Speeches & Testimony

Collaboration Between the Pharmaceutical Industry and Patient Organizations. A Delicate Balance

Abbey S. Meyers

President

National Organization for Rare Disorders (NORD)

Spain

International Conference of Rare Diseases and Orphan Drugs

February 17, 2000

The National Organization for Rare Disorders (NORD) in the United States is the patient coalition that advocated for the American Orphan Drug Act, which became law in 1983. Since that time, NORD has monitored implementation of the law and worked vigorously with pharmaceutical companies, academic researchers, and the government to ensure that orphan drugs will be developed and made available to patients.

This work consumes a great deal of our time and resources because it is not an easy task. One would think that if all parties acted in the cooperative spirit of the Orphan Drug Act, there would be a strong partnership between the government, the industry, and consumer organizations. However, there are many times that we each have divergent interests because we represent different constituencies that have dissimilar goals. There are times, for example, when some companies, and even trade groups, may be upset with NORD because we will not endorse an item on their political agenda. There are also times when consumers will become angry with individual companies or the entire industry.

The government, through the FDA (the Food & Drug Administration), regulates the industry, and the government’s constituency is the general public. The FDA will be criticized if a drug harms people after it reaches the American market, it will be criticized if the drug is ineffective, and it will be criticized for virtually everything else it does (and does not do) because its statutory neutrality is bothersome to both the industry and the general public.

Problems between the industry and patient organizations often arise because pharmaceutical companies sometimes do not understand the nature of our work. Consumer organizations are charities, dedicated to the public’s welfare and regulated by national charity laws. Rare disease organizations are usually created to stimulate medical research that will ultimately cure the disease and put the charity out of business. These patient groups will search out biomedical researchers, and encourage their interest in the disease. If a researcher discovers a new treatment, the patient organization will reach out to patients with that diagnosis and encourage them to participate in clinical trials. When the product reaches the commercial market, they will continue to educate patients about the positive and negative aspects of the treatment because patient organizations must be unbiased and scientifically accurate or they will lose their credibility. They are educators, but they cannot advise patients whether or not to use a treatment. Because their primary role is to find a cure, patient organizations will continue to promote more research and development of improved treatments that may ultimately compete with the original orphan drug.

This work, which is essential to the patient community, is often at odds with the goals of pharmaceutical companies. Drug manufacturers want patients to know the positive aspects of their products, not the negative problems. Companies usually do not appreciate patient organizations encouraging other researchers and companies to develop treatments that will compete with their drug. Thus, the problems begin: It is the work of patient organizations to promote development of many treatment options, and it is the goal of pharmaceutical companies to prevent or delay competition for as long as they possibly can. It is to be expected then, that charities and drug companies will often become adversaries under such circumstances.

NORD’s Role in Orphan Drug Development

NORD often becomes involved in development of new orphan products, especially when there is no patient organization representing a specific disease. Sometimes we are asked to get involved by an academic scientist or a pharmaceutical company that is looking for patients with a certain diagnosis who may wish to participate in a clinical trial.

NORD is primarily a clearinghouse for information about rare diseases. People who contact us are entered into a computer with the name of the disease about which they inquired. Therefore, although we will not give the names and addresses of patients to companies or investigators, we can contact these patients directly and tell them about the clinical trial. If they are interested in participating, we urge them to contact (or have their doctor contact) the investigators. First, however, our medical advisors must review and approve an abbreviated description of the protocol. If the trial is approved, we will also add it to our Rare Disease Database entry on our web site <www.rarediseases.org>. Thus, all people who inquire about the disease in the future will learn about the trial until the investigators inform us that recruitment is no longer needed.

When academic investigators are unable to find a company willing to develop an orphan product commercially, NORD will help locate potential sponsors. In the United States, many small companies have evolved around the Orphan Drug Act, and they are grateful if we can find potential products that may interest them. It is important for us to know the types of product lines in which each company specializes. For example, it would not be appropriate for us to solicit the sponsorship of a company that specializes in dermatology products if the orphan product is for an eye disease.

We also try to help small companies and academic investigators who are looking for research funding. NORD funds a few small clinical trials each year, which can provide academic investigators with enough data to later apply for larger government grants. The FDA has an Orphan Products Research Grant Program that is very competitive because they have only $11.5 million annually. But there are also other government programs such as Small Business Innovative Research Grants and, of course, grants from the National Institutes of Health.

During the development process, companies often ask us for advice. Small companies, especially, may not be very knowledgeable about the FDA drug approval process, and they often wonder why the agency is not very responsive. Even larger companies will ask us about the politics of a research community for certain diseases, and where they might contact additional experts if they want to broaden their clinical trials. They also ask us for names of consultants, or our opinions of certain consultants whom they are considering. They ask us if we know of possible European or Asian partners because small American companies usually do not have a marketing presence in those areas of the world. They ask us a lot about the FDA. We have to tell them there is no magic formula or short cut for getting an orphan drug approved by the agency because we consumers truly want the product to be safe and effective, so we do not want FDA to institute lower standards of approval for orphan drugs.

Sometimes companies take our advice, and sometimes they don’t. And when they don’t, they sometimes come back to us and say, “We should have listened to your advice.” We often hear from them when they are desperately looking for investment capitol. This is an area that we cannot help them with; NORD’s focus is medical, not financial.

While an orphan drug is in phase III testing, the FDA will often agree to (and sometimes request) a “Treatment IND.” This special permission allows pharmaceutical companies to provide the drug to a wider number of patients while it is still experimental. We have helped some companies to do this by administering a “random selection” process. The FDA and the company will agree on how many patients will be allowed to get the drug under the Treatment IND, and NORD will select the patients to ensure that the selection is unbiased and randomized. There are usually a great many more patients who want the orphan drug than the amount of drug that is available. We will monitor the patients until the drug is approved for marketing by the FDA. Once the product is on the market, we can administer indigent care programs to needy patients who cannot afford to purchase the drug. This is because we do not have universal health insurance in the United States, and we have 44 million uninsured Americans, many of whom cannot afford to purchase prescription drugs.

What are the Problems?

In the United States, we designed the Orphan Drug Act with financial incentives that we hoped would entice pharmaceutical companies into developing treatments for rare diseases. The incentives have worked very well, primarily because the companies want to be assured that they can prevent competition by other manufacturers, for the same drug, for seven years. However, some companies unreasonably expect that theirs will be the only treatment for that disease. Patient organizations will always encourage other companies to develop other drugs for the same disease, so that patients will have many options to choose from. Moreover, there is nothing to stop another manufacturer from getting an orphan drug designation for a product that already has seven years exclusivity, and marketing that same drug for a different disease. The designation locks marketing exclusivity to a specific disease, so other companies can market the same product for another disease if they perform the necessary research.

Sometimes companies ask us to lobby for issues that are clearly not in the patient’s best interest. We cannot, and will not, do that. For example, reimbursement issues are not patient centered issues. They are corporate issues, and we will not get involved. Similarly, European patients will not be concerned with orphan drug reimbursement issues unless their national health insurance refuses to pay for the drug, in which case it is likely that they will pressure national governments as well as the orphan drug manufacturer to seek a compromise.

Pricing, however, is often a major factor in the American market because patients are sometimes responsible for paying part or all of the cost of drugs. Additionally, insurance companies often omit expensive drugs from their formulary lists, and the sector of our population that buys the most drugs...the elderly...do not have drug reimbursement benefits through Medicare. Therefore, the great majority of elderly patients must pay for the entire cost of their drugs, and they are a politically powerful group that is quite vocal about drug pricing issues.

We certainly understand that orphan drugs will be more expensive than most other drugs because they are sold to fewer people. Manufacturers must earn a higher profit on each pill or injection than they would on each tablet of an arthritis or hypertension drug. Moreover, biotechnology drugs tend to be very expensive for both rare and common diseases, and many of the new orphan drugs are biotech products.

In the United States, our private insurance system greatly complicates pricing problems because the system is aimed at insuring generally healthy people who suffer common and temporary health events, not chronic diseases. Most Americans have a $1 million life time “cap” on their insurance benefits. If they exceed this maximum, they will be uninsurable for the rest of their life. Many have health insurance that will not pay for drugs, or their pharmaceutical benefits may be capped at only $500 or $1,000 per year. It is not unusual for a biotech drug to cost more than $10,000 per year, and very few people can afford such an annual expense for a chronic disease.

Therefore, pharmaceutical prices are a major concern to the American patient community, and indeed to the entire United States population. Although American politicians have not done anything to solve the problem, drug prices are always a major factor in political campaigns. This year, the politicians are especially vocal about pharmaceutical price increases that are three to four times higher than general inflation.

Unfortunately, American trade organizations such as the Pharmaceutical Research and Manufacturer’s Association (PhRMA), and the Biotechnology Industry Organization (BIO), have simply made the problem worse. Today the pharmaceutical industry is one of the least liked industries in the United States (probably second only to the oil industry). They defend their pricing by insisting that they must earn very high profits in order to invest in research and development of new drugs, but they spend twice as much on marketing and advertising as they do on R&D. Many drugs are sold at a much higher price in the USA than they cost in the rest of the world, which has forced many elderly people to obtain their drugs from Canada and Mexico. Americans are very angry that the brand-name pharmaceutical industry spent $30 million lobbying last year to prevent prescription drugs from being added to Medicare benefits.

Patient organizations who understand the politics of health care are angry and disgusted with the U.S. pharmaceutical industry when issues like these arise. We are astounded when the industry asks patient organizations to support its political agenda when it is in direct conflict with our mission to aid the downtrodden and ease the devastation of illness. I am convinced that the industry doesn’t understand the patient community and trade groups keep repeating mistakes because they have not taken the time to define the parameters of cooperation that are necessary for a productive relationship.

Working with Individual Companies:

Instead of working with trade groups, NORD works closely with individual companies on individual products that will affect specific populations of patients. We find these relationships rewarding because there is a clear understanding of where the ethical line is drawn, and our role as a patient advocate is clearly defined. If their orphan drug is safe and effective, we want it to reach the American market so that patients will be able to obtain it at their local pharmacy. However, we will not endorse their product, we will not tell patients it is the greatest remedy since aspirin, and we will actively encourage competitors to bring other drugs to market for the same disease.

The fact is, patients are their customers, and patient organizations can reach the people who will buy their drug. We can educate the public and physicians so that people with rare diseases will get early diagnosis and treatment. Too often drug companies feel that the doctors who write prescriptions are their customers. But it is the patient who decides whether to take the drug, and whether to continue taking it. In the United States, companies have finally come to recognize this fact because direct-to-consumer advertising has vastly changed the market.

The consequences of working with NORD are very clear. If a company is going to do something that is not helpful to patients, we will not support it. In a way, we are their conscience, and I believe that U.S. companies have come to realize that they can avoid making serious mistakes if they first consult with NORD about the priorities and goals of the patient community.

Maintaining Relationships:

The relationship between patient organizations and the pharmaceutical industry is extremely important, and such relationships must be carefully nurtured not only in the USA, but throughout the world. Any company that decides to develop an orphan drug should recognize that the market for that drug does not end at any national border. It is likely that the rest of the world will need that orphan drug for some time to come because there are no other adequate treatment alternatives.

The protocol for relationships with patient organizations in different cultures will vary widely, and it is imperative that companies understand the differences. For example, Asian cultures demand more privacy than western countries because people want to keep their health problems a secret that is shared only with their families. Conversely, Americans will reach out to strangers and reveal their entire medical history, while Europeans fall somewhere between the two extremes. In all cultures, however, there will be one common thread that ties all patient organizations together, and that is the fact that we will always be patient advocates who want to cure our disease, close our doors, and go out of business. This will only become possible if the pharmaceutical industry develops those cures and makes them commercially available. We need the pharmaceutical industry to achieve this dream, and the pharmaceutical industry needs us because we are their customers.

Since 1983, this relationship between rare disease patients and drug companies in the United States has been productive and rewarding. I have no doubt that it will be replicated throughout the European Union in the years ahead if pharmaceutical companies truly understand and accept the unique role of patient community organizations for rare diseases.

Thank you.

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