aren’t always the same- US and China rated individual companies highly, while rating the overall industry less favorably.
• Top pharma companies are more likely to be perceived positively based on their product quality and industry leadership rather than good corporate citizenship.

Significantly, China’s perception of the industry has soured the most in the past four years, even as pharma made strides in the US. Taken against China’s higher regard for top pharma companies, what accounts for the discrepancy? “Many recent drug scandals in China have been the result of safety issues and corruption associated mostly with local brands,” says Nicolas Boyon, co-author of the report. Global companies, by juxtaposition, stand out because their western origin provides Chinese consumers with reassurance as to the safety and efficacy of their products.”  One such scandal involved the use of chromium, a known carcinogenic, found present in the local manufacture of gel capsules.

In addition, Boyon notes that DTC advertising, while illegal in China, “could benefit from companies leveraging the assurance of quality their trademarks represent as branded medicines.”

Recommended priorities for companies in pharma expressed by citizens around the world were closely aligned, and nothing particularly new to industry ears: innovation, safety, and improved access via financial assistance for patients. What will be interesting to see is how China’s ascent to status as the world’s second largest market for pharma will shape industry’s global reputation. The role and visibility of branded generic firms from China –and other key emerging markets –will begin to attract more attention, raising important questions about the scope of cross-border regulation and inspection.

If you can’t measure it, the saying goes, it doesn’t count.  But what if you’re measuring the wrong things? Peter J.Pitts reports.

The Access to Medicines Index is an attempt to measure and compare the corporate social responsibility of both innovator (20) and generics (7) companies based on a number of different (and often quixotic) indicators. It was developed by the Netherland’s-based non-profit Access to Medicines Foundation (ATMF) with funding from the Dutch Ministry of Foreign Affairs, the UK Department for International Development and The Bill & Melinda Gates Foundation. It was launched in 2008, and comes out every two years.

The Index analyzes the following seven technical areas across four pillars:

1) Commitment (30% weight)

2) Transparency (30% weight)

3) Performance (30% weight)

4) Innovation (10% weight)

Technical Areas:

1) General Access to Medicine Strategy and Governance

2) Public Policy and Advocacy

3) R&D for Index Diseases

4) Patients & Licensing

5) Equitable Pricing & Registration

6) Technology Transfer (Capability Advancement)

7) Drug Donations and Philanthropic Activities

The Index concentrates on the global list of Low and Medium Development Countries based on the UN Human Development Index and World Bank Country Income level categories. The Index has historically covered 33 diseases including: WHO Neglected Tropical Diseases; the top 10 infectious diseases based on DALYs (Disability-Adjusted Life Years) from the WHO Global Burden of Diseases for the Low Development and Medium Development Countries; and the top 10 chronic diseases based on DALYs from the WHO Global Burden of Diseases for the Low Development and Medium Development Countries. The next index will likely broaden the disease scope to additional categories including NCDs, women’s health and some cancers. They also plan to place increased weighting on companies’ actual performance (vs. their commitments).

According the Access to Medicine Foundation, the index “aims to help poor people in developing countries gain access to medicine by encouraging the pharmaceutical industry to improve its commitments and practices related to this issue.” Since it’s a comparison, the theory is that competition among companies will drive desirous “socially responsible” behaviors. That’s a noble goal, but the devil is in the details.

As Goran Tomson, professor of international health systems research at Karolinska Institute points out, the Index’s methodology cannot be reproduced, hence it cannot be considered statistically valid.

There are also troubling issues relative to the index’s metrics for success. According to the Index’s methodological designer, Afshin Mehrpouya (an assistant professor of accounting and management control at the Paris-based HEC international business school) recently said, the only current measurements are “web hits and media coverage.” That’s not very exciting, plausible, or helpful from a health policy analysis perspective.

Another metric is the opinion of patient groups. When asked why certain patient groups were chosen (they are not named in the Index), the answer was that groups were chosen based on their “credibility.” That’s code for groups who do not accept funding from the pharmaceutical industry or may share an  anti-private sector bias.  At minimum, it’s dubious selection criteria.

Karolinska’s Tomson also pointed out that the index’s “review committee” consisted almost entirely of “familiar faces,” thus creating an issue of normative bias.

In other words, when did you stop beating your wife?

To offer better balance and some reference, shouldn’t the Index create a parallel metric that measures the policies and political environments of low and middle-income countries to determine whether they facilitate or hinder their citizens’ access to healthcare? How about creating an index that addresses the lack of transparency in the LDC public sector and (the 800 pound gorilla in the room) corruption. These are all polite ways of saying that the design criterion stacks the deck. But, hey, doesn’t the end justify the means?

Karolinska’s Tomson put the discussion about the index—as well as the entire ICIUM enterprise—into perspective when he said the index lacked for “higher ambitions.”

“Higher ambitions” requires that the Index do more than read its own press releases and talk with its friends in NGO-Land. “Higher ambitions” requires honesty beyond one’s own cognitive mapping.

According to Index CEO and Founder Wim Leerveld, “Today all companies have teams to deliver us the requested data as they see the relevance for them.”

Maybe. Maybe not. Interviews with participating companies showed much displeasure with both the Index questionnaire, the volume and type of information being requested by the Index and the “normative bias” of the ATMi staff. In some cases, participating companies are spending hundreds of hours to collect, verify and prepare final submissions. Is it worth it?

Here are some tough questions the Indexers must ask themselves:

Q: Is the Index moving the needle? Is there evidence that this is worth the effort that companies put into it and does it justify donor funding? Have they had an impact on financial investment patterns? Web hits and press clippings don’t cut it.

Q: What work has ATMF done to validate that their metrics are in fact the right drivers to improving access? Are they selecting an agenda being pushed by activist constituents without assessing a more full-bodied picture of what is happening in the real world?

To take one glaring example, the Index operates from the assumption that the innovator pharmaceutical industry can improve access to essential medicines. But, when you examine the WHO’s model Essential Drug List, very few of the 400 or so drugs deemed essential are new, or patented or were ever patented in the world’s poorest countries.  In category after category, from aspirin to Zithromax, in almost every case and in almost every country, these medicines have always been (or have been for many years) in the public domain.  That is, the medicine is fully open to legal and legitimate generic manufacture.

There are important implications for the world’s poorest patients. If these patients had reliable and affordable access to these several hundred essential medicines, all available theoretically as multi-source, that is from generics companies, then global mortality and morbidity might be cut as much as 10-20% — a huge gain for populations around the world. Strangely, the Index gives a pass to the world’s largest producers of generics drugs in India and China. Those companies are not being asked to spend hundreds of hours assembling data on their contributions to medicines access.  Given the potential hugely positive impact on access to medicines, any reasonable person might ask why?

Q: Is it time to reassess the Index’s bias for certain mechanisms and tools? Its fixation on the medicines patent pool, for example unfairly demoting companies that aren’t in negotiations. This doesn’t seem fair. The pool is only one part of a broader landscape of what’s happening around access to HIV and other treatments and it’s unfair to use one tool as a measure of companies’ commitment when there are other things happening that are very relevant and important.

There are ideological and activist assumptions within the questionnaire’s section on tech transfer question. Is the Index looking at tech transfer as a measure of access to medicines or are they promoting industrial policy? Why isn’t in-country capacity building measured? What about efforts to fight counterfeiting? If ATMi is an ideological exercise, then innovator pharmaceutical industry should question the usefulness of the proposition.

Q: How does the Index ensure each company answers the questions in the same way to allow for an apples-to-apples comparison? Is the Index able to objectively compare companies? According to one corporate participant:

“Some of the questions were just too difficult to understand and required too much interpretation. In the Pricing & Distribution section, (Q1.9) we’re asked to provide product-specific registration status. When I asked for clarification, our Index contact told us to indicate if the product is patented in the countries in which it’s registered. This is not inherent in the question. Also, we think it would be useful for the Index to know where we have filed for registration but where the application may be stuck in a bottleneck since the speed of registration is highly dependent on the speed and efficiency of local countries’ regulatory processes. But our contact indicated this information was not necessary.”

The concern among participating industry is that the Index is getting increasingly arbitrary and opinionated. This is exacerbated by the fact that (per ATMi staff bios on its website) – many of the Index staff has worked for NGOs such as Médecins sans Frontières and other like-minded anti-pharmaceutical industry groups.

Industry should want the Index. They value being recognized for their good work – and the competitive rankings are appreciated and sometimes flaunted. But the agenda isn’t appreciated. Pushing TRIPS+ and penalizing intellectual property rights trivializes the nature of the program. Perhaps it’s inaccurate to say that industry wants the Index.  Maybe a better statement is that industry wants AN  index.

Recognizing the inherent flaws and bias of the ATMi – but also the importance of measuring industry’s commitment to social responsibility and access, Business for Social Responsibility’s (http://www.bsr.org/) Healthcare Working Group has launched an effort to provide an industry-wide lens on this important issue.  The Working Group’s efforts center on the group’s acknowledgement that solving this challenge is a human need, and a business priority that requires a close collaboration with other actors from industry, public and NGO sectors.

It will be interesting to see how these two programs compare – and which one survives.

Peter J. Pitts is President of the Center for Medicine in the Public Interest and a former FDA Associate Commissioner

Public policy activities should be a distinct, mission critical function for Big Pharma — our business  depends increasingly on government as a key customer. However, as companies confront the necessity to plump up the bottom line as patents expire, staffing of non-line functions has taken a big hit, with policy advocacy work emerging as a principal target. But there is a larger long-term trend at work that promises to do serious damage to the industry’s chances in preserving its basic “license to operate.” This is the decision taken by many of the leading companies today to merge  the corporate social responsibility (CSR) function with public policy/government relations advocacy, effectively  subordinating the latter tasks as part of a “reputation management” mandate focused on philanthropy and good works. 

The trend is especially troublesome in the industry’s relationships with multilateral organizations, where responsibility has been  transferred from policy/government relations experts to company philanthropic foundation or CSR managers. No longer is the relationship managed by  policy experts capable of promoting and protecting policy environments conducive to medicines innovation. Rather, those CSR managers are focused simply on extracting a one-off reputation gain through high-profile (and costly) initiatives that often detract from long held policy principles endorsed by the industry as a whole.

As a result, a coordinated, industry-wide approach to  economic and social policy engagement across the UN system, in individual member states and among key NGO influentials is being weakened, at high risk to the intellectual property protection, market-based pricing, balanced and non-discriminatory  regulatory standards and other business friendly objectives that help build opportunities for drug makers.  The risk is particularly high in emerging country markets, which tend to follow closely the work of multilateral organizations like the WHO and WIPO and where much of the industry’s future growth prospects is concentrated.

Quite simply, Philanthropy-minded CSR managers feed the insatiable appetites of government officials, UN bureaucrats and NGOs with “voluntary initiatives” that put multinational corporations in check, using mandatory regulation, through a range of human rights and sustainability instruments.

According to James Roberts and Andrew Markely of the Heritage Foundation[1]: “This new CSR push is an attempt to redefine the very purpose of business by asserting so-called triple-bottom-line obligation of companies to deliver (1) economic, (2) social and (3) environmental “returns” or to justify a theoretical “license to operate” granted to them by society”.

Although future business-school analyses of the decline of innovative biopharmaceutical companies may conclude that R&D failures were the cause, I agree with Roberts and Markely that ill-advised initiatives supported and delivered by the industry’s own foundation and corporate responsibility managers helped to accelerate  the spiral.

Industry actually began this self-inflicted wounding decades ago.  In 1984, Rael Jean and Erich Issac, in “The Coercive Utopians,” warned companies against making contributions to pressure groups in the hope of buying peace – or to encourage them to focus on competitors.  This they called “funding the rope” for ultimate corporate hangings.

The goal of CSR is to engender favorable perceptions among stakeholders. This is often regarded as a “soft” public relations goal. To the extent  that CSR initiatives develop good relationships, that is a worthy objective.

Those relationships cannot lay fallow, however, and a handshake is empty unless all parties benefit. Industry – and the patients it serves – does not benefit unless its public policy work is successful. Intelligence-gathering, position development, advocacy, obtaining buy-in on positions and lobbying are  critical to securing the enabling environments for realizing “hard” corporate performance indicators.  Consider this:  In 1900, aspirin was the only widely-available medicine. Before 1940, four additional scientifically-developed medicines were added, by 1960 there were a total of 392 new molecular entities (NME) and by the end of 2011, FDA had approved 1,135 NMEs for marketing in the US. Much of the success here was due to the seamless relationship between industry policy advocates,  scientists and the clinician community in extending high levels of public funding for basic research combined with strong regulatory incentives for the commercial development of the fruits of that research.  The question that needs to be asked is whether this productive  trend is sustainable if power and resources are directed to a company’s “good Samaritans” while the policy advocates — that first line of defense for the business model — are starved?

Reference
[1] “Backgrounder #2685”,  dated May 4, 2012

About the author
Susan Crowley is a former Merck executive who now advises companies on global issues. scrowley@multilateralconsulting.com

Over the counter drug advertising is less regulated than prescription drug marketing, and selling directly to consumers comes with the benefit of bypassing an expert middleman: your doctor. But companies should still promote with caution.

Comedian Larry the Cable Guy is a spokesperson for Prilosec OTC

The margins may be lower, but skirting access and reimbursement issues in an increasingly skeptical payer environment makes over-the-counter (OTC) products appealing to certain big players. Especially since FDA has signaled an openness to shifting more products into the shopping isle.

Pharma companies with the capability to sell quantity in mature markets are likely to benefit from popular prescription drugs turned OTC. In July, Pfizer said it would pay AstraZeneca $250 million plus royalties for rights to sell OTC Nexium, the blockbuster gastroesophageal reflux disease (GERD) drug, when it goes off patent in 2014.

Selling prescription drugs turned OTC is not without challenges, however. Strong marketing and promotional execution is a critical element of success, particularly since OTC drugs are often competing against generic products that cost the same or less with a prescription. Prilosec, AstraZeneca’s older GERD blockbuster, was ushered OTC by consumer packaged goods behemoth Proctor & Gamble nearly a decade ago; the drug was the first OTC proton pump inhibitor on the market.

Pfizer is known for the prowess of its marketing machine, but a degree of caution is in order, if P&G and the advertising approach of CPG companies in general is going to serve as the promotional model for prescription drugs reborn as OTCs.

A new thirty-second spot for Prilosec OTC, created by Publicis New York (which merged last month with its Publicis sister shop Kaplan Thaler Group to form Publicis Kaplan Thaler) features comedian Larry the Cable Guy, an “actual Prilosec OTC user.” The spot debuted in late June and is running on television and online, according to Kiera Casanova, senior account executive at Publicis Kaplan Thaler. Moviegoers in Brooklyn last weekend saw the ad prior to a screening of The Dark Knight Rises, and they could be forgiven for thinking Prilosec a panacea that obviates any need for dietary restraint.

“Know what I love about this country?” asks Larry in the opening frame, standing in front of someone grilling food on a barbeque. “Trick question! I love everything about this country, including Prilosec OTC!” Just one pill each morning treats your frequent heartburn, Larry says, “so you can enjoy all this great land of ours, as often.” That means demolition derbies and drive-through weddings, but more to the point, corn dogs. The ad ends with Larry and his two compatriots standing in front of a “Colossal Corn Dog” stand holding corn dogs. Instead of treating heartburn as it occurs, “don’t get heartburn in the first place,” by taking…you guessed it. The ad’s tagline – “one pill each morning, twenty-four hours, zero heartburn” – drives the message home: fatty foods have been associated with triggering heartburn, but there’s a pill for that.

Perhaps this is a message that some Americans want to hear. But it’s not a message that most healthcare providers would be comfortable with, and it’s not going to further the pharmaceutical industry’s desire to be seen as more concerned with health outcomes than sales figures. As new drugs climb over the counter from other therapeutic areas – the OTC heartburn category alone earned $1.4 billion in the US in 2010, according to the Consumer Healthcare Products Association – pharma will need to balance its consumer marketing with reputational considerations, even if regulators won’t be watching as closely.

The latest offense, which generated an Untitled Letter from the Office of Prescription Drug Promotion (OPDP), was brought to light through OPDP’s Bad Ad program, which encourages physicians to report on shady sales rep behavior, like off-label promotion or more commonly, risk minimization or broadening the indication of a drug during a sales detail.

Last October, according to the Untitled Letter (dated August 1), Forest reps made statements to physicians that both minimized risks and broadened the indication for Daliresp, a COPD drug. Daliresp launched in August 2011, and earned $31.2 million in Forest’s 2012 fiscal year; the company is forecasting sales of $85 million for 2013. “OPDP is concerned that the violative conduct…occurred despite Forest’s September 15, 2010, Corporate Integrity Agreement (CIA)…that requires Forest to ensure that its policies and procedures address ‘appropriate ways to conduct promotional…functions in compliance with all FDA requirements, and to provide specific training to its employees engaged in promotional functions,” OPDP admonished.

“False and misleading statements from Forest sales representatives are particularly troubling considering OPDP expressed concerns regarding similar violative promotional activities as recently as April 2011,” the letter stated, noting a separate Untitled Letter addressing the promotion of Savella, a fibromyalgia drug, for unapproved uses, as well as a minimization of “serious risks” associated with drug.

Untitled Letters are small potatoes compared with the giant pumpkin of a letter sent from the Office of Inspector General (OIG) to Howard Solomon, chairman, CEO and president of Forest Labs, in April 2011. That letter, the result of a litany of charges including the off-label promotion of two anti-depressants, informed Solomon that he could face exclusion from doing business with government healthcare plans, which would effectively end his career. Solomon has vowed to fight exclusion in court; his attorneys, and their attorney friends, are currently mum on any new developments.

The OIG couldn’t be immediately reached for a status update on Solomon’s exclusion, or whether or not Forest will face additional penalties under its CIA for non-compliance. Check back for updates.

At CBI’s 7th Annual Rare Disease and Orphan Drug Leadership Congress, speakers and attendees identified areas where pharma can improve the way it approaches rare disease and treatment.

It’s no secret that rare diseases, and the orphan drugs pharmaceutical and biotech companies make to treat them, represent a unique business opportunity. Often, there is no treatment available at all for a given disease, which is the most fundamental definition of an unmet need. Today, treatments are available for around 3% of the approximately 6,800 rare diseases identified globally.

For patients with a rare disease, the emergence and value of a new treatment is self-evident; lives are dramatically altered for the better. From a business perspective, orphan drugs have much to recommend them. Many receive an expedited review from FDA and other regulatory bodies; most products are priced at a premium, given the lack of competition; patient populations and their families are often eager and willing participants in clinical trials, and smaller populations are more easily managed; and many orphan drugs, once approved for a rare disease, move on to label expansions for broader indications.

It’s also cost-effective to invest significantly in a rare disease population. Because the population is small, and each patient – or “walking asset,” as one financial analyst put it – represents significant revenue, pharma companies can afford to provide the kinds of services “beyond the pill” that everyone seems so keen to deliver. One of the current problems, according to speakers at CBI’s 7^th Annual Rare Disease and Orphan Drug Leadership Congress in Philadelphia yesterday, is that these services aren’t being administered effectively, because they aren’t being designed from the end user – the patient and physician – point of view. “It’s not like pharma isn’t creating the right programs” – programs around patient assistance, adherence and education, specialty pharmacy distribution, reimbursement, nursing services, REMS compliance, etc. – “they’re checking the boxes but not harmonizing all of the components so that there is a seamless experience for patients and doctors,” said Kristin Keller, EVP at Compass Healthcare Marketers, an agency focused orphan drug patient marketing. “People will drop out of the funnel if everything isn’t integrated from a patient point of view.”

On REMS programs, Keller noted that industry is “being myopic about the opportunity it affords.” As a regulatory action, REMS programs are typically governed by a conservative approach, and not exactly celebrated. “Because REMS programs enable companies to have direct contact with all patients on therapy, there’s an opportunity to go beyond regulatory requirements to use that contact to deliver even greater education and support to the patient,” said Keller. Industry should look at REMS as a data asset instead of a regulatory headache.

With respect to missed opportunities in data management, one speaker wondered why “no one incorporates learnings and protocols from the clinic into adherence and education programs for patients.” This question becomes especially pertinent when a new drug is introduced for a rare disease currently being treated with an older drug that perhaps doesn’t work as well, or has unwanted side effects. Even so, patients are scared to switch to the new drug, and risk going back to a full-blown disease state. Pharma, according to the speaker, isn’t doing a good job explaining the benefits of the new drug, and tying that treatment to the here and now: what life might be like tonight, tomorrow, or a week from now. One reason for this has to do with med/legal and pharma’s inability to make comparisons that aren’t clearly backed up in the clinic or on the label. One speaker proposed a “patient-friendly compliance expert” with medical claims knowledge, but also a knack for translating that knowledge into a dialogue with patients that would leave them with a better understanding of a drug’s benefit, so they can make a more informed decision.

What do rare disease patients think about all of this? Unfortunately, no actual patients took the stage to give presentations, but there were a few in the audience. One sickle cell patient set off a flurry of discussion among the audience when he asked about how to deal with faith – i.e. God and His followers – in the sickle cell community. “Patients will often listen to their pastors over their doctors,” he said. One audience member said her company had dealt with the same problem among members of the Ashkenazi Jewish heritage, which is plagued by high incidences of genetic disease. Her answer? A KOL is a KOL. “We targeted messaging to temples and to rabbis,” she said.

Other topics discussed at the meeting included the importance of managed access programs, also known by many other names including expanded access programs, early access programs, etc., etc., all of which provide medicine to patients where it isn’t commercially available. Interestingly, companies are now beginning to collect real-world data as part of these programs, which can then be used – particularly in the case of a managed access program conducted while a drug is still in phase 3 – to augment a drug’s dossier at registration. “We did incorporate data from an access program in the safety profile submission for a biologics license application, to support the traditional clinical data,” one speaker said.

During a question and answer panel with several patient advocacy group speakers, everyone emphasized the importance of pharma engaging early with their organizations. “Don’t wait for an approval and then show up with an educational piece and say, ‘What do you think of this?’ said one of the speakers. Another underscored the importance of keeping advocacy groups informed about clinical trials and other developments, so that patients’ expectations can be more efficiently managed. Managing expectations is a crucial role of patient advocacy groups, as more and more patients become involved in the system. Three factors are contributing the rise of patient advocacy, one speaker said: the increase in transparency of company pipelines; the growing empowerment via education of patients and their families; and the connection to other patients provided by social media and emerging technologies.

For companies working in the orphan disease space, or looking to move into it, the days of focusing exclusively on developing a treatment, and then chucking it out into the market for patients to purchase are over. From the early development stages, to access, to patient monitoring and adherence, now it pays to take a walk in the patient’s shoes.