Tag Archives: disease

To Cure Rare Diseases, Unleash Orphan Drug Innovations

In September of 2012, the City of Pittsburgh hosted the 35th annual Great Race, a charity run that raises money for the Richard S. Caliguiri Amyloidosis Research Fund. Caliguiri, a former Pittsburgh mayor, died of this rare protein disorder, and a portion of the race proceeds are used to help find a cure.

It should have been an uplifting event. Yet the Pittsburgh Post-Gazette reported that “despite the rally of support … the research fund created in Caliguiri’s name has had little impact on the effort to find a cure.”

Those familiar with the challenges of treating rare conditions like amyloidosis won’t be surprised by this news. The sad truth is that the economic incentives for developing life-saving treatments for rare disorders are less than optimal.

But that doesn’t mean that we’re powerless to fight rare diseases. Policymakers can dramatically improve incentives for researchers and biopharmaceutical firms to create drugs that treat rare conditions — treatments known as “orphan drugs.” And they should. Rare diseases collectively represent a major public-health threat.

Rare diseases — conditions like Huntington disease and Burkitt lymphoma which afflict fewer than 200,000 people — cost Americans more than $474 billion a year.

Over 7 percent of Americans — or more than 25 million people — suffer from the roughly 7,000 illnesses that fall into this category.

So the overall market for treatments for rare diseases is large. Indeed, total global spending on orphan drugs runs between $50 billion and $85 billion — or between 5.7 percent and 9.7 percent of what the world spends on pharmaceuticals, according to “A Primer on the Orphan Drug Market: Addressing the Needs of Patients with Rare Diseases,” a new paper by economist Dr. Wayne Winegarden, senior fellow at the Pacific Research Institute.

But the potential number of beneficiaries for each individual orphan treatment is relatively small. A narrow market of potential buyers can make investing in orphan drugs perilous.

Part of the problem is that developing a treatment for any illness is a long and expensive process. It takes anywhere from 10 to 15 years to usher a treatment from the research phase, through the Food and Drug Administration (FDA) approval process, and into patients’ hands.

At every step of the process, drug firms must spend more money — and face the distinct possibility that their research will fail. According to the best estimates, a single successful drug costs well over $1 billion to develop.

And once a drug goes generic, the firm that created the medicine must deal with fierce competition from other manufacturers.

It’s hardly surprising, then, that pharmaceutical firms tend to bet on treatments that will be useful to the largest number of patients. This state of affairs often leaves those suffering from rare diseases with few treatment options.

Fortunately, policymakers have found ways to improve the incentives for pharmaceutical firms to invest in orphan drug research.

Consider the Orphan Drug Act, passed in 1983. It provided drug developers seven years of market exclusivity for their inventions, ensuring that they would have a reasonable amount of time in which to make back their hefty investment, free of competition from other pharmaceutical firms.

The law also lessened the economic burden of drug development by offering a 50-percent tax credit for the costs incurred during the clinical trial phase. On top of that, the Act waived the fees associated with applying for FDA approval.

The results? In the past decade, the number of new drugs — or New Molecular Entities (NME), as they are called in the trade — released in the United States for the treatment of rare diseases has increased dramatically. In fact, more new NMEs were launched in 2011 than in any of the last ten years.

This is encouraging evidence that those who suffer from rare illnesses shouldn’t give up hope.

It’s now up to our leaders to find new ways to lower the barriers to developing these valuable treatments. They should start by taking the Orphan Drug Act — already over a quarter-century old — and using it as a model.

Drug companies should be rewarded for developing orphan drugs. It’s the only way to guarantee that, no matter how uncommon an illness, there will be somebody, somewhere working to find a cure for it.

Affordability, Effectiveness, and Wellness, Part 4

This is Part 4 in a five-part series which presents a creative solution for today’s health care crisis. Additional articles in the series can be found here: Part 1, Part 2, Part 3, and Part 5.

Emergence Of Health And Wellness Programs As A Solution
For many years wellness has been viewed from somewhat of a skeptical perspective. However recent studies and improved implementation programs are showing considerable health cost savings are associated with health and wellness programs. Our firm’s most recent estimates show that an effectively implemented health and wellness program can reduce aggregate health care costs by as much as 20% – 35%. In addition, these costs savings continue for more than one year. Health and wellness provides a meaningful solution of the affordability crisis. Although there continue to be significant opportunities for efficiency improvements within the health care system, the emergence of the health and wellness solution is encouraging.

Table 7
Illustrative Impact of Health and Wellness Changes

Health & Wellness
Level
Current
Dist.*
% of
Ave $*
$
Dist.
Modest
Shift
$
Dist.
Major
Shift
$
Dist.
Well 50% 20% 10% 52% 10% 55% 11%
Low Risk 20% 50% 10% 22% 11% 23% 12%
Moderate Risk 25% 100% 25% 23% 23% 20% 20%
High Risk 4% 750% 30% 2.5% 19% 1.5% 11%
Complex 1% 2500% 25% 0.5% 13% 0.5% 12%
Total 100% 100% 100% 100% 76% 100% 66%

* Source: Dee W. Eddington, Ph.D., Director of the Health Management Research Center at the University of Michigan

Table 7 shows that with a modest shift in health and wellness level health care costs are reduced 24%, and with a more significant shift costs are reduced 34%. These shifts are very possible under an effectively implemented health and wellness program. These projected savings compare favorably with the potential for efficiency improvements. However, they bring a much more important quality to the table, these are politically correct and desirable initiatives. The efficiency savings, although real savings, have become less desirable for a variety of reasons.

The Change Management Connection
Health and wellness programs are really change management initiatives, focused on personal behavior change management, perhaps the hardest of all changes to make. Traditional change management focuses on moving an entity from its “current world” to a more desired “future world.” Current world in the health and wellness environment is the individual’s current health status. Future world is the hoped-for positive outcome of the health and wellness program (i.e., the individual’s ideal health status). The key ingredient to measure success is determining how to successfully transition the individual from today to the future, from their current world to their future world, from “interested” in true health and wellness to becoming genuinely committed.

It is helpful to look at this from two distinct perspectives: organizational change management and individual change management.

Organizational Change Management
Classical change management principles offer useful ideas and concepts that can be directly applied to the organizational side health and wellness programs to better understand potential success and to design successful programs. A good example of this is shown in Exhibit A. This describes key factors in an organizational change management process. As Exhibit A shows, there are far reaching factors that impact the success of the change management process including leadership, management methods, measurement methods, organizational structure, technology, etc. Only when all are appropriately considered will the organizational change management process achieve its goals. Exhibit A’s overlay of four layers with the individual pieces further demonstrates the forces which can disrupt the change management process.

View Exhibit A

Even the diamond shape of the change management grid is philosophically significant. Organizational and change management experts regularly use the management principle known as “tight-loose”. Exhibit A visually presents an extension of this “tight-loose-tight”. The first tight refers to a tightly defined vision/mission, one that everyone buys into. The second tight refers to a tightly managed execution or process. The loose refers to flexible design and organizational process. Note that the top of Exhibit A comes to a point (i.e., tight, narrow), the middle part is wider, and the bottom again comes to a point. Visually it demonstrates “tight-loose-tight,” which we believe to be the most effective approach.

In terms of Health and Wellness, both the organization’s programmatic health and wellness approach and the individual’s personal interaction with the approach (described in more detail later) can be described in similar terms to that presented in Exhibit A and be mapped to this grid to demonstrate the key success factors. If an organization designs the right strategic program for implementation, the individuals who are participating will respond accordingly. Historically, most health and wellness programs have focused on what the individual should do. The more ideal approach for organizations and individuals is to really focus on and develop the why and how considerations.

The most effective health and wellness programs are ones with the tightest vision and mission statement. This is where the program clearly identifies, incorporates and strengthens the appropriate motivations for change (“the why”). When an organization is truly committed to the process, amazing things can occur. Very specific and definite goals result in the best results. (i.e., Tight and non-negotiable).

We believe a tightly defined health and wellness program vision should include the following three items:

  • A focused program — a program that begins with a particular aspect of health and wellness, rather than trying to be everything to everybody
  • A hope-filled program — a program that provides hope to participants, showing that it can be done and success is likely
  • A redemptive program — a program that encourages participants who participate, motivates them along the way, and incentivizes them to continue their transition. It is a program that gives “partial credit” for sincere participant efforts.

In addition, the most effective health and wellness programs incorporate a loose and flexible process or path. There are so many ways to structure and present programs, many with very similar results, that it is mandatory that the program incorporate flexibility. It is critical that the program tailor the specific application to best meet the needs of the individuals. Historically, this is where most of the narrow focus has occurred, rather than a “loose,” flexible approach. The exact structure and strategy (“the what”) need not be central in the context of genuine commitment and motivation. There is the capacity to customize and design specific strategies based on a host of unique and distinct considerations and preferences (i.e., Loose and flexible).

Finally, the most effective health and wellness programs have attracted highly committed individuals totally invested in the process (i.e., tight commitment). The sustained implementation (“the how”) is key for both short term and long term benefits and outcome. Predictably, having the appropriate commitment in the context of a customized strategy can lead to the implementation of a process that will lead to lasting change and benefit (i.e., Tight and mandatory).

Unfortunately, we find that far too many health and wellness programs are structured Loose-Tight-Loose. There is a very loosely and poorly communicated program vision and goal, nebulous at best, combined with a rigid and tightly defined inflexible structure, and a loosely structured connection with the individuals resulting in less than desired results. A complex and rigid program presented in an apathetic environment will likely lead to extensive expended resources without much meaningful benefit. In contrast, a tight-loose-tight program has a much greater likelihood of success, far above the general programs seen today.

For example, certain weight loss programs provide a rigid manual with points and/or limits forcing people into cookie cutter diet plans while doing little to effectively grow commitment to the end goal. These are clearly loose-tight-loose programs. Yes they oftentimes generate significant revenues to the sponsoring organization through membership and or meeting fees, with less than desired results and discouraged participants.

Individual Change Management
Just as with organization change, focusing on change management is key for any real impact to be made on one’s current health status. Whereas traditional health care for the most part focuses on treating illness and curing diseases, effective health and wellness programs focus on individuals becoming more committed to changing their behavior and hence improving the quality and potential for greater quantity of life. Personal change management principles offer valuable ideas and concepts that individuals can directly apply to improve the very quality of their lives. Exhibit B describes our model of the individual change management process as it relates to obesity. Just like with organizations, there are far reaching factors that impact the success of a person’s journey into new found health and wellness.

View Exhibit B

The egg shape of the personal or individual change management model is again philosophically and symbolically significant. Symbolically, unhealthy personal behavior often leads to carrying around extra weight around our midsections (i.e., egg-shaped physique). Philosophically, like organizational change the proposed individual approach is a tight-loose-tight model but with gentler, softer edges and curves in comparison to the sharp edges of the diamond grid. The top of the egg, being the tightest refers to the need to have tightly defined personal motivation and commitment. The second tighter region at the bottom of the egg refers to the tightly defined outcome of personal empowerment. The larger, “loose,” wider middle of the egg model refers to the customized and flexible design of a personal approach that encompasses certain core healthy pillars that anyone can adopt into the context of their lives. Exhibit B comes to a point of sorts at both ends with a large wider middle area which visually demonstrates the tight-loose-tight of personal change management.

In terms of personal Health and Wellness, an individual’s personal approach can be mapped to this model to demonstrate the key success factors. Just like with an organization, most individuals respond in a very similar way. Most adults who are relatively informed do not just need to be told “what” to do. They may already know at various levels. What they do need the most is the support, encouragement and accountability to just do it. What they need is the inspiration and hope to know it’s possible. Too many times we focus on instruction and neglect inspiration. We prioritize giving people help without adequately providing a sense of hope that change is possible. We emphasize technique and neglect motivation. The ideal approach to individual change management is one that develops motivation, develops sincere commitment to the process, develops hope for the potential that it can be done and builds inspiration about the consequences of it actually occurring.

On the personal or individual level motivation is key! What often is lacking is appropriate and meaningful motivation which translates to a greater sense of commitment. The concepts of health and wellness change are not that complicated. It’s the application of the simple principles into the complexity of an individual’s life which take thoughtful and strategic consideration. The tight-loose-tight structure directly applies here. The individual needs to clearly know (i.e., tight):

  • Why am I part of this program?
  • What is the end goal or outcome?
  • What will I achieve?
  • What are the benefits of me participating?
  • How much personal effort it will take? Too often this is inadequately presented and high recidivism or failure rates result from not buying in up front to what will likely occur.
  • What is the required process to be successful?
  • What are my chances of success?
  • Is this program clinically sound? What are the program’s credentials?

On a loose perspective, the individual needs to know what specific rewards or incentives are involved. Whether personally established by the individual or part of the overall program structure, the participant must value the rewards and let these motivate them in their transition process. There is a wide variety of incentives that could be used and uniquely tailored to each individual. Different individuals respond to different incentives, are motivated differently, and will perform differently. The program needs to react to these different perspectives.

Measurement systems are key to the individual, but their precise structure is less important. The individual needs to see that they apply to their lifestyle and normal thinking process. They have to be comfortable with what is going on.

On the bottom tight perspective, the individual needs to be closely held accountable and encouraged to continue the process they have begun. Without this, the individual may give up, stop the program, lose heart and assume it is hopeless.

1 Change management grid initially developed by Dutch Siedentopf, change management consultant.

Authors
David Axene collaborated with Nicholas Yphantides in writing this series of articles. Dr. Nicholas Yphantides serves as the Consulting Chief Medical Officer for San Diego County and is the National Director for Health & Wellness with Axene Health Partners. He is a cancer survivor and is an advocate for those in his community who need it the most. For nine years, Dr. Nick served as Chief Medical Officer of one the largest network of Community Clinics in San Diego County.