3 Takeaways From MassBio’s Policy Leadership Breakfast

Boston Massachusetts State House golden dome in USA

 

The agenda for MassBio’s 2016 Policy Leadership Breakfast was ambitious and provocative—and the conversation that resulted did not disappoint.

The program went directly at two of the most pressing issues we face as a Commonwealth—a growing opioid addiction epidemic and a healthcare system that can’t accommodate breakthrough therapies and cures.

Watch the entire program online. 

 

Three key takeaways:

1.  If anyone is going to eradicate opioid addiction, it’s us. 

We’ve got companies with treatments to reverse overdoses, to block pleasure receptors when someone does take drugs, and technologies to create tamper-resistant medicine. “There is no greater collection of minds and energies” to solve the opioid crisis than the stakeholders in MA, Attorney General Maura Healey said. And when Marylou Sudders, Secretary of Health and Human Services, shared her dream for a long-term solution—the development of a non-addictive painkiller—we had good news. Blue Therapeutics, a MassBio member and MassCONNECT alumni company is developing a painkiller that’s stronger than morphine but poses zero risk of abuse.

2. The Attorney General’s door is open, and we have to take her up on her offer to come talk. 

AG Healey was explicit—she wants to have “honest conversations” about cost and value and work with industry to ensure all patients have access to therapies and cures. She did share some of the factors she believed should factor in the equation, including accounting for the costs avoided when we cure otherwise chronic conditions and weighing quality of life benefits for patients and their families. But her comments on the impact of Solvaldi on the system and her letter to Gilead urging for discounted pricing for the Commonwealth’s public payers (released publicly yesterday morning) is a glimpse of how she intends to use her authority to shape the conversation and potentially public policy.

3. The pressures the healthcare system has faced in the last 18 months pale in comparison to what we can expect for a robust pipeline of personalized medicines and eventually gene therapy. 

Blue Cross Blue Shield of Massachusetts CEO Andrew Dreyfus said he does not know how to balance cost and access as specialty drugs continue to come to market (although the company is looking at all sorts of options, including indication-specific pricing and other new payment models, as well as new insurance products to reward low-cost solutions.) And ICER COO Sarah Emond admitted her organization’s new value framework is insufficient to measure one-time treatments like gene therapy. But we can’t throw up our hands in resignation. Instead, MassBio CEO Bob Coughlin called for a Payer-Provider-Industry Task Force “to develop solutions together and to bring those solutions forward to our partners in government.” And Dreyfus and Emond agreed. They even shook on it.

So now what?

From MassBio CEO Bob Coughlin’s challenge to industry at the start of the program: We can not shy away from difficult conversations, and we must educate and engage all stakeholders to find a path forward together.

And from MassBio Chairman Glenn Batchelder: It’s time to roll up our sleeves and find solutions, not just trade soundbites.

The time is now, members. We stand by to work hand-in-hand with all of you to tackle these issues.

Looking to get involved in MassBio’s ongoing Value initiatives? Contact Sarah MacDonald at sarah.macdonald@massbio.org or 617-674-5100.

 

Patient Voices: Dear FCS

Handwriting, hand  writes with a pen in a notebookA letter from Lindsey to her rare disease. Lindsey is a young woman living with Familial Chylomicronemia Syndrome, a rare genetic disorder in which the body does not break down fats correctly.

Help raise awareness for rare diseases and share her powerful letter. Don’t forget to join us on February 29th at the Massachusetts State House to recognize Rare Disease Day and the research being done in Massachusetts to treat and cure rare disease.

Dear FCS,

We sure have been through a lot together and after 25 years, I am still trying to figure you out. You make my life so hard almost every single day. I cannot go an hour without stressing, that because of you, I am going to get pancreatitis. I worry every single day about the symptoms you may bring and also how we are going to work together in the future. I want to children and not have to spend my entire pregnancy in the hospital and I definitely do not want my children to endure what I have had to.

Can you ever give me a break?! I think at this point, I deserve that. I know that I am nowhere near perfect when it comes to eating and I am the first to admit when I over do it. You tolerate those times when those brownies look too great to pass up, but really, I only have a bite and the next day you make me pay for it. In fact, I pay for it for multiple days, sometimes more than a week. The worst part is I have grown so accustomed to feeling so crummy every day that I have forgotten that it isn’t normal. At this age, I should feel so fantastic. You sure have helped me become a great actress because I will fake how I feel every day. I even have my family and closest friends fooled. Faking feeling well is mentally draining and I hate you for being the root of my health problems. You have truly succeeded in exhausting me. What I really don’t understand is even when I eat perfect (under 10 grams of fat, low carbohydrates, high protein, low sugar) how you are still unhappy.   I could eat salad and water and you’d find a way to cause that awful and all too familiar rib cage and scapula pain. I can’t take pain meds 24/7! I have things to do: work, spend time with friends/family…you know, have a normal life. I know you love to make me cancel plans and rest all day but life doesn’t work that way. Please show me some mercy. I am thankful I am not hospitalized as often but I just want to wake up and feel amazing. Please let me feel that way!

I don’t think you’re aware of how scared of you I am. You scare me more than anything because you hold my health in your hands. I have been hospitalized nearly more than 30 times, one attack almost taking my life. I know that I have caused a couple of those attacks with poor choices but the rest is all you. I know you have heard me cry myself to sleep countless nights, felt my anxiety and anger. I feel like I try and treat you so nicely but nothing works. You have caused me to shut down around family and best friend because I am so tired of seeing them worry about me all the time. I have seen what you have to my parents, especially when I was sick every 2 months. You have caused us hell – oh, and getting sick on my dad’s birthday a couple of weeks ago, that was really great. Thanks for that one, we really appreciated celebrating in the ER.

I think one of the worst things is that I don’t feel I have an outlet. When I am feeling really bad, I don’t want to be a burden on anyone. I don’t want people to ever think, “Oh, she feels sick again, she never feels well”. I hate that many people do not understand what it is like living with this disease. If you would not make me feel so bad, this problem would go away!

As much as I want to sit here and tell you how much I despise you, I can’t. Although I strongly dislike you, because of you, I have become so strong, an advocate, independent, incredibly positive.   I know no matter what, you will never stand in my way. At the end of the day, you are a part of me and I hope that as time continues, we can learn to better work with one another instead of against.

Lindsey, a young woman living with FCS (LPLD).

 

 

Top 5 News: 1/16-1/22

Top 5 logo1) Dozens of drug makers agree on effort to combat antibiotic resistance – STAT, 1/20/2016
In an unprecedented bid to address antibiotic resistance, more than 80 drug and device makers have issued a declaration urging governments to “support investment” in developing medicines and diagnostic tools to combat drug-resistant infections.
Link

2) Exclusive: Fast-growing gene-editing biotech expanding near Kendall Square BBJ, 1/22/2016
A rapidly growing Cambridge-based gene-editing company has snapped up 65,000 square feet of coveted East Cambridge real estate with plans to house a newly created division in the space.
Link

3) GE Life Sciences to hire 100 more people in 2016 BBJ, 1/21/2016
As General Electric Co. prepares to move to relocate corporate headquarters to Massachusetts, GE Healthcare Life Sciences is finalizing its own move into new space and planning to hire over 100 new employees.
Link

4) MassBio Testimony: Healthcare Financing Committee Hearing on Drug Cost & Value – MassBio, 1/19/2016
On January 19th, MassBio, at the request of the Joint Committee on Healthcare Financing, testified at the first in a series of informational sessions intended to provide legislators, stakeholders, and the public with a comprehensive understanding of issues of drug cost and value.
Link

5) Video: Experts predict the future of Big Data at #JPM16 – FierceBiotech, 1/19/2016
Storing, analyzing and managing the big data sets that are being created around genomics for drug development has created challenges for the biopharma industry. Some leaders in the field, including Google and the Broad Institute in Cambridge, MA, have been battering down some of these barriers, making it possible to explore the data for clues about new drug development opportunities.
Link

Washington Update: December 2015

Washington Dc

Lawmakers wrapped up 2015 with a flurry of legislative accomplishments. Legislators successfully passed a measure to fund the federal government through the remainder of Fiscal Year 2016 (FY16). Paired with the spending bill, Congress approved legislation renewing a host of expired tax extenders, and made a handful of those provisions permanent. Lawmakers also passed a multi-year Highway Bill that reauthorizes surface transportation programs for five years. December saw Congress delay several taxes related to the Affordable Care Act (ACA), reform cybersecurity laws, and pass resolutions condemning the Administration’s new carbon rules.

Contents

CONGRESSIONAL OUTLOOK FOR JANUARY AND 2016

ISSUE—GOVERNMENT FUNDING

ISSUE—TRANSPORTATION

ISSUE—TAX

ISSUE—HEALTH

ISSUE—CYBER SECURITY

ISSUE—PATENT REFORM

ISSUE—ENERGY

ISSUE—FOREIGN POLICY

OTHER ISSUES

RECENT POLLING

CONGRESSIONAL OUTLOOK FOR JANUARY AND 2016

Legislators effectively cleared the decks in December. The passage of several landmark pieces of legislation in 2015 allows Congress to enter 2016 with a relatively light agenda of must-do items.

The top priority for both chambers will be turning to the appropriations process for Fiscal Year 2017. The two-year budget deal that passed in October established the top line spending numbers for FY17, allowing appropriators to immediately begin work on the twelve annual spending bills. Both House Speaker Paul Ryan (R-WI) and Senate Majority Leader Mitch McConnell (R-KY) have vowed to return their chambers to regular order, with an orderly and timely appropriations process key to that goal. The ambitious aim of passing each individual appropriations bill across the House and Senate floor combined with the shortened summer session due to the earlier party conventions will lead to an aggressive schedule by the Appropriations Committees on both sides of the Capitol, with spending bills possibly hitting the House floor as early as March.

In the Senate, lawmakers are expected to turn to the customs bill conference report shortly after returning from the holiday break on January 11. The measure, which contains a range of provisions strengthening customs enforcement, passed the House in December. The House is expected to take up a legal reform bill and the Senate’s amended reconciliation bill when the chamber reconvenes on January 5.

January will also include President Obama’s last State of the Union address, which is scheduled for January 12. In his address, the President is expected to lay out his congressional agenda for his final year in office. The parties will also hold their planning retreats in January.

More broadly, 2016 is likely to be defined by election year politics, with the presidential campaign dominating the news cycle and influencing the congressional agenda. Four sitting Senators are seeking their party’s nomination (three Republicans, one Democrat). Additionally, Speaker Ryan has vowed to present an alternative for voters by developing a positive, message-driven GOP agenda, including renewed efforts on international tax reform.

ISSUE—GOVERNMENT FUNDING

Prior to the holidays, Congress successfully extended funding for the federal government through FY16 and took a step closer to sending the President reconciliation legislation that would repeal large parts of the ACA.

Budget and Government Funding

Congress approved an omnibus spending package in December renewing government funding through the end of September 2016. The legislation came after months of negotiations and the October two-year budget deal that increased spending caps on both domestic and defense spending for FY16 and FY17.

Final passage of the $1.1 trillion omnibus came after two months of intense negotiations. The bipartisan bill also included several policy changes. While Republicans pushed for a slew of policy riders, they walked away with fewer victories that they would have liked but touted the inclusion of provisions lifting the federal prohibition on exporting oil, reforms to the Visa Waiver Program, and pared back funding for the Environmental Protection Agency (EPA) as legislative victories. Democrats managed to prevent GOP attempts to include riders that would have defunded Planned Parenthood and imposed stricter controls on Syrian refugees entering the country, among many others.

The omnibus drew criticism from both Tea Party-aligned Republicans and progressive Democrats. Despite concerns from the left and right flanks, both chambers passed the legislation on a strong bipartisan vote. The bill was signed into law by the President on December 18.

Reconciliation and ACA Repeal

On December 3, the Senate approved a House-passed reconciliation package to repeal key portions of the ACA, including a rollback of the employer and individual mandates and repeal of federal subsidies for health insurance. The bill also defunds Planned Parenthood. Under reconciliation rules, the bill only needed a simple majority to pass the chamber, allowing it to avoid a Democratic filibuster. The final legislation passed on a vote of 52-47. The House will need to approve the Senate changes to the original measure. A vote on the amended legislation is expected to occur during the first week in January.

The bill’s passage will fulfill the GOP pledge to repeal President Obama’s signature legislative achievement. As expected, the White House has indicated that the President will veto the measure, and Republicans will not have the votes needed to override a veto. Read the rest of this entry

Patient Voices: Chris Anselmo

Chris Anselmo
Chris Anselmo shares his journey with dysferlinopathy, a rare, muscle-weakening disease. 

Be sure to share Chris’ story and join him and other rare disease patients on February 29th at the Massachusetts State House to recognize Rare Disease Day and the research being done in Massachusetts to treat and cure rare disease.


A Rare Diagnosis

It all started with a car accident.

Up until one fateful night in October 2003, I had no reason to think that I was living with a rare disease. For the first 17 years of my life, I was a healthy, active, fully functional high school senior with my eyes set on college and the next chapter in my life.

On that night, my friend was driving me home from the movies, when he became distracted by something he saw in the rear view mirror. For whatever reason, he thought the light had turned green. Seconds later, the car we were driving in was slammed into by a pickup truck traveling 45 miles per hour. Our car was a crumpled wreck, and the entire impact was on the passenger side where I was sitting.

It is a miracle that everyone involved emerged unscathed aside from a few cuts and bruises. It was a traumatic experience on its own, but it paled in comparison to the ordeal that was to come for me. Later that night, while I lay awake in my hospital bed, unable to sleep due to the pain on my right side, a doctor came in with news that would begin my patient journey.

I was in a haze from painkillers, so I don’t remember exactly what the doctor said, but I remember the concern on her face. The blood test I had done when I got to the emergency room came back with astronomically high levels of creatine kinase – a marker of muscle breakdown. Initially, they feared I had suffered some sort of internal injury, but it was ruled out after follow-up tests and I was sent home. Yet, in follow-up visits, the levels didn’t go down as they had initially hoped, which raised a red flag. Something was clearly wrong inside of me.

One year later, after testing for and ruling out liver disease, and after a biopsy of my left thigh, I was finally diagnosed with dysferlinopathy. It was explained to me that I was missing something called “dysferlin” – a protein that, if you don’t have it, eventually leads to muscular dystrophy. I was assured after initial fears that symptoms would not manifest until later in life, when there surely would be some sort of treatment. I was told to follow up with an adult neurology clinic, just to be safe, but it was nothing to be preoccupied with. I asked if I could go to college worry-free, and I was told I could.

Four years passed. I attended Northeastern University in Boston, and had a normal, exhilarating college experience. I was asymptomatic during this time, so naturally I let my guard down. I was naive, but in retrospect I am happy I was. If I fully understood the magnitude of what was to come, I would have been devastated. By May 2008, I had graduated from Northeastern with a degree in marketing. It was then that I began to experience weakness, as if a flip had switched inside my body.

With a muscle-weakening disease, you can tell when things are starting to “go.” You remember these indelible, traumatic events because they are seared into your mind. I’ve seen others refer to them as “milestones,” and unfortunately, it is a fitting metaphor. They stay with you, and no matter how much you want them to, they don’t go away.

Milestones

My first milestone where I knew something was wrong was in late 2008 when I went for a run, turned a corner, and my legs tired out. I thought it was odd, but I didn’t think much of it. A few weeks later I went running again, only I couldn’t make it as far as my previous run. For whatever reason, I didn’t realize that it was because of my disease. I was only 22; it wasn’t supposed to happen until much, much later. I chalked it up as being out of shape, and figured that I just didn’t gain strength as fast because of this missing protein.

My second milestone happened about a year later, when I moved with my college buddies to a three-story house in Boston. I was carrying my desk chair up the stairs and was having great difficulty. I had to stop every few steps to regain my strength and balance. I remember it well, because it was at this point where I began to put the pieces together. I remember the unsettling feeling in my stomach, the joy of moving to a new apartment now replaced by anxiety and fear.

The Fall

A year after moving in I experienced my first fall – by far the most traumatic milestone of my life.

It was a Saturday morning. I was walking to the store with my roommate, took a step, and my right leg gave out, causing me to crumple into a heap on the sidewalk. My roommate stopped and asked if I was OK. I told him I didn’t know, but that was a lie. I knew. The emotional pain that resulted was far worse than the physical pain of scraping my knee.

Falling and the sudden reality that I couldn’t get from Point A to Point B without the possibility of crumbling to the ground turned my world upside down. It affected my outlook on life and my relationships with others. It affected how I perceived my future, my attitude and my productivity at work. Every facet of my life suffered with this new reality. It was a terrible time.

Since that day, I have fallen countless more times, ruining every pair of jeans I’ve owned because I inevitably tear holes in the knees on the pavement. When I do fall, I need someone to help me, as I no longer have the strength to get back up. I’ve progressed to Lofstrand crutches and have given up going on long walks, which was very tough as I used to love exploring new neighborhoods in Boston. Stairs are my mortal enemy and can’t be navigated. As of the last few months, I can’t get out of chairs (unless it’s a high chair) without someone picking me up under my armpits.

Picking Myself Back Up

This has all been very difficult to deal with, as the dreams I once had for myself as a young adult have been derailed by unrelenting muscle weakness. Despite the trials and setbacks, this disease taught me an invaluable skill – how to pick myself back up. Without learning resilience I would have given up on my future.

One day I had enough. I came to the conclusion that I wasn’t doing myself any good by being miserable and comparing myself to others my age who seemingly had everything. I sought a way to use my experience as a positive. Through sheer determination, a fire was kindled inside of me to make a better life for myself than I would have had without the disease. I sought ways to get involved and make a difference in finding a cure.

I became more involved with the Jain Foundation, an organization dedicated to finding a cure for dysferlinopathy. I was given the opportunity to share my patient story at the International Dysferlin Conference in April 2013, a huge thrill that cultivated my passion for public speaking. From connections made at that conference, I was introduced to three researchers in the Boston area who have family members with muscular dystrophy. In November 2014 we hosted the second annual Strength, Science and Stories of Inspiration fundraiser at the MIT Museum in Cambridge. It brought together stakeholders in the muscle disease community, and I had the opportunity to share my patient story in front of 200 people. It was the most rewarding night of my life.

I still struggle with the effects of my disease. I accept that I am always going to be “day-to-day”, both physically and emotionally. Any time I experience a new milestone, I regress into a funk, but I have risen every time. Although some dreams are unachievable, I haven’t let my disease stop me from dreaming big. After years of shuffling my feet, I returned to school, and am currently an MBA candidate at Boston College, graduating in May. It has been a long, strange, difficult journey. Through continued funding and research, I know that brighter days are ahead.

Dysferlinopathy has given me a clear purpose for what I want to do in life – to use my story to help others. A purpose for living is incredibly powerful, and I know that without this disease, there is no way I would have the ambition and determination I have today. I am beginning to experience the fruits of my struggle (it is about time!), and that has given me great joy. I have my first speaking engagement at a high school in Connecticut in late February, and four days later will be speaking at Rare Disease Day at the Massachusetts State House.

I always think that someday this will all be worth it, but I am starting to realize that day is already here.