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November Fireside Chat Recap: Patient Access to Drugs

Hi, all! In our second Fireside Chat, on November 1, we started what we’re sure will be an ongoing conversation about patient access to drugs. The discussion kicked off with a blog post by FAST Board Member Lauren Hoffer in advance of the rollout of a caregiver burden survey.

One of the promises I made about these monthly firesides is that I would do my best to bring in leaders from the wider nonprofit and rare disease spaces, to give community members a window into how this work gets done professionally beyond the world of AS. With this in mind, I was thrilled to be able to welcome Preston Campbell, the former president and CEO of the Cystic Fibrosis Foundation (CFF). A leader at the CF Foundation for more than 20 years, Dr. Campbell previously served as the Foundation’s executive vice president for medical affairs, overseeing the Foundation's research, drug discovery, drug development and clinical research programs, the Foundation's network of care centers, clinical training programs and the national patient registry database.

In the course of this chat, Dr. Campbell graciously gave us a sense of how CFF plays a role in patient access to drugs for their community, and provided his own outsider’s sense of how FAST is doing in this space. Here’s why we featured him:

The Two Organizations: At a Glance

Organizational modelVenture philanthropy modelVenture philanthropy model
Profit from royalties$3.3 Billion$52 Million
MissionThe mission is to cure cystic fibrosis and to provide all people with CF the opportunity to lead long, fulfilling lives by funding research and drug development, partnering with the CF community, and advancing high-quality, specialized care.\Our goal is to cure Angelman syndrome and bring practical treatments into current medical practice as quickly as possible.
Paid Employees7505
# of affected persons100,000 in registry500,000 suspected / data collection ongoing
Disease-Specific Treatments Available40
How Foundation initiatives have to date impacted people living with the respective condition-Changed the natural history of Cystic Fibrosis, from less than 10-year life expectancy to now 66 years old for 90% of the population with most of that change in the last 25 years.  
-One genotype still without a targeted drug.
Not yet.

We talk a lot at FAST about all the progress we are making, and how well we are doing--and it’s true!--but it is also important to highlight the ways that the organization must grow in the coming years. The drug development pipeline is the heart of our work, but it won’t do anyone any good if we help to develop treatments that no one can afford. It is our responsibility to make sure we have not only treatment options but the complex infrastructure to support their success--a task that involves layers of regulatory, advocacy and messaging work that, as every other successful rare disease organization shows, cannot be avoided.

Below is a collection of points made during the chat, followed by answers to a series of questions sent in to FAST from community members. We are so thankful to Dr. Campbell for participating, and to everyone in the community who joined for the discussion. --Alana Newhouse

CFF’s strategy for patient access to therapies:

  • The Cystic Fibrosis Foundation serves as a model to FAST and all patient advocacy organizations in that they have dramatically changed the prognosis for those living with cystic fibrosis for the better. Because of CFF’s research efforts, the life expectancy of someone born with CF has gone from less than 10 years old to now 66 years old.  Most of this change has happened in the last 25 years, with Dr. Preston Campbell at the helm.
  • Like FAST, the Cystic Fibrosis Foundation funded the seed research for its drugs and maintained economic interest in those drugs to a certain point. In their case, CFF sold its royalties for $3.3 billion, both to avoid a conflict of interest making money off of their therapies but also to make the process go faster. They didn’t want others to think CFF was making money from patients buying a certain therapy. This $3.3B is in addition to funds they already had and continue to fundraise.
  • Even with its initial investment in the research and its strong advocacy for patient access, the Cystic Fibrosis Foundation did not have a say in how any pharma company priced its drug.
  • CFF does not offset the cost of patient drugs for their community, something they have put a lot of thought into. The reason they do not is because they would be out of money in a very short period of time. 10% of their patient population has no medicine at all, and they have not cured CF for any of the population.  They know that no one else will drive these medicinal innovations.  Even with the money they have now, if they only paid for even the modulator treatments, they would be out of money in 2.5-3 years and still without a cure.
  • They understand that Biotech and Pharma are also fighting for access but with different motives. CFF’s motive has always been patient access to these therapies. 
  • Their fight will focus on how insurance companies want to deal with copays, caps, etc. and how to support patients along the way. That is why they launched the platform Compass® to help their community work with insurance companies and Medicaid.
  • They formed a policy team that started as 3-4 volunteers and is now over 30 to help tackle real world issues that arise when a drug is approved. They make sure CF patients have access to care and therapies. To do that, they integrate at the federal level and have a CF caucus with over 130 members with multiple events throughout the year in order to push issues important at that time.
  • They understand the importance of their community to help them fight for patient access and realize that the strength of the community will define how well the foundation does.

FAST’s strategy for patient access to therapies:

  • As mentioned in Lauren’s blog, FAST has been working for the past 2 years to learn what it will take to support patient access to drugs, once available. 
  • We have hired experts to map out a plan and are happy to share that a lot has been done already.
  • We are currently working on this in multiple areas: evidence to understand the present monetary cost of living with Angelman syndrome, a study to understand true life expectancy for AS, Medicaid advocacy, and supporting numerous newborn screening initiatives.
  • Our advocacy to support future access will be done in conjunction with the pharmaceutical companies. Currently, our consulting firm is working pro bono. FAST’s initial task to them is to map out a plan for which states to go to when, what meetings with what material and which people. If there are opportunities to pre-wire decision makers around access, such as the Centers for Medicare and Medicaid Services (CMS)- not about a specific drug, but about AS in general--we don’t want to miss those opportunities. We want to make sure we have the material ready to show them--from a patient perspective. We want to make sure that whatever opportunities there are for a patient organization to advocate, we know when they are, when the right time is to go, and get on the meeting agenda.  
  • We have also formed a policy team through our FAST Advisory Council (FAC) of volunteers. They are identifying openings in state rare disease committees and boards for potential to place Angelman advocates on those boards.
  • With regard to a patient assistance program: This is a very important topic, and healthy debate and advocacy is welcome and appreciated. We are so lucky to be at a point where we are even discussing these topics. Under past and present leadership, in its history, FAST has not had a patient assistance program. From its inception, FAST has not paid for drugs currently on the market that our community may need - e.g., seizure or behavior drugs or supplements. We are focused on research to find a cure for AS.  With regard to the potential disease-modifying medicines currently in trials, it is too early to answer the question as to whether FAST will have a patient assistance program and how it would work. Many factors would influence the decision.
  • We are not aware of any rare disease patient organization that has a patient access program that offsets the cost of approved drugs. We have checked with Cystic Fibrosis Foundation, CureSMA, CureDuchenne, and our experts have checked with colleagues and none have heard of any patient org having this. This includes organizations that work on fatal diseases, including cancer. The National Organization of Rare Diseases, which advocates broadly for over 300 rare disease member groups, is one of the only non-profit advocacy groups with an independent program to provide financial support for co-pays and out of pocket costs to individuals with limited resources.  NORD determines which rare diseases will be part of its program and invests $30M annually to provide assistance to patients with > 30 rare diseases.  
  • The FAST board will continue to monitor the landscape, debate, listen to the community, and make decisions to the best of their ability that they believe are in the overall best interest of every person living with AS.
  • Why is it too early to make this decision? The money we have, while it is substantial for any personal wealth, it is not substantial in the universe of drug development. The clinical trials in AS are still in early phases, and these drugs have not even been tried in some age groups and some genotypes. Until we are further along and know if these drugs will be approved, and for whom, we must stay committed to research and drug development for all.
  • We are working hard on this economic initiative so that, when the day comes, we won’t have to choose between further research and helping pay for medicines on the market. We want to be ready when any of these medicines is approved, and we want to use the time we have in anticipation of an approval wisely.  We want insurance/Medicaid/governments to cover the therapies because that is best for the community as a whole. This is what we are working on: Getting it covered--for everyone. With our community's help, we feel optimistic we can make a positive difference here.

Next steps for the Angelman syndrome community to help:

  • Raise your hand if you are interested in volunteering on the policy team so we can have representation in all 50 US states! Email
  • Fill out the Caregiver Impact Survey (that will be released later this year) so the AS community can showcase the cost of caring for a person with AS when negotiating drug prices in the future. 
  • Ensure your loved one with AS has the ICD10 code: Q93.51 on all of their health records so we can gather as much data as we can on the cost of supporting a loved one with Angelman syndrome.
  • According to Preston, one of the best ways to work on this issue is to actively engage in the advocacy policy space. At CFF, they engage at all levels--from federal work; a CF caucus with over 130 members; 2 events per year; teen advocacy day to visit representatives on Capitol Hill, as well as a march on the Hill for adults; and more. They’re also active in state programs – having grown their policy group because every state is different. In short, we need a policy team that works throughout the country, and is driven not only by staff but by an army of volunteers! We need people sharing the AS story on a local level, explaining to our local representatives the hope and obstacles and all that we are doing to make a change. Let them see that we are making history, and they can be part of it if they help us.
  • Work together! Once therapies are approved, the community will be up against very real world issues. These will need to be lined up, prioritized, and tackled. As Preston stated: "You need the community for this. The strength of the AS community will define how well it does.” We need to be ready to act, call, write to knock off issues--together.


Q: My understanding is that many companies might have charitable access programs --or support programs helping families navigate insurance hurdles, and out-of-pocket costs. If that’s the case, why does FAST need to do this? Aren’t you just duplicating this work?

A: We are definitely not duplicating pharma’s work when it comes to the economic survey. In fact, we have been in discussions with all of them throughout, and are even starting a working group. The work we are doing to develop the economic report from the survey is more compelling to the groups/payers who will review it when it is done by a patient group, because it is not tagged as biased as by one pharmaceutical company. This is also so that we can collaborate and do things in a pre-competitive space so that the surveys we spend time on, the analysis that is done, is available to everyone who will pursue approval of a drug for AS.  This again makes Angelman syndrome the low hanging fruit for many pharmaceutical or biotechnology companies to be motivated to work in the AS space.

We want to be ready to be best situated to have access to approved drugs as soon as possible. It is important for us to think about the process of getting drugs to the community, which does not end with regulatory approvals, but includes insurance companies/payers/governments who make reimbursement decisions. Understanding what it really costs to care for an individual with AS is an important part of this process. That said, each pharma company will be in charge of the economic analysis regarding the safety and efficacy of their drug.   

Q: What is the difference between how FAST approaches this with regard to drugs that FAST has interests in and/or receives, or will receive, cash from, and those it won’t?

A: Since FAST’s mandate is broader than any one drug, FAST is dedicated to the long-term development of new therapies for the community as a whole being agnostic to the platform or the specific company sponsoring the program. 

It is GOOD news that the Angelman community has as many therapeutics in the pipeline as we do, and that there are more coming down the pike--and we have to chase them all. This is how every successful rare disease community works. For example, there are three oligonucleotides approved for patients with TTR mediated amyloidosis; 4 oligonucleotides for Duchenne muscular dystrophy; CF has 4 approved drugs. All of these rare diseases still have other new therapies in clinical trials because none of them have yet to reach a perfect solution. We, at FAST, expect that we will need to do the same and will work hard to ensure we find the best solution for every individual.

As for negotiating on behalf of patients, which we’ll do as much as we can in all areas, we actually have to be MORE circumspect when it comes to companies we have financial stakes in. FAST’s financial interest in GTX-102, for example, raises potential regulatory concerns.  

Q: Is FAST talking with pharma about price?

A: It is too early to do this now, but when the time comes we will do everything we can to help ensure equitable access for patients. Our understanding is that patient advocacy groups always do this. Patient groups frequently push for manufacturers to price low, of course, but ultimately pricing is up to each company who markets the drug and beyond our control. That’s why the key here is to focus first on ensuring insurers (public and private) fulfill their responsibility to insure/cover treatments for their insured population and that they understand how the drug cost compares to the standard cost of care for a patient. 

Q: Does this mean FAST won’t even TRY to negotiate with pharmaceutical companies on behalf of the community?

A: Not at all. It means this is only one step in the process! There is so much more we can do, and that we WILL do. We are looking ahead, and building the evidence story, because that’s the first step in this space. It will be our mission to promote widespread access to therapies for AS--regardless of age, regardless of genotype, and regardless of specific therapy. We hope you’ll join us as we work toward that future.