Speeches & Testimony

Exploring the Role of Patient Organizations in the Development of Orphan Products

Orphan Products Conference
Vienna, Virginia: September 16-17, 1999

The admirable progress of orphan drug development since 1983 can be credited to a remarkable partnership between the federal government, the pharmaceutical industry, academia, and consumer organizations representing people with rare diseases. The public and private sectors have been firmly committed to the success of the law, and voluntary health agencies are sensitive to the Act’s potential to ease the pain and suffering of the patients they represent. The main goal of each patient organization is to cure its disease and put itself out of business; this would not be possible if the pharmaceutical industry did not bring each orphan drug to market.

In 1983 the pharmaceutical industry did not recognize that we - the patients - are their customers. They thought that physicians were their customers, and focused on enticing doctors to prescribe particular brands of drugs. However, the Orphan Drug Act proved that the market for a drug could be driven by consumers, and today the Internet and television advertising confirm that it is good business to treat patients as your customers. In most cases, orphan medications are breakthrough therapies for which there is little or no competition. Patients demand these drugs from their doctors; if the doctor refuses to prescribe them, patients will go to another doctor.

How do rare disease patients find out about new orphan drugs? Patient organizations disseminate newsletters, they operate web sites, they communicate with the general public, physicians, the research community, etc. Companies hire expensive public relations firms to broadcast information about new drugs, but no one can directly reach the patients -- your customers -- more efficiently than a voluntary health agency. It is simply good business to work with patient organizations.

The problem is, voluntary health agencies and pharmaceutical companies don’t share a common goal. The role of the voluntary agency is to encourage research and try to get as many treatment options as possible for the patients they represent. The role of the pharmaceutical company is to market its drug and prevail over competitors. It’s important to enter relationships with patient organizations with a clear understanding of this fundamental difference . . . and to find ways to work to the mutual good in spite of it.

During the development stages of any orphan drug, companies need to work closely with a consumer group. That group’s Medical Advisory Committee will likely be the most prestigious group of experts with the largest number of patients with a particular diagnosis. The outreach of each voluntary health agency is more pervasive than your company could possibly accomplish, both to the scientific community and to the patients you need for clinical trials.

On the other hand, the relationship of any pharmaceutical company with a patient organization can be difficult and complex. It is important for the industry to understand this unique non-profit sector, and how you can maintain an ongoing relationship with your most important link to the consumers who will buy your products.

Support groups for rare diseases are usually started by families who are directly affected by the disorder. They may be parents of an affected child, a spouse, a sibling, etc. Most begin on a kitchen table because you cannot create programs and hire staff unless you succeed at fundraising. In general, the pioneer founders of voluntary health agencies are people unwilling to accept the awful fate of their loved ones, so they roll up their sleeves and do something about it.

As their organization grows, they attract doctors to their cause, and the doctors refer more patients to them. It soon becomes obvious that credibility is a major factor of success, and that any medical information distributed to the public must be reviewed and edited by physicians.

When an organization succeeds at fundraising, its members will undoubtedly develop a research grant program. They want to cure the disease and go out of business -- that’s their goal. But it sounds easier than it actually is. A lot of good money can be wasted on bad research. Every organization eventually realizes that laymen cannot judge the quality of research proposals, so they must institute a Medical Advisory Committee to formalize the peer review process and recommend which grants should be funded.

This process of building a Medical Advisory Committee means they must reach out and make friends with the scientific experts for their disease all over the country and the world. Moreover, they need to befriend basic scientists who may know nothing about the disease and interest them in integrating the disease into their research portfolios. For example, geneticists who may be studying related diseases might be enticed into adding another disease if they are told that the consumer organization may provide funding and encourage patients to submit blood samples.

So once a consumer organization experiences some success, has a degree of stability and achieves credibility, it is well underway. Meanwhile it continues to build its mailing list of patients -- lots of patients -- and when a disease is genetic the whole extended family may want to receive the organization’s newsletter because they’re eager for news of medical progress. The consumer organization is the repository for information about the diseases on which they focus.

Research grants may make a good newsletter article when they are awarded, but when they are completed it is not often very newsworthy. Progress is so incremental; expectations that there will be a major breakthrough because of any one research grant are unrealistic. This is why development of a treatment, whether in academia or the corporate world, is newsworthy in our non-profit environment. But a sophisticated organization knows that what they hope may be a breakthrough, may be a major disappointment as clinical trials progress. They will check with their medical advisors every step of the way before they print anything too positive in their newsletters because they don’t want to raise false hopes in their constituents. The only thing worse than no hope is false hope.

Medical advisors will want information: Is it a controlled trial or uncontrolled? What data do you have on preliminary clinical trials? Summarize the toxicology, etc. They will want to speak with the clinicians who have some experience with the drug, and they will report back to the consumer organization and carefully phrase any newsletter articles regarding the product.

The medical advisors will recommend whether the organization should cooperate in finding patients. If you are working on a rare disease that doesn’t have a patient organization, contact NORD and we’ll find the patients for you. But you must remember that consumer organizations will not release the names and addresses of patients to any outside source. Rather, they will mail a letter to patients, or print a newsletter article recommending that patients contact the clinical sites. Since these organizations are usually overwhelmed with work and very understaffed, you should make a donation to cover the costs of this project.

This is where the ethical line is drawn. Remember, a strong organization is built on credibility. So while it needs your donations, it will not take a donation with strings attached because its credibility might be threatened. Moreover, it cannot endorse your product or make it sound like the best invention since penicillin. Voluntary health agencies must maintain neutrality and any newsletter reports about your product must be unbiased. They must tell people the good news along with the bad, and your marketing people may not be happy about this. But this is their duty because they must always keep the welfare of patients foremost in mind.

Voluntary health agencies can evolve into your best friend or your worst nightmare, particularly if you are not open and honest with them. For example, the recent trend toward limiting the inventory of investigational drugs, and the need to create "lottery" systems when the demand outstrips the supply, does not encourage the respect and trust of the patient community. Additionally, once a drug is approved for marketing, patients will not be silent about extraordinarily high prices. To counter this, you must prepare for an indigent care program long before the drug is launched.

It is critically important that any medical assistance program is fair and unbiased, and that income parameters are not set too low. Remember, the rich can afford to buy your drug at any price, and the very poor will get it through Medicaid. It is the middle class who need medication assistance programs -- not just the uninsured, but also the underinsured. If you neglect to do this, you will find newspaper articles popping up all over the country complaining about your pricing, asking why local teachers and farmers have to hold a bake sale to raise money for their next dose of your medicine.

Similarly, a Treatment IND for an orphan drug should provide early access when no satisfactory alternative treatment exists, and particularly when there has been any degree of publicity about your drug. One of the big mistakes of the industry is failing to recognize that the information you release to Wall Street does not stay on Wall Street. It reaches Main Street. So when pre-launch publicity talks about the fabulous expectations you have for your drug, patients’ hopes are inflated, sometimes artificially if the FDA does not get the data to back up any hype.

What happens after your drug is launched? After it becomes apparent to your marketing people that the voluntary health agency can reach your customers more easily than you can? How can you sustain a good relationship with the patient organization? And how can a non-profit agency benefit from your support without appearing like it’s been bribed by your company?

One way to ruin a relationship is to expect the organization to praise your drug in response to a hefty donation. Don’t get me wrong, patient organizations need donations. They often exist from hand to mouth. However, it is their responsibility to be unbiased and accurate, so they must talk about the negative aspects of your drug, as well as the positives. It is much more rational for them to praise your company in their newsletters than it is to praise your drug, because they cannot be perceived as endorsing your product or demeaning the product of your competitors.

Keep in mind that you may have one drug, but it is the role of the patient organization to encourage other companies to develop additional treatment options. Thus your company may be hoping to prevent competition, but the patient organization will be working to promote competition because this is their mission. This is usually the core of any conflict that may arise in your relationship with consumer organizations. If you have realistic expectations when you start this relationship, you can avoid conflicts.

The field of voluntary agencies is very sensitive to scandal. Organizations accused by their members of being bribed by business interests usually never recover. Even new leaderships will be tainted by past scandals, and donations from the public will be greatly hampered. This is why your support must come with no strings attached. Even if it appears to have strings, it could be disastrous.

For example, one organization accepted a grant from a company that manufactured drug to promote growth in children, to go to schools and measure the height of children. If a child was below the 90th percentile of normal height for his or her age, a note was sent to the parents suggesting that they talk to the child’s doctor about this drug. This grant does not pass the New York Times test; if the story appears in the Times, would it look bad? Well, it did appear in the Times as well as the Washington Post and virtually every other newspaper in the country. The organization still hasn’t recovered from the bad publicity, and the company paid a hefty fine to the Federal Trade Commission. It’s just not worth it.

Your goal should be to keep your good name in front of your customers, the people who need your orphan drug. You can do this through unrestricted educational grants to the patient organization. For example, they can print credit to your company in their newsletters for supporting their educational programs; you can sponsor their meetings or publications through which they will acknowledge your support, but you cannot have editorial control over what they print or say; or you can give them a grant to support outreach efforts that will get patients diagnosed earlier, as long as you don’t suggest these patients should take your drug. You need to build an image of a compassionate and socially responsible corporation who wants to help patients and their organization to ease pain and diminish the suffering of families.

To this end, it is critically important that you are honest and open with the patient community so they will never lose faith in your willingness to be truthful, even if the news is bad. Patients talk to each other, and a rumor on the World Wide Web can have devastating consequences for your company, particularly because the Internet is a technological wonder that never erases old messages left on bulletin boards.

I was recently monitoring a bulletin board for a disease that is treated with a drug that is experiencing manufacturing problems. A patient noticed that his pharmacist would no longer give him a three-month supply of the drug and was limiting refills to 30 days. After three or four months, he compared each bottle and noticed they all had the same expiration date. He got on the Internet and spread a rumor that the company was ceasing manufacture of the drug and was leaving the patients to die.

After a few days of this rumor scaring the daylights out of patients all over the world, I picked up the phone and talked to the company. They could have intervened earlier and eased the fright of these patients, but I learned that their legal department had been undecided about what to do, and so had done nothing. By that time, the messages on the web regarding the company were transitioning from "R" rated to "X" rated words. This was a big multi-national company that should not have risked any one of those patients going to 60 Minutes or Dateline with their story. Accusing a respected multi-national company of being a murderer makes for interesting television entertainment, and this should be avoided at all costs with rapid and decisive crisis intervention.

In this case, the group of patients did not have a formal non-profit organization to referee the argument, so NORD had to step in. But had a formal patient organization existed, a spokesman from the company should have been selected to create an alliance with the patient community and keep them informed of the temporary manufacturing difficulties and how the company was trying to overcome them. The company could have been a hero to the patients; instead it was a villain because of indecisiveness and inaction.

What I am saying here is that this partnership between the industry, the government, and patient organizations is a marriage that has to be worked on every day. Marriage calls for compromise, and it is fraught with daily challenges, but we each need to work at keeping the marriage intact. Divorce is not an option.

We patients need pharmaceutical companies to manufacture our drugs. The companies need us to buy their products. We both need the government to fund the research and ensure that the treatments are safe and effective. If the FDA wasn’t there, you would be in the nutritional supplement business and we would probably be dead. Then there would be no market for your products.

So it is to your benefit to keep the voluntary health sector strong, and to our benefit to make sure you are innovative and profitable. This is why patient organizations are important in the development of orphan products.

In 1983 the pharmaceutical industry did not recognize that we - the patients - are their customers. They thought that physicians were their customers, and focused on enticing doctors to prescribe particular brands of drugs. However, the Orphan Drug Act proved that the market for a drug could be driven by consumers, and today the Internet and television advertising confirm that it is good business to treat patients as your customers. In most cases, orphan medications are breakthrough therapies for which there is little or no competition. Patients demand these drugs from their doctors; if the doctor refuses to prescribe them, patients will go to another doctor.

How do rare disease patients find out about new orphan drugs? Patient organizations disseminate newsletters, they operate web sites, they communicate with the general public, physicians, the research community, etc. Companies hire expensive public relations firms to broadcast information about new drugs, but no one can directly reach the patients -- your customers -- more efficiently than a voluntary health agency. It is simply good business to work with patient organizations.

The problem is, voluntary health agencies and pharmaceutical companies don’t share a common goal. The role of the voluntary agency is to encourage research and try to get as many treatment options as possible for the patients they represent. The role of the pharmaceutical company is to market its drug and prevail over competitors. It’s important to enter relationships with patient organizations with a clear understanding of this fundamental difference . . . and to find ways to work to the mutual good in spite of it.

During the development stages of any orphan drug, companies need to work closely with a consumer group. That group’s Medical Advisory Committee will likely be the most prestigious group of experts with the largest number of patients with a particular diagnosis. The outreach of each voluntary health agency is more pervasive than your company could possibly accomplish, both to the scientific community and to the patients you need for clinical trials.

On the other hand, the relationship of any pharmaceutical company with a patient organization can be difficult and complex. It is important for the industry to understand this unique non-profit sector, and how you can maintain an ongoing relationship with your most important link to the consumers who will buy your products.

Support groups for rare diseases are usually started by families who are directly affected by the disorder. They may be parents of an affected child, a spouse, a sibling, etc. Most begin on a kitchen table because you cannot create programs and hire staff unless you succeed at fundraising. In general, the pioneer founders of voluntary health agencies are people unwilling to accept the awful fate of their loved ones, so they roll up their sleeves and do something about it.

As their organization grows, they attract doctors to their cause, and the doctors refer more patients to them. It soon becomes obvious that credibility is a major factor of success, and that any medical information distributed to the public must be reviewed and edited by physicians.

When an organization succeeds at fundraising, its members will undoubtedly develop a research grant program. They want to cure the disease and go out of business -- that’s their goal. But it sounds easier than it actually is. A lot of good money can be wasted on bad research. Every organization eventually realizes that laymen cannot judge the quality of research proposals, so they must institute a Medical Advisory Committee to formalize the peer review process and recommend which grants should be funded.

This process of building a Medical Advisory Committee means they must reach out and make friends with the scientific experts for their disease all over the country and the world. Moreover, they need to befriend basic scientists who may know nothing about the disease and interest them in integrating the disease into their research portfolios. For example, geneticists who may be studying related diseases might be enticed into adding another disease if they are told that the consumer organization may provide funding and encourage patients to submit blood samples.

So once a consumer organization experiences some success, has a degree of stability and achieves credibility, it is well underway. Meanwhile it continues to build its mailing list of patients -- lots of patients -- and when a disease is genetic the whole extended family may want to receive the organization’s newsletter because they’re eager for news of medical progress. The consumer organization is the repository for information about the diseases on which they focus.

Research grants may make a good newsletter article when they are awarded, but when they are completed it is not often very newsworthy. Progress is so incremental; expectations that there will be a major breakthrough because of any one research grant are unrealistic. This is why development of a treatment, whether in academia or the corporate world, is newsworthy in our non-profit environment. But a sophisticated organization knows that what they hope may be a breakthrough, may be a major disappointment as clinical trials progress. They will check with their medical advisors every step of the way before they print anything too positive in their newsletters because they don’t want to raise false hopes in their constituents. The only thing worse than no hope is false hope.

Medical advisors will want information: Is it a controlled trial or uncontrolled? What data do you have on preliminary clinical trials? Summarize the toxicology, etc. They will want to speak with the clinicians who have some experience with the drug, and they will report back to the consumer organization and carefully phrase any newsletter articles regarding the product.

The medical advisors will recommend whether the organization should cooperate in finding patients. If you are working on a rare disease that doesn’t have a patient organization, contact NORD and we’ll find the patients for you. But you must remember that consumer organizations will not release the names and addresses of patients to any outside source. Rather, they will mail a letter to patients, or print a newsletter article recommending that patients contact the clinical sites. Since these organizations are usually overwhelmed with work and very understaffed, you should make a donation to cover the costs of this project.

This is where the ethical line is drawn. Remember, a strong organization is built on credibility. So while it needs your donations, it will not take a donation with strings attached because its credibility might be threatened. Moreover, it cannot endorse your product or make it sound like the best invention since penicillin. Voluntary health agencies must maintain neutrality and any newsletter reports about your product must be unbiased. They must tell people the good news along with the bad, and your marketing people may not be happy about this. But this is their duty because they must always keep the welfare of patients foremost in mind.

Voluntary health agencies can evolve into your best friend or your worst nightmare, particularly if you are not open and honest with them. For example, the recent trend toward limiting the inventory of investigational drugs, and the need to create "lottery" systems when the demand outstrips the supply, does not encourage the respect and trust of the patient community. Additionally, once a drug is approved for marketing, patients will not be silent about extraordinarily high prices. To counter this, you must prepare for an indigent care program long before the drug is launched.

It is critically important that any medical assistance program is fair and unbiased, and that income parameters are not set too low. Remember, the rich can afford to buy your drug at any price, and the very poor will get it through Medicaid. It is the middle class who need medication assistance programs -- not just the uninsured, but also the underinsured. If you neglect to do this, you will find newspaper articles popping up all over the country complaining about your pricing, asking why local teachers and farmers have to hold a bake sale to raise money for their next dose of your medicine.

Similarly, a Treatment IND for an orphan drug should provide early access when no satisfactory alternative treatment exists, and particularly when there has been any degree of publicity about your drug. One of the big mistakes of the industry is failing to recognize that the information you release to Wall Street does not stay on Wall Street. It reaches Main Street. So when pre-launch publicity talks about the fabulous expectations you have for your drug, patients’ hopes are inflated, sometimes artificially if the FDA does not get the data to back up any hype.

What happens after your drug is launched? After it becomes apparent to your marketing people that the voluntary health agency can reach your customers more easily than you can? How can you sustain a good relationship with the patient organization? And how can a non-profit agency benefit from your support without appearing like it’s been bribed by your company?

One way to ruin a relationship is to expect the organization to praise your drug in response to a hefty donation. Don’t get me wrong, patient organizations need donations. They often exist from hand to mouth. However, it is their responsibility to be unbiased and accurate, so they must talk about the negative aspects of your drug, as well as the positives. It is much more rational for them to praise your company in their newsletters than it is to praise your drug, because they cannot be perceived as endorsing your product or demeaning the product of your competitors.

Keep in mind that you may have one drug, but it is the role of the patient organization to encourage other companies to develop additional treatment options. Thus your company may be hoping to prevent competition, but the patient organization will be working to promote competition because this is their mission. This is usually the core of any conflict that may arise in your relationship with consumer organizations. If you have realistic expectations when you start this relationship, you can avoid conflicts.

The field of voluntary agencies is very sensitive to scandal. Organizations accused by their members of being bribed by business interests usually never recover. Even new leaderships will be tainted by past scandals, and donations from the public will be greatly hampered. This is why your support must come with no strings attached. Even if it appears to have strings, it could be disastrous.

For example, one organization accepted a grant from a company that manufactured drug to promote growth in children, to go to schools and measure the height of children. If a child was below the 90th percentile of normal height for his or her age, a note was sent to the parents suggesting that they talk to the child’s doctor about this drug. This grant does not pass the New York Times test; if the story appears in the Times, would it look bad? Well, it did appear in the Times as well as the Washington Post and virtually every other newspaper in the country. The organization still hasn’t recovered from the bad publicity, and the company paid a hefty fine to the Federal Trade Commission. It’s just not worth it.

Your goal should be to keep your good name in front of your customers, the people who need your orphan drug. You can do this through unrestricted educational grants to the patient organization. For example, they can print credit to your company in their newsletters for supporting their educational programs; you can sponsor their meetings or publications through which they will acknowledge your support, but you cannot have editorial control over what they print or say; or you can give them a grant to support outreach efforts that will get patients diagnosed earlier, as long as you don’t suggest these patients should take your drug. You need to build an image of a compassionate and socially responsible corporation who wants to help patients and their organization to ease pain and diminish the suffering of families.

To this end, it is critically important that you are honest and open with the patient community so they will never lose faith in your willingness to be truthful, even if the news is bad. Patients talk to each other, and a rumor on the World Wide Web can have devastating consequences for your company, particularly because the Internet is a technological wonder that never erases old messages left on bulletin boards.

I was recently monitoring a bulletin board for a disease that is treated with a drug that is experiencing manufacturing problems. A patient noticed that his pharmacist would no longer give him a three-month supply of the drug and was limiting refills to 30 days. After three or four months, he compared each bottle and noticed they all had the same expiration date. He got on the Internet and spread a rumor that the company was ceasing manufacture of the drug and was leaving the patients to die.

After a few days of this rumor scaring the daylights out of patients all over the world, I picked up the phone and talked to the company. They could have intervened earlier and eased the fright of these patients, but I learned that their legal department had been undecided about what to do, and so had done nothing. By that time, the messages on the web regarding the company were transitioning from "R" rated to "X" rated words. This was a big multi-national company that should not have risked any one of those patients going to 60 Minutes or Dateline with their story. Accusing a respected multi-national company of being a murderer makes for interesting television entertainment, and this should be avoided at all costs with rapid and decisive crisis intervention.

In this case, the group of patients did not have a formal non-profit organization to referee the argument, so NORD had to step in. But had a formal patient organization existed, a spokesman from the company should have been selected to create an alliance with the patient community and keep them informed of the temporary manufacturing difficulties and how the company was trying to overcome them. The company could have been a hero to the patients; instead it was a villain because of indecisiveness and inaction.

What I am saying here is that this partnership between the industry, the government, and patient organizations is a marriage that has to be worked on every day. Marriage calls for compromise, and it is fraught with daily challenges, but we each need to work at keeping the marriage intact. Divorce is not an option.

We patients need pharmaceutical companies to manufacture our drugs. The companies need us to buy their products. We both need the government to fund the research and ensure that the treatments are safe and effective. If the FDA wasn’t there, you would be in the nutritional supplement business and we would probably be dead. Then there would be no market for your products.

So it is to your benefit to keep the voluntary health sector strong, and to our benefit to make sure you are innovative and profitable. This is why patient organizations are important in the development of orphan products.

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